MX2012009318A - Metodos y compuestos para el crecimiento muscular. - Google Patents

Info

Publication number

MX2012009318A

MX2012009318A MX2012009318A MX2012009318A MX2012009318A MX 2012009318 A MX2012009318 A MX 2012009318A MX 2012009318 A MX2012009318 A MX 2012009318A MX 2012009318 A MX2012009318 A MX 2012009318A MX 2012009318 A MX2012009318 A MX 2012009318A

Authority

MX

Mexico

Prior art keywords

fbxo40

antagonist

antibody

muscle

sirna

Prior art date

2010-02-10

Links

• Espacenet
• Global Dossier
• Discuss

• 238000000034 method Methods 0.000 title claims abstract description 183
• 150000001875 compounds Chemical class 0.000 title abstract description 33
• 230000037257 muscle growth Effects 0.000 title description 11
• 239000005557 antagonist Substances 0.000 claims abstract description 150
• 210000003205 muscle Anatomy 0.000 claims abstract description 143
• 108090000623 proteins and genes Proteins 0.000 claims abstract description 141
• 230000000694 effects Effects 0.000 claims abstract description 93
• 102000004169 proteins and genes Human genes 0.000 claims abstract description 92
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims abstract description 81
• 150000007523 nucleic acids Chemical class 0.000 claims abstract description 80
• 102000039446 nucleic acids Human genes 0.000 claims abstract description 80
• 108020004707 nucleic acids Proteins 0.000 claims abstract description 80
• 230000014509 gene expression Effects 0.000 claims abstract description 71
• 108020004459 Small interfering RNA Proteins 0.000 claims abstract description 70
• 208000035475 disorder Diseases 0.000 claims abstract description 57
• 201000000585 muscular atrophy Diseases 0.000 claims abstract description 54
• 206010028289 Muscle atrophy Diseases 0.000 claims abstract description 48
• 238000012216 screening Methods 0.000 claims abstract description 18
• 238000012423 maintenance Methods 0.000 claims abstract description 6
• 238000012544 monitoring process Methods 0.000 claims abstract description 4
• 239000000203 mixture Substances 0.000 claims description 85
• 238000011282 treatment Methods 0.000 claims description 66
• 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 54
• 150000001413 amino acids Chemical class 0.000 claims description 39
• 239000003112 inhibitor Substances 0.000 claims description 37
• 229940024606 amino acid Drugs 0.000 claims description 32
• 230000007423 decrease Effects 0.000 claims description 30
• 150000003384 small molecules Chemical group 0.000 claims description 30
• 206010028980 Neoplasm Diseases 0.000 claims description 28
• 206010006895 Cachexia Diseases 0.000 claims description 24
• -1 flavone glycoside Chemical class 0.000 claims description 24
• 208000001076 sarcopenia Diseases 0.000 claims description 19
• NOESYZHRGYRDHS-UHFFFAOYSA-N insulin Chemical compound N1C(=O)C(NC(=O)C(CCC(N)=O)NC(=O)C(CCC(O)=O)NC(=O)C(C(C)C)NC(=O)C(NC(=O)CN)C(C)CC)CSSCC(C(NC(CO)C(=O)NC(CC(C)C)C(=O)NC(CC=2C=CC(O)=CC=2)C(=O)NC(CCC(N)=O)C(=O)NC(CC(C)C)C(=O)NC(CCC(O)=O)C(=O)NC(CC(N)=O)C(=O)NC(CC=2C=CC(O)=CC=2)C(=O)NC(CSSCC(NC(=O)C(C(C)C)NC(=O)C(CC(C)C)NC(=O)C(CC=2C=CC(O)=CC=2)NC(=O)C(CC(C)C)NC(=O)C(C)NC(=O)C(CCC(O)=O)NC(=O)C(C(C)C)NC(=O)C(CC(C)C)NC(=O)C(CC=2NC=NC=2)NC(=O)C(CO)NC(=O)CNC2=O)C(=O)NCC(=O)NC(CCC(O)=O)C(=O)NC(CCCNC(N)=N)C(=O)NCC(=O)NC(CC=3C=CC=CC=3)C(=O)NC(CC=3C=CC=CC=3)C(=O)NC(CC=3C=CC(O)=CC=3)C(=O)NC(C(C)O)C(=O)N3C(CCC3)C(=O)NC(CCCCN)C(=O)NC(C)C(O)=O)C(=O)NC(CC(N)=O)C(O)=O)=O)NC(=O)C(C(C)CC)NC(=O)C(CO)NC(=O)C(C(C)O)NC(=O)C1CSSCC2NC(=O)C(CC(C)C)NC(=O)C(NC(=O)C(CCC(N)=O)NC(=O)C(CC(N)=O)NC(=O)C(NC(=O)C(N)CC=1C=CC=CC=1)C(C)C)CC1=CN=CN1 NOESYZHRGYRDHS-UHFFFAOYSA-N 0.000 claims description 18
• 108090000765 processed proteins & peptides Proteins 0.000 claims description 18
• 102000018700 F-Box Proteins Human genes 0.000 claims description 16
• 201000011510 cancer Diseases 0.000 claims description 16
• 102000004196 processed proteins & peptides Human genes 0.000 claims description 16
• 102000005962 receptors Human genes 0.000 claims description 16
• 108020003175 receptors Proteins 0.000 claims description 16
• 108010066805 F-Box Proteins Proteins 0.000 claims description 15
• 108060008682 Tumor Necrosis Factor Proteins 0.000 claims description 14
• 102000000852 Tumor Necrosis Factor-alpha Human genes 0.000 claims description 14
• 208000013738 Sleep Initiation and Maintenance disease Diseases 0.000 claims description 13
• 206010022437 insomnia Diseases 0.000 claims description 13
• 102100026992 Dermcidin Human genes 0.000 claims description 12
• 101000911659 Homo sapiens Dermcidin Proteins 0.000 claims description 12
• 229920001184 polypeptide Polymers 0.000 claims description 12
• 208000016261 weight loss Diseases 0.000 claims description 12
• 230000004580 weight loss Effects 0.000 claims description 12
• 230000001419 dependent effect Effects 0.000 claims description 11
• 206010012601 diabetes mellitus Diseases 0.000 claims description 11
• 239000002243 precursor Substances 0.000 claims description 11
• ROHFNLRQFUQHCH-YFKPBYRVSA-N L-leucine Chemical compound CC(C)C[C@H](N)C(O)=O ROHFNLRQFUQHCH-YFKPBYRVSA-N 0.000 claims description 10
• ROHFNLRQFUQHCH-UHFFFAOYSA-N Leucine Natural products CC(C)CC(N)C(O)=O ROHFNLRQFUQHCH-UHFFFAOYSA-N 0.000 claims description 10
• 206010040047 Sepsis Diseases 0.000 claims description 10
• MUMGGOZAMZWBJJ-DYKIIFRCSA-N Testostosterone Chemical compound O=C1CC[C@]2(C)[C@H]3CC[C@](C)([C@H](CC4)O)[C@@H]4[C@@H]3CCC2=C1 MUMGGOZAMZWBJJ-DYKIIFRCSA-N 0.000 claims description 10
• HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims description 10
• 230000001010 compromised effect Effects 0.000 claims description 10
• 102000004877 Insulin Human genes 0.000 claims description 9
• 108090001061 Insulin Proteins 0.000 claims description 9
• 208000002193 Pain Diseases 0.000 claims description 9
• 102000043299 Parathyroid hormone-related Human genes 0.000 claims description 9
• 101710123753 Parathyroid hormone-related protein Proteins 0.000 claims description 9
• 208000018737 Parkinson disease Diseases 0.000 claims description 9
• 239000000284 extract Substances 0.000 claims description 9
• 229940125396 insulin Drugs 0.000 claims description 9
• 206010039073 rheumatoid arthritis Diseases 0.000 claims description 9
• 206010008748 Chorea Diseases 0.000 claims description 8
• 208000012661 Dyskinesia Diseases 0.000 claims description 8
• 206010016654 Fibrosis Diseases 0.000 claims description 8
• 206010020772 Hypertension Diseases 0.000 claims description 8
• 208000013016 Hypoglycemia Diseases 0.000 claims description 8
• 208000012601 choreatic disease Diseases 0.000 claims description 8
• 230000007882 cirrhosis Effects 0.000 claims description 8
• 208000019425 cirrhosis of liver Diseases 0.000 claims description 8
• 230000002596 correlated effect Effects 0.000 claims description 8
• 206010013663 drug dependence Diseases 0.000 claims description 8
• 239000003102 growth factor Substances 0.000 claims description 8
• 230000002218 hypoglycaemic effect Effects 0.000 claims description 8
• 208000017169 kidney disease Diseases 0.000 claims description 8
• 230000003908 liver function Effects 0.000 claims description 8
• 208000011117 substance-related disease Diseases 0.000 claims description 8
• 108090000712 Cathepsin B Proteins 0.000 claims description 7
• 102000004225 Cathepsin B Human genes 0.000 claims description 7
• 101710186630 Insulin-1 Proteins 0.000 claims description 7
• 230000001965 increasing effect Effects 0.000 claims description 7
• 150000003626 triacylglycerols Chemical class 0.000 claims description 7
• 208000030507 AIDS Diseases 0.000 claims description 6
• 208000037157 Azotemia Diseases 0.000 claims description 6
• 102100030013 Endoribonuclease Human genes 0.000 claims description 6
• 101710199605 Endoribonuclease Proteins 0.000 claims description 6
• 101710113029 Serine/threonine-protein kinase Proteins 0.000 claims description 6
• 230000001684 chronic effect Effects 0.000 claims description 6
• 208000009852 uremia Diseases 0.000 claims description 6
• AXFYFNCPONWUHW-UHFFFAOYSA-N 3-hydroxyisovaleric acid Chemical compound CC(C)(O)CC(O)=O AXFYFNCPONWUHW-UHFFFAOYSA-N 0.000 claims description 5
• BKAJNAXTPSGJCU-UHFFFAOYSA-N 4-methyl-2-oxopentanoic acid Chemical compound CC(C)CC(=O)C(O)=O BKAJNAXTPSGJCU-UHFFFAOYSA-N 0.000 claims description 5
• 229930000680 A04AD01 - Scopolamine Natural products 0.000 claims description 5
• PQSUYGKTWSAVDQ-ZVIOFETBSA-N Aldosterone Chemical compound C([C@@]1([C@@H](C(=O)CO)CC[C@H]1[C@@H]1CC2)C=O)[C@H](O)[C@@H]1[C@]1(C)C2=CC(=O)CC1 PQSUYGKTWSAVDQ-ZVIOFETBSA-N 0.000 claims description 5
• PQSUYGKTWSAVDQ-UHFFFAOYSA-N Aldosterone Natural products C1CC2C3CCC(C(=O)CO)C3(C=O)CC(O)C2C2(C)C1=CC(=O)CC2 PQSUYGKTWSAVDQ-UHFFFAOYSA-N 0.000 claims description 5
• 108090000227 Chymases Proteins 0.000 claims description 5
• 102000003858 Chymases Human genes 0.000 claims description 5
• 102000010180 Endothelin receptor Human genes 0.000 claims description 5
• 108050001739 Endothelin receptor Proteins 0.000 claims description 5
• 102000003951 Erythropoietin Human genes 0.000 claims description 5
• 108090000394 Erythropoietin Proteins 0.000 claims description 5
• 102100040669 F-box only protein 32 Human genes 0.000 claims description 5
• 101710191029 F-box only protein 32 Proteins 0.000 claims description 5
• 244000194101 Ginkgo biloba Species 0.000 claims description 5
• 108010051696 Growth Hormone Proteins 0.000 claims description 5
• 206010019280 Heart failures Diseases 0.000 claims description 5
• STECJAGHUSJQJN-GAUPFVANSA-N Hyoscine Natural products C1([C@H](CO)C(=O)OC2C[C@@H]3N([C@H](C2)[C@@H]2[C@H]3O2)C)=CC=CC=C1 STECJAGHUSJQJN-GAUPFVANSA-N 0.000 claims description 5
• 102000009032 Imidazoline Receptors Human genes 0.000 claims description 5
• 108010049134 Imidazoline Receptors Proteins 0.000 claims description 5
• 102000014150 Interferons Human genes 0.000 claims description 5
• 108010050904 Interferons Proteins 0.000 claims description 5
• AFVFQIVMOAPDHO-UHFFFAOYSA-N Methanesulfonic acid Chemical compound CS(O)(=O)=O AFVFQIVMOAPDHO-UHFFFAOYSA-N 0.000 claims description 5
• 108010056852 Myostatin Proteins 0.000 claims description 5
• STECJAGHUSJQJN-UHFFFAOYSA-N N-Methyl-scopolamin Natural products C1C(C2C3O2)N(C)C3CC1OC(=O)C(CO)C1=CC=CC=C1 STECJAGHUSJQJN-UHFFFAOYSA-N 0.000 claims description 5
• 108010044843 Peptide Initiation Factors Proteins 0.000 claims description 5
• 102000005877 Peptide Initiation Factors Human genes 0.000 claims description 5
• LCTONWCANYUPML-UHFFFAOYSA-M Pyruvate Chemical compound CC(=O)C([O-])=O LCTONWCANYUPML-UHFFFAOYSA-M 0.000 claims description 5
• GAMYVSCDDLXAQW-AOIWZFSPSA-N Thermopsosid Natural products O(C)c1c(O)ccc(C=2Oc3c(c(O)cc(O[C@H]4[C@H](O)[C@@H](O)[C@H](O)[C@H](CO)O4)c3)C(=O)C=2)c1 GAMYVSCDDLXAQW-AOIWZFSPSA-N 0.000 claims description 5
• 102100033220 Xanthine oxidase Human genes 0.000 claims description 5
• 108010093894 Xanthine oxidase Proteins 0.000 claims description 5
• 229960002478 aldosterone Drugs 0.000 claims description 5
• 235000005550 amino acid supplement Nutrition 0.000 claims description 5
• 102000012740 beta Adrenergic Receptors Human genes 0.000 claims description 5
• 108010079452 beta Adrenergic Receptors Proteins 0.000 claims description 5
• 150000005693 branched-chain amino acids Chemical class 0.000 claims description 5
• 229940105423 erythropoietin Drugs 0.000 claims description 5
• 229930003944 flavone Natural products 0.000 claims description 5
• 235000011949 flavones Nutrition 0.000 claims description 5
• 229930184727 ginkgolide Natural products 0.000 claims description 5
• 229930182470 glycoside Natural products 0.000 claims description 5
• 239000000122 growth hormone Substances 0.000 claims description 5
• 239000003324 growth hormone secretagogue Substances 0.000 claims description 5
• 229940088597 hormone Drugs 0.000 claims description 5
• 239000005556 hormone Substances 0.000 claims description 5
• 229940079322 interferon Drugs 0.000 claims description 5
• 230000001537 neural effect Effects 0.000 claims description 5
• 230000002232 neuromuscular Effects 0.000 claims description 5
• 229940127240 opiate Drugs 0.000 claims description 5
• OXCMYAYHXIHQOA-UHFFFAOYSA-N potassium;[2-butyl-5-chloro-3-[[4-[2-(1,2,4-triaza-3-azanidacyclopenta-1,4-dien-5-yl)phenyl]phenyl]methyl]imidazol-4-yl]methanol Chemical compound [K+].CCCCC1=NC(Cl)=C(CO)N1CC1=CC=C(C=2C(=CC=CC=2)C2=N[N-]N=N2)C=C1 OXCMYAYHXIHQOA-UHFFFAOYSA-N 0.000 claims description 5
• STECJAGHUSJQJN-FWXGHANASA-N scopolamine Chemical compound C1([C@@H](CO)C(=O)O[C@H]2C[C@@H]3N([C@H](C2)[C@@H]2[C@H]3O2)C)=CC=CC=C1 STECJAGHUSJQJN-FWXGHANASA-N 0.000 claims description 5
• 229960002646 scopolamine Drugs 0.000 claims description 5
• 208000011580 syndromic disease Diseases 0.000 claims description 5
• 229960003604 testosterone Drugs 0.000 claims description 5
• VHBFFQKBGNRLFZ-UHFFFAOYSA-N vitamin p Natural products O1C2=CC=CC=C2C(=O)C=C1C1=CC=CC=C1 VHBFFQKBGNRLFZ-UHFFFAOYSA-N 0.000 claims description 5
• 102000008873 Angiotensin II receptor Human genes 0.000 claims description 4
• 108050000824 Angiotensin II receptor Proteins 0.000 claims description 4
• 102100033367 Appetite-regulating hormone Human genes 0.000 claims description 4
• 101710111255 Appetite-regulating hormone Proteins 0.000 claims description 4
• 206010007558 Cardiac failure chronic Diseases 0.000 claims description 4
• 102100025014 E3 ubiquitin-protein ligase TRIM63 Human genes 0.000 claims description 4
• 101710164910 E3 ubiquitin-protein ligase TRIM63 Proteins 0.000 claims description 4
• 150000004728 pyruvic acid derivatives Chemical class 0.000 claims description 4
• 150000003431 steroids Chemical class 0.000 claims description 4
• 230000000638 stimulation Effects 0.000 claims description 4
• 238000000554 physical therapy Methods 0.000 claims description 3
• 230000002829 reductive effect Effects 0.000 claims description 3
• 102000003982 Parathyroid hormone Human genes 0.000 claims description 2
• 108090000445 Parathyroid hormone Proteins 0.000 claims description 2
• 201000006370 kidney failure Diseases 0.000 claims description 2
• 235000015097 nutrients Nutrition 0.000 claims description 2
• 239000000199 parathyroid hormone Substances 0.000 claims description 2
• 229960001319 parathyroid hormone Drugs 0.000 claims description 2
• 102000018997 Growth Hormone Human genes 0.000 claims 2
• 102000004472 Myostatin Human genes 0.000 claims 2
• 208000029483 Acquired immunodeficiency Diseases 0.000 claims 1
• 210000000232 gallbladder Anatomy 0.000 claims 1
• 230000002401 inhibitory effect Effects 0.000 abstract description 19
• 210000004027 cell Anatomy 0.000 description 115
• 235000018102 proteins Nutrition 0.000 description 87
• 108010034219 Insulin Receptor Substrate Proteins Proteins 0.000 description 85
• 102000009433 Insulin Receptor Substrate Proteins Human genes 0.000 description 85
• 108020004999 messenger RNA Proteins 0.000 description 70
• 241000282414 Homo sapiens Species 0.000 description 62
• 230000027455 binding Effects 0.000 description 61
• 239000000523 sample Substances 0.000 description 41
• 125000003729 nucleotide group Chemical group 0.000 description 40
• 230000000692 anti-sense effect Effects 0.000 description 37
• 239000002773 nucleotide Substances 0.000 description 37
• 108010047041 Complementarity Determining Regions Proteins 0.000 description 36
• 235000001014 amino acid Nutrition 0.000 description 34
• 230000002068 genetic effect Effects 0.000 description 31
• 239000000427 antigen Substances 0.000 description 29
• 108091007433 antigens Proteins 0.000 description 29
• 102000036639 antigens Human genes 0.000 description 29
• 239000003814 drug Substances 0.000 description 29
• 102000040650 (ribonucleotides)n+m Human genes 0.000 description 28
• 230000015556 catabolic process Effects 0.000 description 26
• 238000006731 degradation reaction Methods 0.000 description 26
• 230000004048 modification Effects 0.000 description 26
• 238000012986 modification Methods 0.000 description 26
• 238000012228 RNA interference-mediated gene silencing Methods 0.000 description 24
• 201000010099 disease Diseases 0.000 description 24
• 230000009368 gene silencing by RNA Effects 0.000 description 24
• 239000002585 base Substances 0.000 description 23
• 229940079593 drug Drugs 0.000 description 23
• 102100023877 E3 ubiquitin-protein ligase RBX1 Human genes 0.000 description 22
• 101710095156 E3 ubiquitin-protein ligase RBX1 Proteins 0.000 description 22
• 101710178916 RING-box protein 1 Proteins 0.000 description 22
• 230000000295 complement effect Effects 0.000 description 22
• 230000008030 elimination Effects 0.000 description 19
• 238000003379 elimination reaction Methods 0.000 description 19
• 239000012634 fragment Substances 0.000 description 19
• LOKCTEFSRHRXRJ-UHFFFAOYSA-I dipotassium trisodium dihydrogen phosphate hydrogen phosphate dichloride Chemical compound P(=O)(O)(O)[O-].[K+].P(=O)(O)([O-])[O-].[Na+].[Na+].[Cl-].[K+].[Cl-].[Na+] LOKCTEFSRHRXRJ-UHFFFAOYSA-I 0.000 description 18
• 239000002953 phosphate buffered saline Substances 0.000 description 18
• 238000000338 in vitro Methods 0.000 description 17
• 210000001519 tissue Anatomy 0.000 description 17
• 108060003951 Immunoglobulin Proteins 0.000 description 16
• 241001465754 Metazoa Species 0.000 description 16
• 102000018358 immunoglobulin Human genes 0.000 description 16
• 210000002966 serum Anatomy 0.000 description 16
• 238000006467 substitution reaction Methods 0.000 description 16
• 238000012360 testing method Methods 0.000 description 16
• 108020004414 DNA Proteins 0.000 description 15
• 206010028311 Muscle hypertrophy Diseases 0.000 description 15
• 238000001514 detection method Methods 0.000 description 15
• 230000012042 muscle hypertrophy Effects 0.000 description 15
• 230000014616 translation Effects 0.000 description 14
• 241000283973 Oryctolagus cuniculus Species 0.000 description 13
• 102000000341 S-Phase Kinase-Associated Proteins Human genes 0.000 description 13
• 108010055623 S-Phase Kinase-Associated Proteins Proteins 0.000 description 13
• 238000006243 chemical reaction Methods 0.000 description 13
• 238000001727 in vivo Methods 0.000 description 13
• 230000005764 inhibitory process Effects 0.000 description 13
• 238000003752 polymerase chain reaction Methods 0.000 description 13
• 150000003839 salts Chemical class 0.000 description 13
• 241000701161 unidentified adenovirus Species 0.000 description 13
• 239000003153 chemical reaction reagent Substances 0.000 description 12
• 238000005516 engineering process Methods 0.000 description 12
• 230000006870 function Effects 0.000 description 12
• 239000002502 liposome Substances 0.000 description 12
• 239000000126 substance Substances 0.000 description 12
• 108010067306 Fibronectins Proteins 0.000 description 11
• 241000700159 Rattus Species 0.000 description 11
• 238000002474 experimental method Methods 0.000 description 11
• 238000005259 measurement Methods 0.000 description 11
• 230000001105 regulatory effect Effects 0.000 description 11
• 238000001262 western blot Methods 0.000 description 11
• 241000699666 Mus <mouse, genus> Species 0.000 description 10
• 239000002253 acid Substances 0.000 description 10
• 238000003556 assay Methods 0.000 description 10
• 210000004602 germ cell Anatomy 0.000 description 10
• 210000002027 skeletal muscle Anatomy 0.000 description 10
• 238000010798 ubiquitination Methods 0.000 description 10
• 108091023037 Aptamer Proteins 0.000 description 9
• 102100037362 Fibronectin Human genes 0.000 description 9
• 241000699670 Mus sp. Species 0.000 description 9
• 208000021642 Muscular disease Diseases 0.000 description 9
• 108091034117 Oligonucleotide Proteins 0.000 description 9
• 239000011324 bead Substances 0.000 description 9
• 230000008859 change Effects 0.000 description 9
• 239000003795 chemical substances by application Substances 0.000 description 9
• 230000004069 differentiation Effects 0.000 description 9
• 238000002347 injection Methods 0.000 description 9
• 239000007924 injection Substances 0.000 description 9
• 230000003993 interaction Effects 0.000 description 9
• 230000000670 limiting effect Effects 0.000 description 9
• 238000012384 transportation and delivery Methods 0.000 description 9
• 230000034512 ubiquitination Effects 0.000 description 9
• IJGRMHOSHXDMSA-UHFFFAOYSA-N Atomic nitrogen Chemical compound N#N IJGRMHOSHXDMSA-UHFFFAOYSA-N 0.000 description 8
• 101100066410 Mus musculus Fbxo40 gene Proteins 0.000 description 8
• 201000009623 Myopathy Diseases 0.000 description 8
• 108010003723 Single-Domain Antibodies Proteins 0.000 description 8
• 125000003275 alpha amino acid group Chemical group 0.000 description 8
• 238000002372 labelling Methods 0.000 description 8
• 239000000463 material Substances 0.000 description 8
• 239000012528 membrane Substances 0.000 description 8
• 238000002360 preparation method Methods 0.000 description 8
• 208000002320 spinal muscular atrophy Diseases 0.000 description 8
• 239000000758 substrate Substances 0.000 description 8
• 230000001225 therapeutic effect Effects 0.000 description 8
• 238000013518 transcription Methods 0.000 description 8
• XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 8
• 239000006144 Dulbecco’s modified Eagle's medium Substances 0.000 description 7
• 102000004190 Enzymes Human genes 0.000 description 7
• 108090000790 Enzymes Proteins 0.000 description 7
• 102000006275 Ubiquitin-Protein Ligases Human genes 0.000 description 7
• 108010083111 Ubiquitin-Protein Ligases Proteins 0.000 description 7
• 230000015572 biosynthetic process Effects 0.000 description 7
• 238000002405 diagnostic procedure Methods 0.000 description 7
• 229940088598 enzyme Drugs 0.000 description 7
• 230000013595 glycosylation Effects 0.000 description 7
• 238000006206 glycosylation reaction Methods 0.000 description 7
• 210000002414 leg Anatomy 0.000 description 7
• 239000003446 ligand Substances 0.000 description 7
• 210000004165 myocardium Anatomy 0.000 description 7
• 210000001087 myotubule Anatomy 0.000 description 7
• 238000003199 nucleic acid amplification method Methods 0.000 description 7
• 230000037361 pathway Effects 0.000 description 7
• 238000001243 protein synthesis Methods 0.000 description 7
• 230000004044 response Effects 0.000 description 7
• 238000002560 therapeutic procedure Methods 0.000 description 7
• 230000035897 transcription Effects 0.000 description 7
• 238000001890 transfection Methods 0.000 description 7
• 238000013519 translation Methods 0.000 description 7
• 238000011269 treatment regimen Methods 0.000 description 7
• 239000013598 vector Substances 0.000 description 7
• 241000282472 Canis lupus familiaris Species 0.000 description 6
• 108091035707 Consensus sequence Proteins 0.000 description 6
• 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 description 6
• LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 description 6
• 241000287828 Gallus gallus Species 0.000 description 6
• 102000002254 Glycogen Synthase Kinase 3 Human genes 0.000 description 6
• 108010014905 Glycogen Synthase Kinase 3 Proteins 0.000 description 6
• 108010021625 Immunoglobulin Fragments Proteins 0.000 description 6
• 102000008394 Immunoglobulin Fragments Human genes 0.000 description 6
• 229910019142 PO4 Inorganic materials 0.000 description 6
• 241001494479 Pecora Species 0.000 description 6
• DNIAPMSPPWPWGF-UHFFFAOYSA-N Propylene glycol Chemical compound CC(O)CO DNIAPMSPPWPWGF-UHFFFAOYSA-N 0.000 description 6
• FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 6
• 108090000848 Ubiquitin Proteins 0.000 description 6
• 102000044159 Ubiquitin Human genes 0.000 description 6
• 150000007513 acids Chemical class 0.000 description 6
• 230000003321 amplification Effects 0.000 description 6
• 238000004458 analytical method Methods 0.000 description 6
• 238000010171 animal model Methods 0.000 description 6
• 230000008499 blood brain barrier function Effects 0.000 description 6
• 210000001218 blood-brain barrier Anatomy 0.000 description 6
• 238000007385 chemical modification Methods 0.000 description 6
• 230000021615 conjugation Effects 0.000 description 6
• 230000007812 deficiency Effects 0.000 description 6
• 238000012217 deletion Methods 0.000 description 6
• 230000037430 deletion Effects 0.000 description 6
• 239000012530 fluid Substances 0.000 description 6
• 239000000499 gel Substances 0.000 description 6
• 238000001802 infusion Methods 0.000 description 6
• 125000005647 linker group Chemical group 0.000 description 6
• 239000010452 phosphate Substances 0.000 description 6
• 229920001223 polyethylene glycol Polymers 0.000 description 6
• 238000003127 radioimmunoassay Methods 0.000 description 6
• 230000009467 reduction Effects 0.000 description 6
• 239000007790 solid phase Substances 0.000 description 6
• 241000894007 species Species 0.000 description 6
• GPRLSGONYQIRFK-MNYXATJNSA-N triton Chemical compound [3H+] GPRLSGONYQIRFK-MNYXATJNSA-N 0.000 description 6
• 229940035893 uracil Drugs 0.000 description 6
• 206010003694 Atrophy Diseases 0.000 description 5
• 108090000994 Catalytic RNA Proteins 0.000 description 5
• 102000053642 Catalytic RNA Human genes 0.000 description 5
• 108091026890 Coding region Proteins 0.000 description 5
• IAZDPXIOMUYVGZ-UHFFFAOYSA-N Dimethylsulphoxide Chemical compound CS(C)=O IAZDPXIOMUYVGZ-UHFFFAOYSA-N 0.000 description 5
• 238000002965 ELISA Methods 0.000 description 5
• 241000196324 Embryophyta Species 0.000 description 5
• 102000003746 Insulin Receptor Human genes 0.000 description 5
• 108010001127 Insulin Receptor Proteins 0.000 description 5
• 229940124647 MEK inhibitor Drugs 0.000 description 5
• 241000282560 Macaca mulatta Species 0.000 description 5
• 238000000636 Northern blotting Methods 0.000 description 5
• 108091000080 Phosphotransferase Proteins 0.000 description 5
• 239000002202 Polyethylene glycol Substances 0.000 description 5
• 238000010240 RT-PCR analysis Methods 0.000 description 5
• 241000282898 Sus scrofa Species 0.000 description 5
• 230000004913 activation Effects 0.000 description 5
• 230000037444 atrophy Effects 0.000 description 5
• 230000004071 biological effect Effects 0.000 description 5
• 239000013592 cell lysate Substances 0.000 description 5
• 230000000875 corresponding effect Effects 0.000 description 5
• 231100000599 cytotoxic agent Toxicity 0.000 description 5
• 230000006378 damage Effects 0.000 description 5
• 208000020694 gallbladder disease Diseases 0.000 description 5
• 230000030279 gene silencing Effects 0.000 description 5
• 230000001900 immune effect Effects 0.000 description 5
• 229940127121 immunoconjugate Drugs 0.000 description 5
• 238000001114 immunoprecipitation Methods 0.000 description 5
• 239000007788 liquid Substances 0.000 description 5
• 238000004519 manufacturing process Methods 0.000 description 5
• 238000004949 mass spectrometry Methods 0.000 description 5
• 239000002609 medium Substances 0.000 description 5
• NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 description 5
• 230000026731 phosphorylation Effects 0.000 description 5
• 238000006366 phosphorylation reaction Methods 0.000 description 5
• 102000020233 phosphotransferase Human genes 0.000 description 5
• 238000003753 real-time PCR Methods 0.000 description 5
• 108091092562 ribozyme Proteins 0.000 description 5
• 230000019491 signal transduction Effects 0.000 description 5
• 239000007787 solid Substances 0.000 description 5
• 239000006228 supernatant Substances 0.000 description 5
• 238000003786 synthesis reaction Methods 0.000 description 5
• 102000000905 Cadherin Human genes 0.000 description 4
• 108050007957 Cadherin Proteins 0.000 description 4
• 108091033380 Coding strand Proteins 0.000 description 4
• 241000252212 Danio rerio Species 0.000 description 4
• AOJJSUZBOXZQNB-TZSSRYMLSA-N Doxorubicin Chemical compound O([C@H]1C[C@@](O)(CC=2C(O)=C3C(=O)C=4C=CC=C(C=4C(=O)C3=C(O)C=21)OC)C(=O)CO)[C@H]1C[C@H](N)[C@H](O)[C@H](C)O1 AOJJSUZBOXZQNB-TZSSRYMLSA-N 0.000 description 4
• 102000053187 Glucuronidase Human genes 0.000 description 4
• 108010060309 Glucuronidase Proteins 0.000 description 4
• OUYCCCASQSFEME-QMMMGPOBSA-N L-tyrosine Chemical compound OC(=O)[C@@H](N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-QMMMGPOBSA-N 0.000 description 4
• 241000282553 Macaca Species 0.000 description 4
• 238000005481 NMR spectroscopy Methods 0.000 description 4
• 102100027063 Overexpressed in colon carcinoma 1 protein Human genes 0.000 description 4
• 101710120324 Overexpressed in colon carcinoma 1 protein homolog Proteins 0.000 description 4
• 102000038030 PI3Ks Human genes 0.000 description 4
• 108091007960 PI3Ks Proteins 0.000 description 4
• 241000282577 Pan troglodytes Species 0.000 description 4
• 102000035195 Peptidases Human genes 0.000 description 4
• 108091005804 Peptidases Proteins 0.000 description 4
• 102000004160 Phosphoric Monoester Hydrolases Human genes 0.000 description 4
• 108090000608 Phosphoric Monoester Hydrolases Proteins 0.000 description 4
• 239000004365 Protease Substances 0.000 description 4
• 108090000708 Proteasome Endopeptidase Complex Proteins 0.000 description 4
• 102000004245 Proteasome Endopeptidase Complex Human genes 0.000 description 4
• AUVVAXYIELKVAI-UHFFFAOYSA-N SJ000285215 Natural products N1CCC2=CC(OC)=C(OC)C=C2C1CC1CC2C3=CC(OC)=C(OC)C=C3CCN2CC1CC AUVVAXYIELKVAI-UHFFFAOYSA-N 0.000 description 4
• 108091027967 Small hairpin RNA Proteins 0.000 description 4
• 238000002105 Southern blotting Methods 0.000 description 4
• 108010065917 TOR Serine-Threonine Kinases Proteins 0.000 description 4
• 102000013530 TOR Serine-Threonine Kinases Human genes 0.000 description 4
• 108091023045 Untranslated Region Proteins 0.000 description 4
• 208000027418 Wounds and injury Diseases 0.000 description 4
• HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 description 4
• JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 description 4
• RJURFGZVJUQBHK-UHFFFAOYSA-N actinomycin D Natural products CC1OC(=O)C(C(C)C)N(C)C(=O)CN(C)C(=O)C2CCCN2C(=O)C(C(C)C)NC(=O)C1NC(=O)C1=C(N)C(=O)C(C)=C2OC(C(C)=CC=C3C(=O)NC4C(=O)NC(C(N5CCCC5C(=O)N(C)CC(=O)N(C)C(C(C)C)C(=O)OC4C)=O)C(C)C)=C3N=C21 RJURFGZVJUQBHK-UHFFFAOYSA-N 0.000 description 4
• 238000007792 addition Methods 0.000 description 4
• 208000022531 anorexia Diseases 0.000 description 4
• 230000000890 antigenic effect Effects 0.000 description 4
• 230000033228 biological regulation Effects 0.000 description 4
• 239000000090 biomarker Substances 0.000 description 4
• 210000004369 blood Anatomy 0.000 description 4
• 239000008280 blood Substances 0.000 description 4
• 230000037396 body weight Effects 0.000 description 4
• 125000002091 cationic group Chemical group 0.000 description 4
• 230000005754 cellular signaling Effects 0.000 description 4
• 235000013330 chicken meat Nutrition 0.000 description 4
• DQLATGHUWYMOKM-UHFFFAOYSA-L cisplatin Chemical compound N[Pt](N)(Cl)Cl DQLATGHUWYMOKM-UHFFFAOYSA-L 0.000 description 4
• 239000002299 complementary DNA Substances 0.000 description 4
• 206010061428 decreased appetite Diseases 0.000 description 4
• 230000002638 denervation Effects 0.000 description 4
• 238000013461 design Methods 0.000 description 4
• 229960003957 dexamethasone Drugs 0.000 description 4
• UREBDLICKHMUKA-CXSFZGCWSA-N dexamethasone Chemical compound C1CC2=CC(=O)C=C[C@]2(C)[C@]2(F)[C@@H]1[C@@H]1C[C@@H](C)[C@@](C(=O)CO)(O)[C@@]1(C)C[C@@H]2O UREBDLICKHMUKA-CXSFZGCWSA-N 0.000 description 4
• 238000010494 dissociation reaction Methods 0.000 description 4
• 230000005593 dissociations Effects 0.000 description 4
• AUVVAXYIELKVAI-CKBKHPSWSA-N emetine Chemical compound N1CCC2=CC(OC)=C(OC)C=C2[C@H]1C[C@H]1C[C@H]2C3=CC(OC)=C(OC)C=C3CCN2C[C@@H]1CC AUVVAXYIELKVAI-CKBKHPSWSA-N 0.000 description 4
• 229960002694 emetine Drugs 0.000 description 4
• 239000000835 fiber Substances 0.000 description 4
• 230000004927 fusion Effects 0.000 description 4
• 239000003862 glucocorticoid Substances 0.000 description 4
• 230000012010 growth Effects 0.000 description 4
• 238000009396 hybridization Methods 0.000 description 4
• 230000001969 hypertrophic effect Effects 0.000 description 4
• 238000003018 immunoassay Methods 0.000 description 4
• 238000003119 immunoblot Methods 0.000 description 4
• 230000005847 immunogenicity Effects 0.000 description 4
• 208000014674 injury Diseases 0.000 description 4
• 210000003734 kidney Anatomy 0.000 description 4
• 239000012139 lysis buffer Substances 0.000 description 4
• 230000001404 mediated effect Effects 0.000 description 4
• 230000020763 muscle atrophy Effects 0.000 description 4
• 210000003098 myoblast Anatomy 0.000 description 4
• 229910052757 nitrogen Inorganic materials 0.000 description 4
• 239000002777 nucleoside Substances 0.000 description 4
• 238000002823 phage display Methods 0.000 description 4
• 239000008194 pharmaceutical composition Substances 0.000 description 4
• 230000008569 process Effects 0.000 description 4
• AQHHHDLHHXJYJD-UHFFFAOYSA-N propranolol Chemical compound C1=CC=C2C(OCC(O)CNC(C)C)=CC=CC2=C1 AQHHHDLHHXJYJD-UHFFFAOYSA-N 0.000 description 4
• 230000011664 signaling Effects 0.000 description 4
• 239000004055 small Interfering RNA Substances 0.000 description 4
• 239000000243 solution Substances 0.000 description 4
• 235000000346 sugar Nutrition 0.000 description 4
• 239000003981 vehicle Substances 0.000 description 4
• 239000011701 zinc Substances 0.000 description 4
• 229910052725 zinc Inorganic materials 0.000 description 4
• YSAJFXWTVFGPAX-UHFFFAOYSA-N 2-[(2,4-dioxo-1h-pyrimidin-5-yl)oxy]acetic acid Chemical compound OC(=O)COC1=CNC(=O)NC1=O YSAJFXWTVFGPAX-UHFFFAOYSA-N 0.000 description 3
• FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 description 3
• OVONXEQGWXGFJD-UHFFFAOYSA-N 4-sulfanylidene-1h-pyrimidin-2-one Chemical compound SC=1C=CNC(=O)N=1 OVONXEQGWXGFJD-UHFFFAOYSA-N 0.000 description 3
• LQLQRFGHAALLLE-UHFFFAOYSA-N 5-bromouracil Chemical compound BrC1=CNC(=O)NC1=O LQLQRFGHAALLLE-UHFFFAOYSA-N 0.000 description 3
• KSNXJLQDQOIRIP-UHFFFAOYSA-N 5-iodouracil Chemical compound IC1=CNC(=O)NC1=O KSNXJLQDQOIRIP-UHFFFAOYSA-N 0.000 description 3
• STQGQHZAVUOBTE-UHFFFAOYSA-N 7-Cyan-hept-2t-en-4,6-diinsaeure Natural products C1=2C(O)=C3C(=O)C=4C(OC)=CC=CC=4C(=O)C3=C(O)C=2CC(O)(C(C)=O)CC1OC1CC(N)C(O)C(C)O1 STQGQHZAVUOBTE-UHFFFAOYSA-N 0.000 description 3
• WEVYAHXRMPXWCK-UHFFFAOYSA-N Acetonitrile Chemical compound CC#N WEVYAHXRMPXWCK-UHFFFAOYSA-N 0.000 description 3
• 201000006935 Becker muscular dystrophy Diseases 0.000 description 3
• 108010092160 Dactinomycin Proteins 0.000 description 3
• 241000255581 Drosophila <fruit fly, genus> Species 0.000 description 3
• 102100031480 Dual specificity mitogen-activated protein kinase kinase 1 Human genes 0.000 description 3
• 101710146526 Dual specificity mitogen-activated protein kinase kinase 1 Proteins 0.000 description 3
• 102100031780 Endonuclease Human genes 0.000 description 3
• XEKOWRVHYACXOJ-UHFFFAOYSA-N Ethyl acetate Chemical compound CCOC(C)=O XEKOWRVHYACXOJ-UHFFFAOYSA-N 0.000 description 3
• SXRSQZLOMIGNAQ-UHFFFAOYSA-N Glutaraldehyde Chemical compound O=CCCCC=O SXRSQZLOMIGNAQ-UHFFFAOYSA-N 0.000 description 3
• PEDCQBHIVMGVHV-UHFFFAOYSA-N Glycerine Chemical compound OCC(O)CO PEDCQBHIVMGVHV-UHFFFAOYSA-N 0.000 description 3
• 102100039939 Growth/differentiation factor 8 Human genes 0.000 description 3
• 101710088172 HTH-type transcriptional regulator RipA Proteins 0.000 description 3
• 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 description 3
• 102000038455 IGF Type 1 Receptor Human genes 0.000 description 3
• 108010031794 IGF Type 1 Receptor Proteins 0.000 description 3
• 229930010555 Inosine Natural products 0.000 description 3
• UGQMRVRMYYASKQ-KQYNXXCUSA-N Inosine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(O)=C2N=C1 UGQMRVRMYYASKQ-KQYNXXCUSA-N 0.000 description 3
• 102000000589 Interleukin-1 Human genes 0.000 description 3
• 108010002352 Interleukin-1 Proteins 0.000 description 3
• 208000027747 Kennedy disease Diseases 0.000 description 3
• 241000282842 Lama glama Species 0.000 description 3
• 201000002481 Myositis Diseases 0.000 description 3
• NWIBSHFKIJFRCO-WUDYKRTCSA-N Mytomycin Chemical compound C1N2C(C(C(C)=C(N)C3=O)=O)=C3[C@@H](COC(N)=O)[C@@]2(OC)[C@@H]2[C@H]1N2 NWIBSHFKIJFRCO-WUDYKRTCSA-N 0.000 description 3
• 208000034965 Nemaline Myopathies Diseases 0.000 description 3
• 101710163270 Nuclease Proteins 0.000 description 3
• 108020004711 Nucleic Acid Probes Proteins 0.000 description 3
• 208000001132 Osteoporosis Diseases 0.000 description 3
• 229920002873 Polyethylenimine Polymers 0.000 description 3
• 208000033526 Proximal spinal muscular atrophy type 3 Diseases 0.000 description 3
• 102100038803 Somatotropin Human genes 0.000 description 3
• 108010090804 Streptavidin Proteins 0.000 description 3
• 102100021947 Survival motor neuron protein Human genes 0.000 description 3
• 108010092262 T-Cell Antigen Receptors Proteins 0.000 description 3
• 102000016266 T-Cell Antigen Receptors Human genes 0.000 description 3
• RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical group OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 description 3
• 125000000539 amino acid group Chemical group 0.000 description 3
• 206010002026 amyotrophic lateral sclerosis Diseases 0.000 description 3
• 230000003042 antagnostic effect Effects 0.000 description 3
• 239000000074 antisense oligonucleotide Substances 0.000 description 3
• 238000012230 antisense oligonucleotides Methods 0.000 description 3
• 238000013459 approach Methods 0.000 description 3
• 230000001588 bifunctional effect Effects 0.000 description 3
• 230000000747 cardiac effect Effects 0.000 description 3
• 238000012875 competitive assay Methods 0.000 description 3
• 238000010276 construction Methods 0.000 description 3
• 239000013068 control sample Substances 0.000 description 3
• 229940127089 cytotoxic agent Drugs 0.000 description 3
• 239000002254 cytotoxic agent Substances 0.000 description 3
• STQGQHZAVUOBTE-VGBVRHCVSA-N daunorubicin Chemical compound O([C@H]1C[C@@](O)(CC=2C(O)=C3C(=O)C=4C=CC=C(C=4C(=O)C3=C(O)C=21)OC)C(C)=O)[C@H]1C[C@H](N)[C@H](O)[C@H](C)O1 STQGQHZAVUOBTE-VGBVRHCVSA-N 0.000 description 3
• 230000003247 decreasing effect Effects 0.000 description 3
• CFCUWKMKBJTWLW-UHFFFAOYSA-N deoliosyl-3C-alpha-L-digitoxosyl-MTM Natural products CC=1C(O)=C2C(O)=C3C(=O)C(OC4OC(C)C(O)C(OC5OC(C)C(O)C(OC6OC(C)C(O)C(C)(O)C6)C5)C4)C(C(OC)C(=O)C(O)C(C)O)CC3=CC2=CC=1OC(OC(C)C1O)CC1OC1CC(O)C(O)C(C)O1 CFCUWKMKBJTWLW-UHFFFAOYSA-N 0.000 description 3
• 239000005547 deoxyribonucleotide Substances 0.000 description 3
• 125000002637 deoxyribonucleotide group Chemical group 0.000 description 3
• 238000011161 development Methods 0.000 description 3
• 230000018109 developmental process Effects 0.000 description 3
• 239000000539 dimer Substances 0.000 description 3
• 201000009338 distal myopathy Diseases 0.000 description 3
• 238000012377 drug delivery Methods 0.000 description 3
• 239000012636 effector Substances 0.000 description 3
• 238000011156 evaluation Methods 0.000 description 3
• 239000012737 fresh medium Substances 0.000 description 3
• 238000012226 gene silencing method Methods 0.000 description 3
• 230000036541 health Effects 0.000 description 3
• 239000007943 implant Substances 0.000 description 3
• 238000011534 incubation Methods 0.000 description 3
• 208000015181 infectious disease Diseases 0.000 description 3
• 229960003786 inosine Drugs 0.000 description 3
• 230000002452 interceptive effect Effects 0.000 description 3
• 150000002500 ions Chemical class 0.000 description 3
• 108010076401 isopeptidase Proteins 0.000 description 3
• 210000004185 liver Anatomy 0.000 description 3
• 229940126619 mouse monoclonal antibody Drugs 0.000 description 3
• 239000002105 nanoparticle Substances 0.000 description 3
• 230000007935 neutral effect Effects 0.000 description 3
• 231100000252 nontoxic Toxicity 0.000 description 3
• 230000003000 nontoxic effect Effects 0.000 description 3
• 239000002853 nucleic acid probe Substances 0.000 description 3
• 125000003835 nucleoside group Chemical group 0.000 description 3
• 235000016709 nutrition Nutrition 0.000 description 3
• 230000036961 partial effect Effects 0.000 description 3
• 239000002245 particle Substances 0.000 description 3
• 230000006320 pegylation Effects 0.000 description 3
• 239000013612 plasmid Substances 0.000 description 3
• 239000000047 product Substances 0.000 description 3
• 210000002307 prostate Anatomy 0.000 description 3
• 238000010814 radioimmunoprecipitation assay Methods 0.000 description 3
• 238000012552 review Methods 0.000 description 3
• 239000011780 sodium chloride Substances 0.000 description 3
• 210000000952 spleen Anatomy 0.000 description 3
• 238000007619 statistical method Methods 0.000 description 3
• 208000024891 symptom Diseases 0.000 description 3
• 239000003826 tablet Substances 0.000 description 3
• RWRDLPDLKQPQOW-UHFFFAOYSA-N tetrahydropyrrole Natural products C1CCNC1 RWRDLPDLKQPQOW-UHFFFAOYSA-N 0.000 description 3
• 230000009261 transgenic effect Effects 0.000 description 3
• OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Natural products OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 description 3
• XRASPMIURGNCCH-UHFFFAOYSA-N zoledronic acid Chemical compound OP(=O)(O)C(P(O)(O)=O)(O)CN1C=CN=C1 XRASPMIURGNCCH-UHFFFAOYSA-N 0.000 description 3
• YBJHBAHKTGYVGT-ZKWXMUAHSA-N (+)-Biotin Chemical compound N1C(=O)N[C@@H]2[C@H](CCCCC(=O)O)SC[C@@H]21 YBJHBAHKTGYVGT-ZKWXMUAHSA-N 0.000 description 2
• ZEZGJKSEBRELAS-PEDHHIEDSA-N (2s)-1-[(2s,3s)-3-methyl-2-[[(2s,3s)-3-(propylcarbamoyl)oxirane-2-carbonyl]amino]pentanoyl]pyrrolidine-2-carboxylic acid Chemical compound CCCNC(=O)[C@H]1O[C@@H]1C(=O)N[C@@H]([C@@H](C)CC)C(=O)N1[C@H](C(O)=O)CCC1 ZEZGJKSEBRELAS-PEDHHIEDSA-N 0.000 description 2
• IAKHMKGGTNLKSZ-INIZCTEOSA-N (S)-colchicine Chemical compound C1([C@@H](NC(C)=O)CC2)=CC(=O)C(OC)=CC=C1C1=C2C=C(OC)C(OC)=C1OC IAKHMKGGTNLKSZ-INIZCTEOSA-N 0.000 description 2
• RFLVMTUMFYRZCB-UHFFFAOYSA-N 1-methylguanine Chemical compound O=C1N(C)C(N)=NC2=C1N=CN2 RFLVMTUMFYRZCB-UHFFFAOYSA-N 0.000 description 2
• FZWGECJQACGGTI-UHFFFAOYSA-N 2-amino-7-methyl-1,7-dihydro-6H-purin-6-one Chemical compound NC1=NC(O)=C2N(C)C=NC2=N1 FZWGECJQACGGTI-UHFFFAOYSA-N 0.000 description 2
• OIVLITBTBDPEFK-UHFFFAOYSA-N 5,6-dihydrouracil Chemical compound O=C1CCNC(=O)N1 OIVLITBTBDPEFK-UHFFFAOYSA-N 0.000 description 2
• LRFVTYWOQMYALW-UHFFFAOYSA-N 9H-xanthine Chemical compound O=C1NC(=O)NC2=C1NC=N2 LRFVTYWOQMYALW-UHFFFAOYSA-N 0.000 description 2
• 102000006267 AMP Deaminase Human genes 0.000 description 2
• 108700016228 AMP deaminases Proteins 0.000 description 2
• 206010069754 Acquired gene mutation Diseases 0.000 description 2
• 108091029845 Aminoallyl nucleotide Proteins 0.000 description 2
• 108010032595 Antibody Binding Sites Proteins 0.000 description 2
• 102100026189 Beta-galactosidase Human genes 0.000 description 2
• 241000283690 Bos taurus Species 0.000 description 2
• 208000029402 Bulbospinal muscular atrophy Diseases 0.000 description 2
• COVZYZSDYWQREU-UHFFFAOYSA-N Busulfan Chemical compound CS(=O)(=O)OCCCCOS(C)(=O)=O COVZYZSDYWQREU-UHFFFAOYSA-N 0.000 description 2
• 101100447653 Caenorhabditis elegans phg-1 gene Proteins 0.000 description 2
• 241000282836 Camelus dromedarius Species 0.000 description 2
• 206010058892 Carnitine deficiency Diseases 0.000 description 2
• 208000010693 Charcot-Marie-Tooth Disease Diseases 0.000 description 2
• 208000006545 Chronic Obstructive Pulmonary Disease Diseases 0.000 description 2
• 206010010356 Congenital anomaly Diseases 0.000 description 2
• 108010002947 Connectin Proteins 0.000 description 2
• 102100034741 Cyclin-dependent kinase 20 Human genes 0.000 description 2
• 102000004127 Cytokines Human genes 0.000 description 2
• 108090000695 Cytokines Proteins 0.000 description 2
• 101710112752 Cytotoxin Proteins 0.000 description 2
• 239000003155 DNA primer Substances 0.000 description 2
• 108700022150 Designed Ankyrin Repeat Proteins Proteins 0.000 description 2
• RTZKZFJDLAIYFH-UHFFFAOYSA-N Diethyl ether Chemical compound CCOCC RTZKZFJDLAIYFH-UHFFFAOYSA-N 0.000 description 2
• 206010014418 Electrolyte imbalance Diseases 0.000 description 2
• 102100034239 Emerin Human genes 0.000 description 2
• 201000009344 Emery-Dreifuss muscular dystrophy Diseases 0.000 description 2
• 108010075944 Erythropoietin Receptors Proteins 0.000 description 2
• 102100036509 Erythropoietin receptor Human genes 0.000 description 2
• 108700024394 Exon Proteins 0.000 description 2
• 208000037149 Facioscapulohumeral dystrophy Diseases 0.000 description 2
• 241000282326 Felis catus Species 0.000 description 2
• 102000016359 Fibronectins Human genes 0.000 description 2
• GHASVSINZRGABV-UHFFFAOYSA-N Fluorouracil Chemical compound FC1=CNC(=O)NC1=O GHASVSINZRGABV-UHFFFAOYSA-N 0.000 description 2
• 208000024412 Friedreich ataxia Diseases 0.000 description 2
• 108091093094 Glycol nucleic acid Proteins 0.000 description 2
• 229940122069 Glycosidase inhibitor Drugs 0.000 description 2
• 102100039619 Granulocyte colony-stimulating factor Human genes 0.000 description 2
• 108010043121 Green Fluorescent Proteins Proteins 0.000 description 2
• 102000004144 Green Fluorescent Proteins Human genes 0.000 description 2
• 102100020948 Growth hormone receptor Human genes 0.000 description 2
• NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-UUOKFMHZSA-N 0.000 description 2
• 101000746367 Homo sapiens Granulocyte colony-stimulating factor Proteins 0.000 description 2
• 206010020880 Hypertrophy Diseases 0.000 description 2
• 108010054477 Immunoglobulin Fab Fragments Proteins 0.000 description 2
• 102000001706 Immunoglobulin Fab Fragments Human genes 0.000 description 2
• 108700005091 Immunoglobulin Genes Proteins 0.000 description 2
• 108010067060 Immunoglobulin Variable Region Proteins 0.000 description 2
• 102000017727 Immunoglobulin Variable Region Human genes 0.000 description 2
• 108090001005 Interleukin-6 Proteins 0.000 description 2
• 102000004889 Interleukin-6 Human genes 0.000 description 2
• KFZMGEQAYNKOFK-UHFFFAOYSA-N Isopropanol Chemical compound CC(C)O KFZMGEQAYNKOFK-UHFFFAOYSA-N 0.000 description 2
• DAQAKHDKYAWHCG-UHFFFAOYSA-N Lactacystin Natural products CC(=O)NC(C(O)=O)CSC(=O)C1(C(O)C(C)C)NC(=O)C(C)C1O DAQAKHDKYAWHCG-UHFFFAOYSA-N 0.000 description 2
• 102100022745 Laminin subunit alpha-2 Human genes 0.000 description 2
• 108091026898 Leader sequence (mRNA) Proteins 0.000 description 2
• 102100032352 Leukemia inhibitory factor Human genes 0.000 description 2
• 108090000581 Leukemia inhibitory factor Proteins 0.000 description 2
• 201000009342 Limb-girdle muscular dystrophy Diseases 0.000 description 2
• GQYIWUVLTXOXAJ-UHFFFAOYSA-N Lomustine Chemical compound ClCCN(N=O)C(=O)NC1CCCCC1 GQYIWUVLTXOXAJ-UHFFFAOYSA-N 0.000 description 2
• 102000043129 MHC class I family Human genes 0.000 description 2
• 108091054437 MHC class I family Proteins 0.000 description 2
• 241000124008 Mammalia Species 0.000 description 2
• 201000002169 Mitochondrial myopathy Diseases 0.000 description 2
• 101150097381 Mtor gene Proteins 0.000 description 2
• 241000699660 Mus musculus Species 0.000 description 2
• 102000006386 Myelin Proteins Human genes 0.000 description 2
• 108010083674 Myelin Proteins Proteins 0.000 description 2
• 102000003505 Myosin Human genes 0.000 description 2
• 108060008487 Myosin Proteins 0.000 description 2
• 102100030626 Myosin-binding protein H Human genes 0.000 description 2
• 101710139548 Myosin-binding protein H Proteins 0.000 description 2
• 206010068871 Myotonic dystrophy Diseases 0.000 description 2
• HYVABZIGRDEKCD-UHFFFAOYSA-N N(6)-dimethylallyladenine Chemical compound CC(C)=CCNC1=NC=NC2=C1N=CN2 HYVABZIGRDEKCD-UHFFFAOYSA-N 0.000 description 2
• 108050000637 N-cadherin Proteins 0.000 description 2
• 241000244206 Nematoda Species 0.000 description 2
• 108010069196 Neural Cell Adhesion Molecules Proteins 0.000 description 2
• 102100027347 Neural cell adhesion molecule 1 Human genes 0.000 description 2
• 101710089162 Neuroglian Proteins 0.000 description 2
• 108091028043 Nucleic acid sequence Proteins 0.000 description 2
• 201000009110 Oculopharyngeal muscular dystrophy Diseases 0.000 description 2
• 239000002033 PVDF binder Substances 0.000 description 2
• 108091093037 Peptide nucleic acid Proteins 0.000 description 2
• ISWSIDIOOBJBQZ-UHFFFAOYSA-N Phenol Chemical compound OC1=CC=CC=C1 ISWSIDIOOBJBQZ-UHFFFAOYSA-N 0.000 description 2
• 102000003993 Phosphatidylinositol 3-kinases Human genes 0.000 description 2
• 108090000430 Phosphatidylinositol 3-kinases Proteins 0.000 description 2
• 108700010203 Phosphoglycerate Kinase 1 Deficiency Proteins 0.000 description 2
• 108010002519 Prolactin Receptors Proteins 0.000 description 2
• 102100029000 Prolactin receptor Human genes 0.000 description 2
• 229940124158 Protease/peptidase inhibitor Drugs 0.000 description 2
• 102000001253 Protein Kinase Human genes 0.000 description 2
• 208000032225 Proximal spinal muscular atrophy type 1 Diseases 0.000 description 2
• 208000033522 Proximal spinal muscular atrophy type 2 Diseases 0.000 description 2
• 108091008109 Pseudogenes Proteins 0.000 description 2
• 102000057361 Pseudogenes Human genes 0.000 description 2
• 239000013614 RNA sample Substances 0.000 description 2
• 102000000574 RNA-Induced Silencing Complex Human genes 0.000 description 2
• 108010016790 RNA-Induced Silencing Complex Proteins 0.000 description 2
• 108010092799 RNA-directed DNA polymerase Proteins 0.000 description 2
• 241000700157 Rattus norvegicus Species 0.000 description 2
• 108091028664 Ribonucleotide Proteins 0.000 description 2
• 108010039491 Ricin Proteins 0.000 description 2
• 102000000369 SOCS box domains Human genes 0.000 description 2
• 108050008939 SOCS box domains Proteins 0.000 description 2
• 240000004808 Saccharomyces cerevisiae Species 0.000 description 2
• 101710184528 Scaffolding protein Proteins 0.000 description 2
• 108091060271 Small temporal RNA Proteins 0.000 description 2
• 108010068542 Somatotropin Receptors Proteins 0.000 description 2
• 241000282887 Suidae Species 0.000 description 2
• 102100025237 T-cell surface antigen CD2 Human genes 0.000 description 2
• 102100024219 T-cell surface glycoprotein CD1a Human genes 0.000 description 2
• 102100036011 T-cell surface glycoprotein CD4 Human genes 0.000 description 2
• 102100034922 T-cell surface glycoprotein CD8 alpha chain Human genes 0.000 description 2
• 210000001744 T-lymphocyte Anatomy 0.000 description 2
• 239000006180 TBST buffer Substances 0.000 description 2
• 108091036066 Three prime untranslated region Proteins 0.000 description 2
• 108091046915 Threose nucleic acid Proteins 0.000 description 2
• IQFYYKKMVGJFEH-XLPZGREQSA-N Thymidine Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-XLPZGREQSA-N 0.000 description 2
• 102100026260 Titin Human genes 0.000 description 2
• 108091023040 Transcription factor Proteins 0.000 description 2
• 102000040945 Transcription factor Human genes 0.000 description 2
• 108060008539 Transglutaminase Proteins 0.000 description 2
• VGQOVCHZGQWAOI-UHFFFAOYSA-N UNPD55612 Natural products N1C(O)C2CC(C=CC(N)=O)=CN2C(=O)C2=CC=C(C)C(O)=C12 VGQOVCHZGQWAOI-UHFFFAOYSA-N 0.000 description 2
• 108010005705 Ubiquitinated Proteins Proteins 0.000 description 2
• ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 description 2
• DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 description 2
• 108010000134 Vascular Cell Adhesion Molecule-1 Proteins 0.000 description 2
• JXLYSJRDGCGARV-WWYNWVTFSA-N Vinblastine Natural products O=C(O[C@H]1[C@](O)(C(=O)OC)[C@@H]2N(C)c3c(cc(c(OC)c3)[C@]3(C(=O)OC)c4[nH]c5c(c4CCN4C[C@](O)(CC)C[C@H](C3)C4)cccc5)[C@@]32[C@H]2[C@@]1(CC)C=CCN2CC3)C JXLYSJRDGCGARV-WWYNWVTFSA-N 0.000 description 2
• 208000010399 Wasting Syndrome Diseases 0.000 description 2
• RJURFGZVJUQBHK-IIXSONLDSA-N actinomycin D Chemical compound C[C@H]1OC(=O)[C@H](C(C)C)N(C)C(=O)CN(C)C(=O)[C@@H]2CCCN2C(=O)[C@@H](C(C)C)NC(=O)[C@H]1NC(=O)C1=C(N)C(=O)C(C)=C2OC(C(C)=CC=C3C(=O)N[C@@H]4C(=O)N[C@@H](C(N5CCC[C@H]5C(=O)N(C)CC(=O)N(C)[C@@H](C(C)C)C(=O)O[C@@H]4C)=O)C(C)C)=C3N=C21 RJURFGZVJUQBHK-IIXSONLDSA-N 0.000 description 2
• 230000009471 action Effects 0.000 description 2
• 210000000577 adipose tissue Anatomy 0.000 description 2
• 230000009824 affinity maturation Effects 0.000 description 2
• 239000000556 agonist Substances 0.000 description 2
• 150000001412 amines Chemical class 0.000 description 2
• 208000007502 anemia Diseases 0.000 description 2
• 230000008485 antagonism Effects 0.000 description 2
• 210000001264 anterior cruciate ligament Anatomy 0.000 description 2
• VGQOVCHZGQWAOI-HYUHUPJXSA-N anthramycin Chemical compound N1[C@@H](O)[C@@H]2CC(\C=C\C(N)=O)=CN2C(=O)C2=CC=C(C)C(O)=C12 VGQOVCHZGQWAOI-HYUHUPJXSA-N 0.000 description 2
• 239000003242 anti bacterial agent Substances 0.000 description 2
• 230000010056 antibody-dependent cellular cytotoxicity Effects 0.000 description 2
• 239000002246 antineoplastic agent Substances 0.000 description 2
• 229940041181 antineoplastic drug Drugs 0.000 description 2
• 230000004596 appetite loss Effects 0.000 description 2
• 206010003549 asthenia Diseases 0.000 description 2
• 230000008901 benefit Effects 0.000 description 2
• 108010005774 beta-Galactosidase Proteins 0.000 description 2
• 230000003115 biocidal effect Effects 0.000 description 2
• 239000003124 biologic agent Substances 0.000 description 2
• 239000012472 biological sample Substances 0.000 description 2
• OWMVSZAMULFTJU-UHFFFAOYSA-N bis-tris Chemical compound OCCN(CCO)C(CO)(CO)CO OWMVSZAMULFTJU-UHFFFAOYSA-N 0.000 description 2
• 210000001124 body fluid Anatomy 0.000 description 2
• 210000004556 brain Anatomy 0.000 description 2
• 150000001720 carbohydrates Chemical group 0.000 description 2
• 210000000170 cell membrane Anatomy 0.000 description 2
• 210000003679 cervix uteri Anatomy 0.000 description 2
• OSASVXMJTNOKOY-UHFFFAOYSA-N chlorobutanol Chemical compound CC(C)(O)C(Cl)(Cl)Cl OSASVXMJTNOKOY-UHFFFAOYSA-N 0.000 description 2
• 238000000749 co-immunoprecipitation Methods 0.000 description 2
• 238000000576 coating method Methods 0.000 description 2
• 210000001072 colon Anatomy 0.000 description 2
• 230000004540 complement-dependent cytotoxicity Effects 0.000 description 2
• 201000006815 congenital muscular dystrophy Diseases 0.000 description 2
• 230000008878 coupling Effects 0.000 description 2
• 238000010168 coupling process Methods 0.000 description 2
• 238000005859 coupling reaction Methods 0.000 description 2
• YPHMISFOHDHNIV-FSZOTQKASA-N cycloheximide Chemical compound C1[C@@H](C)C[C@H](C)C(=O)[C@@H]1[C@H](O)CC1CC(=O)NC(=O)C1 YPHMISFOHDHNIV-FSZOTQKASA-N 0.000 description 2
• 125000000151 cysteine group Chemical class N[C@@H](CS)C(=O)* 0.000 description 2
• 108010057085 cytokine receptors Proteins 0.000 description 2
• 102000003675 cytokine receptors Human genes 0.000 description 2
• 239000002619 cytotoxin Substances 0.000 description 2
• 229960000640 dactinomycin Drugs 0.000 description 2
• 229960000975 daunorubicin Drugs 0.000 description 2
• 231100000673 dose–response relationship Toxicity 0.000 description 2
• 229960004679 doxorubicin Drugs 0.000 description 2
• 230000002255 enzymatic effect Effects 0.000 description 2
• 210000003238 esophagus Anatomy 0.000 description 2
• 239000013604 expression vector Substances 0.000 description 2
• 208000008570 facioscapulohumeral muscular dystrophy Diseases 0.000 description 2
• 235000019197 fats Nutrition 0.000 description 2
• 230000003176 fibrotic effect Effects 0.000 description 2
• 229960002949 fluorouracil Drugs 0.000 description 2
• 238000009472 formulation Methods 0.000 description 2
• 239000003316 glycosidase inhibitor Substances 0.000 description 2
• 239000008187 granular material Substances 0.000 description 2
• 239000005090 green fluorescent protein Substances 0.000 description 2
• 230000002209 hydrophobic effect Effects 0.000 description 2
• FDGQSTZJBFJUBT-UHFFFAOYSA-N hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 description 2
• 238000003384 imaging method Methods 0.000 description 2
• 238000010166 immunofluorescence Methods 0.000 description 2
• 230000001976 improved effect Effects 0.000 description 2
• 230000006872 improvement Effects 0.000 description 2
• 208000023692 inborn mitochondrial myopathy Diseases 0.000 description 2
• 210000003000 inclusion body Anatomy 0.000 description 2
• 238000010348 incorporation Methods 0.000 description 2
• 102000006495 integrins Human genes 0.000 description 2
• 108010044426 integrins Proteins 0.000 description 2
• 108010085650 interferon gamma receptor Proteins 0.000 description 2
• 230000003834 intracellular effect Effects 0.000 description 2
• 201000004815 juvenile spinal muscular atrophy Diseases 0.000 description 2
• DAQAKHDKYAWHCG-RWTHQLGUSA-N lactacystin Chemical compound CC(=O)N[C@H](C(O)=O)CSC(=O)[C@]1([C@@H](O)C(C)C)NC(=O)[C@H](C)[C@@H]1O DAQAKHDKYAWHCG-RWTHQLGUSA-N 0.000 description 2
• 235000005772 leucine Nutrition 0.000 description 2
• 150000002632 lipids Chemical class 0.000 description 2
• 235000021266 loss of appetite Nutrition 0.000 description 2
• 208000019017 loss of appetite Diseases 0.000 description 2
• 210000004072 lung Anatomy 0.000 description 2
• HQKMJHAJHXVSDF-UHFFFAOYSA-L magnesium stearate Chemical compound [Mg+2].CCCCCCCCCCCCCCCCCC([O-])=O.CCCCCCCCCCCCCCCCCC([O-])=O HQKMJHAJHXVSDF-UHFFFAOYSA-L 0.000 description 2
• 210000004962 mammalian cell Anatomy 0.000 description 2
• GLVAUDGFNGKCSF-UHFFFAOYSA-N mercaptopurine Chemical compound S=C1NC=NC2=C1NC=N2 GLVAUDGFNGKCSF-UHFFFAOYSA-N 0.000 description 2
• 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 description 2
• 125000000325 methylidene group Chemical group [H]C([H])=* 0.000 description 2
• 239000002679 microRNA Substances 0.000 description 2
• 238000002493 microarray Methods 0.000 description 2
• 150000007522 mineralic acids Chemical class 0.000 description 2
• CFCUWKMKBJTWLW-BKHRDMLASA-N mithramycin Chemical compound O([C@@H]1C[C@@H](O[C@H](C)[C@H]1O)OC=1C=C2C=C3C[C@H]([C@@H](C(=O)C3=C(O)C2=C(O)C=1C)O[C@@H]1O[C@H](C)[C@@H](O)[C@H](O[C@@H]2O[C@H](C)[C@H](O)[C@H](O[C@@H]3O[C@H](C)[C@@H](O)[C@@](C)(O)C3)C2)C1)[C@H](OC)C(=O)[C@@H](O)[C@@H](C)O)[C@H]1C[C@@H](O)[C@H](O)[C@@H](C)O1 CFCUWKMKBJTWLW-BKHRDMLASA-N 0.000 description 2
• 239000002829 mitogen activated protein kinase inhibitor Substances 0.000 description 2
• 229960004857 mitomycin Drugs 0.000 description 2
• 230000003387 muscular Effects 0.000 description 2
• 201000006938 muscular dystrophy Diseases 0.000 description 2
• 238000002703 mutagenesis Methods 0.000 description 2
• 231100000350 mutagenesis Toxicity 0.000 description 2
• 230000035772 mutation Effects 0.000 description 2
• 206010028417 myasthenia gravis Diseases 0.000 description 2
• 210000005012 myelin Anatomy 0.000 description 2
• 239000013642 negative control Substances 0.000 description 2
• 238000007899 nucleic acid hybridization Methods 0.000 description 2
• 230000035764 nutrition Effects 0.000 description 2
• 210000000056 organ Anatomy 0.000 description 2
• 150000007524 organic acids Chemical class 0.000 description 2
• 235000005985 organic acids Nutrition 0.000 description 2
• 239000003960 organic solvent Substances 0.000 description 2
• 230000003204 osmotic effect Effects 0.000 description 2
• 210000001672 ovary Anatomy 0.000 description 2
• 208000029308 periodic paralysis Diseases 0.000 description 2
• 150000003916 phosphatidylinositol 3,4,5-trisphosphates Chemical class 0.000 description 2
• 239000000902 placebo Substances 0.000 description 2
• 229940068196 placebo Drugs 0.000 description 2
• 210000002826 placenta Anatomy 0.000 description 2
• 229960003171 plicamycin Drugs 0.000 description 2
• 229920000642 polymer Polymers 0.000 description 2
• 102000040430 polynucleotide Human genes 0.000 description 2
• 108091033319 polynucleotide Proteins 0.000 description 2
• 239000002157 polynucleotide Substances 0.000 description 2
• 229920002981 polyvinylidene fluoride Polymers 0.000 description 2
• 230000004481 post-translational protein modification Effects 0.000 description 2
• 230000002265 prevention Effects 0.000 description 2
• 230000003449 preventive effect Effects 0.000 description 2
• 238000012545 processing Methods 0.000 description 2
• 229960003712 propranolol Drugs 0.000 description 2
• 230000004952 protein activity Effects 0.000 description 2
• 238000002331 protein detection Methods 0.000 description 2
• 230000004853 protein function Effects 0.000 description 2
• 108060006633 protein kinase Proteins 0.000 description 2
• 238000001742 protein purification Methods 0.000 description 2
• RXWNCPJZOCPEPQ-NVWDDTSBSA-N puromycin Chemical compound C1=CC(OC)=CC=C1C[C@H](N)C(=O)N[C@H]1[C@@H](O)[C@H](N2C3=NC=NC(=C3N=C2)N(C)C)O[C@@H]1CO RXWNCPJZOCPEPQ-NVWDDTSBSA-N 0.000 description 2
• 238000011002 quantification Methods 0.000 description 2
• 230000002285 radioactive effect Effects 0.000 description 2
• ZAHRKKWIAAJSAO-UHFFFAOYSA-N rapamycin Natural products COCC(O)C(=C/C(C)C(=O)CC(OC(=O)C1CCCCN1C(=O)C(=O)C2(O)OC(CC(OC)C(=CC=CC=CC(C)CC(C)C(=O)C)C)CCC2C)C(C)CC3CCC(O)C(C3)OC)C ZAHRKKWIAAJSAO-UHFFFAOYSA-N 0.000 description 2
• 239000011541 reaction mixture Substances 0.000 description 2
• 230000009257 reactivity Effects 0.000 description 2
• 238000003259 recombinant expression Methods 0.000 description 2
• 238000011160 research Methods 0.000 description 2
• 238000010839 reverse transcription Methods 0.000 description 2
• 239000002336 ribonucleotide Substances 0.000 description 2
• 239000012723 sample buffer Substances 0.000 description 2
• 238000000926 separation method Methods 0.000 description 2
• 229960002930 sirolimus Drugs 0.000 description 2
• QFJCIRLUMZQUOT-HPLJOQBZSA-N sirolimus Chemical compound C1C[C@@H](O)[C@H](OC)C[C@@H]1C[C@@H](C)[C@H]1OC(=O)[C@@H]2CCCCN2C(=O)C(=O)[C@](O)(O2)[C@H](C)CC[C@H]2C[C@H](OC)/C(C)=C/C=C/C=C/[C@@H](C)C[C@@H](C)C(=O)[C@H](OC)[C@H](O)/C(C)=C/[C@@H](C)C(=O)C1 QFJCIRLUMZQUOT-HPLJOQBZSA-N 0.000 description 2
• 210000000813 small intestine Anatomy 0.000 description 2
• 239000002904 solvent Substances 0.000 description 2
• 230000037439 somatic mutation Effects 0.000 description 2
• 230000009870 specific binding Effects 0.000 description 2
• 239000000829 suppository Substances 0.000 description 2
• 208000016505 systemic primary carnitine deficiency disease Diseases 0.000 description 2
• 210000001550 testis Anatomy 0.000 description 2
• RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 description 2
• RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 description 2
• 210000001541 thymus gland Anatomy 0.000 description 2
• 210000001685 thyroid gland Anatomy 0.000 description 2
• WYWHKKSPHMUBEB-UHFFFAOYSA-N tioguanine Chemical compound N1C(N)=NC(=S)C2=C1N=CN2 WYWHKKSPHMUBEB-UHFFFAOYSA-N 0.000 description 2
• 239000003053 toxin Substances 0.000 description 2
• 231100000765 toxin Toxicity 0.000 description 2
• 108700012359 toxins Proteins 0.000 description 2
• 210000003437 trachea Anatomy 0.000 description 2
• 238000010361 transduction Methods 0.000 description 2
• 230000026683 transduction Effects 0.000 description 2
• 239000012096 transfection reagent Substances 0.000 description 2
• 238000012546 transfer Methods 0.000 description 2
• 102000003601 transglutaminase Human genes 0.000 description 2
• 238000007492 two-way ANOVA Methods 0.000 description 2
• 208000032471 type 1 spinal muscular atrophy Diseases 0.000 description 2
• 208000032521 type II spinal muscular atrophy Diseases 0.000 description 2
• 208000032527 type III spinal muscular atrophy Diseases 0.000 description 2
• 108010016317 ubiquitin-aldehyde Proteins 0.000 description 2
• 101150003485 unc-22 gene Proteins 0.000 description 2
• 210000000689 upper leg Anatomy 0.000 description 2
• 210000003932 urinary bladder Anatomy 0.000 description 2
• 229960003048 vinblastine Drugs 0.000 description 2
• JXLYSJRDGCGARV-XQKSVPLYSA-N vincaleukoblastine Chemical compound C([C@@H](C[C@]1(C(=O)OC)C=2C(=CC3=C([C@]45[C@H]([C@@]([C@H](OC(C)=O)[C@]6(CC)C=CCN([C@H]56)CC4)(O)C(=O)OC)N3C)C=2)OC)C[C@@](C2)(O)CC)N2CCC2=C1NC1=CC=CC=C21 JXLYSJRDGCGARV-XQKSVPLYSA-N 0.000 description 2
• OGWKCGZFUXNPDA-UHFFFAOYSA-N vincristine Natural products C1C(CC)(O)CC(CC2(C(=O)OC)C=3C(=CC4=C(C56C(C(C(OC(C)=O)C7(CC)C=CCN(C67)CC5)(O)C(=O)OC)N4C=O)C=3)OC)CN1CCC1=C2NC2=CC=CC=C12 OGWKCGZFUXNPDA-UHFFFAOYSA-N 0.000 description 2
• OGWKCGZFUXNPDA-XQKSVPLYSA-N vincristine Chemical compound C([N@]1C[C@@H](C[C@]2(C(=O)OC)C=3C(=CC4=C([C@]56[C@H]([C@@]([C@H](OC(C)=O)[C@]7(CC)C=CCN([C@H]67)CC5)(O)C(=O)OC)N4C=O)C=3)OC)C[C@@](C1)(O)CC)CC1=C2NC2=CC=CC=C12 OGWKCGZFUXNPDA-XQKSVPLYSA-N 0.000 description 2
• 229960004528 vincristine Drugs 0.000 description 2
• 239000001993 wax Substances 0.000 description 2
• QDLHCMPXEPAAMD-QAIWCSMKSA-N wortmannin Chemical compound C1([C@]2(C)C3=C(C4=O)OC=C3C(=O)O[C@@H]2COC)=C4[C@@H]2CCC(=O)[C@@]2(C)C[C@H]1OC(C)=O QDLHCMPXEPAAMD-QAIWCSMKSA-N 0.000 description 2
• 229960004276 zoledronic acid Drugs 0.000 description 2
• HDTRYLNUVZCQOY-UHFFFAOYSA-N &alpha;-D-glucopyranosyl-&alpha;-D-glucopyranoside Natural products OC1C(O)C(O)C(CO)OC1OC1C(O)C(O)C(O)C(CO)O1 HDTRYLNUVZCQOY-UHFFFAOYSA-N 0.000 description 1
• CEMAWMOMDPGJMB-UHFFFAOYSA-N (+-)-Oxprenolol Chemical compound CC(C)NCC(O)COC1=CC=CC=C1OCC=C CEMAWMOMDPGJMB-UHFFFAOYSA-N 0.000 description 1
• LNAZSHAWQACDHT-XIYTZBAFSA-N (2r,3r,4s,5r,6s)-4,5-dimethoxy-2-(methoxymethyl)-3-[(2s,3r,4s,5r,6r)-3,4,5-trimethoxy-6-(methoxymethyl)oxan-2-yl]oxy-6-[(2r,3r,4s,5r,6r)-4,5,6-trimethoxy-2-(methoxymethyl)oxan-3-yl]oxyoxane Chemical compound CO[C@@H]1[C@@H](OC)[C@H](OC)[C@@H](COC)O[C@H]1O[C@H]1[C@H](OC)[C@@H](OC)[C@H](O[C@H]2[C@@H]([C@@H](OC)[C@H](OC)O[C@@H]2COC)OC)O[C@@H]1COC LNAZSHAWQACDHT-XIYTZBAFSA-N 0.000 description 1
• IRZNDKKKFMEHTG-XNIJJKJLSA-N (2r,3s,4r,5r)-2-(hydroxymethyl)-5-[6-[(5-hydroxypyridin-2-yl)methylamino]purin-9-yl]oxolane-3,4-diol Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(NCC=3N=CC(O)=CC=3)=C2N=C1 IRZNDKKKFMEHTG-XNIJJKJLSA-N 0.000 description 1
• CUKWUWBLQQDQAC-VEQWQPCFSA-N (3s)-3-amino-4-[[(2s)-1-[[(2s)-1-[[(2s)-1-[[(2s,3s)-1-[[(2s)-1-[(2s)-2-[[(1s)-1-carboxyethyl]carbamoyl]pyrrolidin-1-yl]-3-(1h-imidazol-5-yl)-1-oxopropan-2-yl]amino]-3-methyl-1-oxopentan-2-yl]amino]-3-(4-hydroxyphenyl)-1-oxopropan-2-yl]amino]-3-methyl-1-ox Chemical compound C([C@@H](C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H](CC=1NC=NC=1)C(=O)N1[C@@H](CCC1)C(=O)N[C@@H](C)C(O)=O)NC(=O)[C@@H](NC(=O)[C@H](CCCN=C(N)N)NC(=O)[C@@H](N)CC(O)=O)C(C)C)C1=CC=C(O)C=C1 CUKWUWBLQQDQAC-VEQWQPCFSA-N 0.000 description 1
• FDKXTQMXEQVLRF-ZHACJKMWSA-N (E)-dacarbazine Chemical compound CN(C)\N=N\c1[nH]cnc1C(N)=O FDKXTQMXEQVLRF-ZHACJKMWSA-N 0.000 description 1
• METKIMKYRPQLGS-GFCCVEGCSA-N (R)-atenolol Chemical compound CC(C)NC[C@@H](O)COC1=CC=C(CC(N)=O)C=C1 METKIMKYRPQLGS-GFCCVEGCSA-N 0.000 description 1
• TWBNMYSKRDRHAT-RCWTXCDDSA-N (S)-timolol hemihydrate Chemical compound O.CC(C)(C)NC[C@H](O)COC1=NSN=C1N1CCOCC1.CC(C)(C)NC[C@H](O)COC1=NSN=C1N1CCOCC1 TWBNMYSKRDRHAT-RCWTXCDDSA-N 0.000 description 1
• KOHIRBRYDXPAMZ-YHBROIRLSA-N (S,R,R,R)-nebivolol Chemical compound C1CC2=CC(F)=CC=C2O[C@H]1[C@H](O)CNC[C@@H](O)[C@H]1OC2=CC=C(F)C=C2CC1 KOHIRBRYDXPAMZ-YHBROIRLSA-N 0.000 description 1
• TZJUVVIWVWFLCD-UHFFFAOYSA-N 1,1-dioxo-2-[4-[4-(2-pyrimidinyl)-1-piperazinyl]butyl]-1,2-benzothiazol-3-one Chemical compound O=S1(=O)C2=CC=CC=C2C(=O)N1CCCCN(CC1)CCN1C1=NC=CC=N1 TZJUVVIWVWFLCD-UHFFFAOYSA-N 0.000 description 1
• SNKAWJBJQDLSFF-NVKMUCNASA-N 1,2-dioleoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCC\C=C/CCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCC\C=C/CCCCCCCC SNKAWJBJQDLSFF-NVKMUCNASA-N 0.000 description 1
• UHDGCWIWMRVCDJ-UHFFFAOYSA-N 1-beta-D-Xylofuranosyl-NH-Cytosine Natural products O=C1N=C(N)C=CN1C1C(O)C(O)C(CO)O1 UHDGCWIWMRVCDJ-UHFFFAOYSA-N 0.000 description 1
• NFGXHKASABOEEW-UHFFFAOYSA-N 1-methylethyl 11-methoxy-3,7,11-trimethyl-2,4-dodecadienoate Chemical compound COC(C)(C)CCCC(C)CC=CC(C)=CC(=O)OC(C)C NFGXHKASABOEEW-UHFFFAOYSA-N 0.000 description 1
• WJNGQIYEQLPJMN-IOSLPCCCSA-N 1-methylinosine Chemical compound C1=NC=2C(=O)N(C)C=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O WJNGQIYEQLPJMN-IOSLPCCCSA-N 0.000 description 1
• IIZPXYDJLKNOIY-JXPKJXOSSA-N 1-palmitoyl-2-arachidonoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCC\C=C/C\C=C/C\C=C/C\C=C/CCCCC IIZPXYDJLKNOIY-JXPKJXOSSA-N 0.000 description 1
• OWEGMIWEEQEYGQ-UHFFFAOYSA-N 100676-05-9 Natural products OC1C(O)C(O)C(CO)OC1OCC1C(O)C(O)C(O)C(OC2C(OC(O)C(O)C2O)CO)O1 OWEGMIWEEQEYGQ-UHFFFAOYSA-N 0.000 description 1
• FMKLAIBZMCURLI-BFVRRIQPSA-N 15-Keto-13,14-dihydroprostaglandin A2 Chemical compound CCCCCC(=O)CC[C@H]1C=CC(=O)[C@@H]1C\C=C/CCCC(O)=O FMKLAIBZMCURLI-BFVRRIQPSA-N 0.000 description 1
• CKTSBUTUHBMZGZ-SHYZEUOFSA-N 2'‐deoxycytidine Chemical compound O=C1N=C(N)C=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 CKTSBUTUHBMZGZ-SHYZEUOFSA-N 0.000 description 1
• YBBNVCVOACOHIG-UHFFFAOYSA-N 2,2-diamino-1,4-bis(4-azidophenyl)-3-butylbutane-1,4-dione Chemical compound C=1C=C(N=[N+]=[N-])C=CC=1C(=O)C(N)(N)C(CCCC)C(=O)C1=CC=C(N=[N+]=[N-])C=C1 YBBNVCVOACOHIG-UHFFFAOYSA-N 0.000 description 1
• KSXTUUUQYQYKCR-LQDDAWAPSA-M 2,3-bis[[(z)-octadec-9-enoyl]oxy]propyl-trimethylazanium;chloride Chemical compound [Cl-].CCCCCCCC\C=C/CCCCCCCC(=O)OCC(C[N+](C)(C)C)OC(=O)CCCCCCC\C=C/CCCCCCCC KSXTUUUQYQYKCR-LQDDAWAPSA-M 0.000 description 1
• JTTIOYHBNXDJOD-UHFFFAOYSA-N 2,4,6-triaminopyrimidine Chemical compound NC1=CC(N)=NC(N)=N1 JTTIOYHBNXDJOD-UHFFFAOYSA-N 0.000 description 1
• HLYBTPMYFWWNJN-UHFFFAOYSA-N 2-(2,4-dioxo-1h-pyrimidin-5-yl)-2-hydroxyacetic acid Chemical compound OC(=O)C(O)C1=CNC(=O)NC1=O HLYBTPMYFWWNJN-UHFFFAOYSA-N 0.000 description 1
• FBMYKMYQHCBIGU-UHFFFAOYSA-N 2-[2-hydroxy-3-[[1-(1h-indol-3-yl)-2-methylpropan-2-yl]amino]propoxy]benzonitrile Chemical compound C=1NC2=CC=CC=C2C=1CC(C)(C)NCC(O)COC1=CC=CC=C1C#N FBMYKMYQHCBIGU-UHFFFAOYSA-N 0.000 description 1
• JKMHFZQWWAIEOD-UHFFFAOYSA-N 2-[4-(2-hydroxyethyl)piperazin-1-yl]ethanesulfonic acid Chemical compound OCC[NH+]1CCN(CCS([O-])(=O)=O)CC1 JKMHFZQWWAIEOD-UHFFFAOYSA-N 0.000 description 1
• LLWPKTDSDUQBFY-UHFFFAOYSA-N 2-[6-(aminomethyl)-2,4-dioxo-1H-pyrimidin-5-yl]acetic acid Chemical compound C(=O)(O)CC=1C(NC(NC=1CN)=O)=O LLWPKTDSDUQBFY-UHFFFAOYSA-N 0.000 description 1
• FZDFGHZZPBUTGP-UHFFFAOYSA-N 2-[[2-[bis(carboxymethyl)amino]-3-(4-isothiocyanatophenyl)propyl]-[2-[bis(carboxymethyl)amino]propyl]amino]acetic acid Chemical compound OC(=O)CN(CC(O)=O)C(C)CN(CC(O)=O)CC(N(CC(O)=O)CC(O)=O)CC1=CC=C(N=C=S)C=C1 FZDFGHZZPBUTGP-UHFFFAOYSA-N 0.000 description 1
• 125000005273 2-acetoxybenzoic acid group Chemical class 0.000 description 1
• QKNYBSVHEMOAJP-UHFFFAOYSA-N 2-amino-2-(hydroxymethyl)propane-1,3-diol;hydron;chloride Chemical compound Cl.OCC(N)(CO)CO QKNYBSVHEMOAJP-UHFFFAOYSA-N 0.000 description 1
• XMSMHKMPBNTBOD-UHFFFAOYSA-N 2-dimethylamino-6-hydroxypurine Chemical compound N1C(N(C)C)=NC(=O)C2=C1N=CN2 XMSMHKMPBNTBOD-UHFFFAOYSA-N 0.000 description 1
• SGUAFYQXFOLMHL-UHFFFAOYSA-N 2-hydroxy-5-{1-hydroxy-2-[(4-phenylbutan-2-yl)amino]ethyl}benzamide Chemical compound C=1C=C(O)C(C(N)=O)=CC=1C(O)CNC(C)CCC1=CC=CC=C1 SGUAFYQXFOLMHL-UHFFFAOYSA-N 0.000 description 1
• SMADWRYCYBUIKH-UHFFFAOYSA-N 2-methyl-7h-purin-6-amine Chemical compound CC1=NC(N)=C2NC=NC2=N1 SMADWRYCYBUIKH-UHFFFAOYSA-N 0.000 description 1
• 108020005345 3' Untranslated Regions Proteins 0.000 description 1
• KOLPWZCZXAMXKS-UHFFFAOYSA-N 3-methylcytosine Chemical compound CN1C(N)=CC=NC1=O KOLPWZCZXAMXKS-UHFFFAOYSA-N 0.000 description 1
• FJKROLUGYXJWQN-UHFFFAOYSA-N 4-hydroxybenzoic acid Chemical compound OC(=O)C1=CC=C(O)C=C1 FJKROLUGYXJWQN-UHFFFAOYSA-N 0.000 description 1
• ZFTBZKVVGZNMJR-UHFFFAOYSA-N 5-chlorouracil Chemical compound ClC1=CNC(=O)NC1=O ZFTBZKVVGZNMJR-UHFFFAOYSA-N 0.000 description 1
• KELXHQACBIUYSE-UHFFFAOYSA-N 5-methoxy-1h-pyrimidine-2,4-dione Chemical compound COC1=CNC(=O)NC1=O KELXHQACBIUYSE-UHFFFAOYSA-N 0.000 description 1
• LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 description 1
• UDZRZGNQQSUDNP-UHFFFAOYSA-N 6-(aminomethyl)-5-methoxy-2-sulfanylidene-1H-pyrimidin-4-one Chemical compound COC=1C(NC(NC=1CN)=S)=O UDZRZGNQQSUDNP-UHFFFAOYSA-N 0.000 description 1
• BYPCVKNBGDKXLB-UHFFFAOYSA-N 6-(aminomethyl)-5-methyl-1h-pyrimidine-2,4-dione Chemical compound CC1=C(CN)NC(=O)NC1=O BYPCVKNBGDKXLB-UHFFFAOYSA-N 0.000 description 1
• DCPSTSVLRXOYGS-UHFFFAOYSA-N 6-amino-1h-pyrimidine-2-thione Chemical compound NC1=CC=NC(S)=N1 DCPSTSVLRXOYGS-UHFFFAOYSA-N 0.000 description 1
• 102100038222 60 kDa heat shock protein, mitochondrial Human genes 0.000 description 1
• 108010013238 70-kDa Ribosomal Protein S6 Kinases Proteins 0.000 description 1
• MSSXOMSJDRHRMC-UHFFFAOYSA-N 9H-purine-2,6-diamine Chemical compound NC1=NC(N)=C2NC=NC2=N1 MSSXOMSJDRHRMC-UHFFFAOYSA-N 0.000 description 1
• 102100032922 ATP-dependent 6-phosphofructokinase, muscle type Human genes 0.000 description 1
• 108010066676 Abrin Proteins 0.000 description 1
• 102100027211 Albumin Human genes 0.000 description 1
• 108010088751 Albumins Proteins 0.000 description 1
• 208000007848 Alcoholism Diseases 0.000 description 1
• OGSPWJRAVKPPFI-UHFFFAOYSA-N Alendronic Acid Chemical compound NCCCC(O)(P(O)(O)=O)P(O)(O)=O OGSPWJRAVKPPFI-UHFFFAOYSA-N 0.000 description 1
• GUBGYTABKSRVRQ-XLOQQCSPSA-N Alpha-Lactose Chemical compound O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)O[C@H]1O[C@@H]1[C@@H](CO)O[C@H](O)[C@H](O)[C@H]1O GUBGYTABKSRVRQ-XLOQQCSPSA-N 0.000 description 1
• 102400000345 Angiotensin-2 Human genes 0.000 description 1
• 101800000733 Angiotensin-2 Proteins 0.000 description 1
• 102000008102 Ankyrins Human genes 0.000 description 1
• 108010049777 Ankyrins Proteins 0.000 description 1
• 108010039627 Aprotinin Proteins 0.000 description 1
• 101000669426 Aspergillus restrictus Ribonuclease mitogillin Proteins 0.000 description 1
• 238000009020 BCA Protein Assay Kit Methods 0.000 description 1
• 241000894006 Bacteria Species 0.000 description 1
• DWRXFEITVBNRMK-UHFFFAOYSA-N Beta-D-1-Arabinofuranosylthymine Natural products O=C1NC(=O)C(C)=CN1C1C(O)C(O)C(CO)O1 DWRXFEITVBNRMK-UHFFFAOYSA-N 0.000 description 1
• 108010029692 Bisphosphoglycerate mutase Proteins 0.000 description 1
• 108010006654 Bleomycin Proteins 0.000 description 1
• 206010065687 Bone loss Diseases 0.000 description 1
• 206010068597 Bulbospinal muscular atrophy congenital Diseases 0.000 description 1
• 238000011746 C57BL/6J (JAX™ mouse strain) Methods 0.000 description 1
• 108091007381 CBL proteins Proteins 0.000 description 1
• 102100021786 CMP-N-acetylneuraminate-poly-alpha-2,8-sialyltransferase Human genes 0.000 description 1
• 101100454807 Caenorhabditis elegans lgg-1 gene Proteins 0.000 description 1
• 101100454808 Caenorhabditis elegans lgg-2 gene Proteins 0.000 description 1
• 101100217502 Caenorhabditis elegans lgg-3 gene Proteins 0.000 description 1
• 101000909256 Caldicellulosiruptor bescii (strain ATCC BAA-1888 / DSM 6725 / Z-1320) DNA polymerase I Proteins 0.000 description 1
• 241000282826 Camelus Species 0.000 description 1
• 241000282828 Camelus bactrianus Species 0.000 description 1
• 101710158575 Cap-specific mRNA (nucleoside-2'-O-)-methyltransferase Proteins 0.000 description 1
• 101710167800 Capsid assembly scaffolding protein Proteins 0.000 description 1
• OKTJSMMVPCPJKN-NJFSPNSNSA-N Carbon-14 Chemical compound [14C] OKTJSMMVPCPJKN-NJFSPNSNSA-N 0.000 description 1
• 201000009030 Carcinoma Diseases 0.000 description 1
• 208000024172 Cardiovascular disease Diseases 0.000 description 1
• DLGOEMSEDOSKAD-UHFFFAOYSA-N Carmustine Chemical compound ClCCNC(=O)N(N=O)CCCl DLGOEMSEDOSKAD-UHFFFAOYSA-N 0.000 description 1
• 102000014914 Carrier Proteins Human genes 0.000 description 1
• JOATXPAWOHTVSZ-UHFFFAOYSA-N Celiprolol Chemical compound CCN(CC)C(=O)NC1=CC=C(OCC(O)CNC(C)(C)C)C(C(C)=O)=C1 JOATXPAWOHTVSZ-UHFFFAOYSA-N 0.000 description 1
• 102000000844 Cell Surface Receptors Human genes 0.000 description 1
• 108010001857 Cell Surface Receptors Proteins 0.000 description 1
• 201000003728 Centronuclear myopathy Diseases 0.000 description 1
• 108010058432 Chaperonin 60 Proteins 0.000 description 1
• 201000006868 Charcot-Marie-Tooth disease type 3 Diseases 0.000 description 1
• GJSURZIOUXUGAL-UHFFFAOYSA-N Clonidine Chemical compound ClC1=CC=CC(Cl)=C1NC1=NCCN1 GJSURZIOUXUGAL-UHFFFAOYSA-N 0.000 description 1
• 235000016795 Cola Nutrition 0.000 description 1
• 244000228088 Cola acuminata Species 0.000 description 1
• 235000011824 Cola pachycarpa Nutrition 0.000 description 1
• 108020004635 Complementary DNA Proteins 0.000 description 1
• 108020004394 Complementary RNA Proteins 0.000 description 1
• 208000029323 Congenital myotonia Diseases 0.000 description 1
• MIKUYHXYGGJMLM-GIMIYPNGSA-N Crotonoside Natural products C1=NC2=C(N)NC(=O)N=C2N1[C@H]1O[C@@H](CO)[C@H](O)[C@@H]1O MIKUYHXYGGJMLM-GIMIYPNGSA-N 0.000 description 1
• 108010088874 Cullin 1 Proteins 0.000 description 1
• 102100039195 Cullin-1 Human genes 0.000 description 1
• 102100021906 Cyclin-O Human genes 0.000 description 1
• CMSMOCZEIVJLDB-UHFFFAOYSA-N Cyclophosphamide Chemical compound ClCCN(CCCl)P1(=O)NCCCO1 CMSMOCZEIVJLDB-UHFFFAOYSA-N 0.000 description 1
• 108010061641 Cystatin A Proteins 0.000 description 1
• 108010061642 Cystatin C Proteins 0.000 description 1
• 102100031237 Cystatin-A Human genes 0.000 description 1
• 102100026897 Cystatin-C Human genes 0.000 description 1
• UHDGCWIWMRVCDJ-CCXZUQQUSA-N Cytarabine Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@@H](O)[C@H](O)[C@@H](CO)O1 UHDGCWIWMRVCDJ-CCXZUQQUSA-N 0.000 description 1
• UHDGCWIWMRVCDJ-PSQAKQOGSA-N Cytidine Natural products O=C1N=C(N)C=CN1[C@@H]1[C@@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-PSQAKQOGSA-N 0.000 description 1
• 102100025287 Cytochrome b Human genes 0.000 description 1
• 108010052832 Cytochromes Proteins 0.000 description 1
• 102000018832 Cytochromes Human genes 0.000 description 1
• 108010075028 Cytochromes b Proteins 0.000 description 1
• FBPFZTCFMRRESA-FSIIMWSLSA-N D-Glucitol Natural products OC[C@H](O)[C@H](O)[C@@H](O)[C@H](O)CO FBPFZTCFMRRESA-FSIIMWSLSA-N 0.000 description 1
• FBPFZTCFMRRESA-KVTDHHQDSA-N D-Mannitol Chemical compound OC[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-KVTDHHQDSA-N 0.000 description 1
• FBPFZTCFMRRESA-JGWLITMVSA-N D-glucitol Chemical compound OC[C@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-JGWLITMVSA-N 0.000 description 1
• NYHBQMYGNKIUIF-UHFFFAOYSA-N D-guanosine Natural products C1=2NC(N)=NC(=O)C=2N=CN1C1OC(CO)C(O)C1O NYHBQMYGNKIUIF-UHFFFAOYSA-N 0.000 description 1
• 102000053602 DNA Human genes 0.000 description 1
• 238000000018 DNA microarray Methods 0.000 description 1
• 101000678286 Danio rerio Eukaryotic translation initiation factor 4E-binding protein 3-like Proteins 0.000 description 1
• WEAHRLBPCANXCN-UHFFFAOYSA-N Daunomycin Natural products CCC1(O)CC(OC2CC(N)C(O)C(C)O2)c3cc4C(=O)c5c(OC)cccc5C(=O)c4c(O)c3C1 WEAHRLBPCANXCN-UHFFFAOYSA-N 0.000 description 1
• CKTSBUTUHBMZGZ-UHFFFAOYSA-N Deoxycytidine Natural products O=C1N=C(N)C=CN1C1OC(CO)C(O)C1 CKTSBUTUHBMZGZ-UHFFFAOYSA-N 0.000 description 1
• 241000702421 Dependoparvovirus Species 0.000 description 1
• 229920002307 Dextran Polymers 0.000 description 1
• 239000004375 Dextrin Substances 0.000 description 1
• 229920001353 Dextrin Polymers 0.000 description 1
• 101000800913 Dictyostelium discoideum Eukaryotic translation initiation factor 4E-1A-binding protein homolog Proteins 0.000 description 1
• 108700043208 Dimauro disease Proteins 0.000 description 1
• 235000017274 Diospyros sandwicensis Nutrition 0.000 description 1
• 102000016607 Diphtheria Toxin Human genes 0.000 description 1
• 108010053187 Diphtheria Toxin Proteins 0.000 description 1
• 101000800906 Drosophila melanogaster Eukaryotic translation initiation factor 4E-binding protein Proteins 0.000 description 1
• 102100023266 Dual specificity mitogen-activated protein kinase kinase 2 Human genes 0.000 description 1
• 101710146529 Dual specificity mitogen-activated protein kinase kinase 2 Proteins 0.000 description 1
• 102100031918 E3 ubiquitin-protein ligase NEDD4 Human genes 0.000 description 1
• KCXVZYZYPLLWCC-UHFFFAOYSA-N EDTA Chemical compound OC(=O)CN(CC(O)=O)CCN(CC(O)=O)CC(O)=O KCXVZYZYPLLWCC-UHFFFAOYSA-N 0.000 description 1
• LVGKNOAMLMIIKO-UHFFFAOYSA-N Elaidinsaeure-aethylester Natural products CCCCCCCCC=CCCCCCCCC(=O)OCC LVGKNOAMLMIIKO-UHFFFAOYSA-N 0.000 description 1
• MBYXEBXZARTUSS-QLWBXOBMSA-N Emetamine Natural products O(C)c1c(OC)cc2c(c(C[C@@H]3[C@H](CC)CN4[C@H](c5c(cc(OC)c(OC)c5)CC4)C3)ncc2)c1 MBYXEBXZARTUSS-QLWBXOBMSA-N 0.000 description 1
• 108010042407 Endonucleases Proteins 0.000 description 1
• 241000283086 Equidae Species 0.000 description 1
• 102000008014 Eukaryotic Initiation Factor-2 Human genes 0.000 description 1
• 108010089791 Eukaryotic Initiation Factor-2 Proteins 0.000 description 1
• 101710082714 Exotoxin A Proteins 0.000 description 1
• 108091072033 F-box protein family Proteins 0.000 description 1
• 102000002090 Fibronectin type III Human genes 0.000 description 1
• 108050009401 Fibronectin type III Proteins 0.000 description 1
• 229930091371 Fructose Natural products 0.000 description 1
• RFSUNEUAIZKAJO-ARQDHWQXSA-N Fructose Chemical compound OC[C@H]1O[C@](O)(CO)[C@@H](O)[C@@H]1O RFSUNEUAIZKAJO-ARQDHWQXSA-N 0.000 description 1
• 239000005715 Fructose Substances 0.000 description 1
• 108010010803 Gelatin Proteins 0.000 description 1
• 108700004714 Gelonium multiflorum GEL Proteins 0.000 description 1
• WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 description 1
• 108010001483 Glycogen Synthase Proteins 0.000 description 1
• 102100029492 Glycogen phosphorylase, muscle form Human genes 0.000 description 1
• 102000003886 Glycoproteins Human genes 0.000 description 1
• 108090000288 Glycoproteins Proteins 0.000 description 1
• 108010026389 Gramicidin Proteins 0.000 description 1
• 108010017080 Granulocyte Colony-Stimulating Factor Proteins 0.000 description 1
• 102000004269 Granulocyte Colony-Stimulating Factor Human genes 0.000 description 1
• 102000016285 Guanine Nucleotide Exchange Factors Human genes 0.000 description 1
• 108010067218 Guanine Nucleotide Exchange Factors Proteins 0.000 description 1
• 239000007995 HEPES buffer Substances 0.000 description 1
• XLYOFNOQVPJJNP-ZSJDYOACSA-N Heavy water Chemical compound [2H]O[2H] XLYOFNOQVPJJNP-ZSJDYOACSA-N 0.000 description 1
• 208000006411 Hereditary Sensory and Motor Neuropathy Diseases 0.000 description 1
• 101000730838 Homo sapiens ATP-dependent 6-phosphofructokinase, muscle type Proteins 0.000 description 1
• 101000616698 Homo sapiens CMP-N-acetylneuraminate-poly-alpha-2,8-sialyltransferase Proteins 0.000 description 1
• 101000897441 Homo sapiens Cyclin-O Proteins 0.000 description 1
• 101000636713 Homo sapiens E3 ubiquitin-protein ligase NEDD4 Proteins 0.000 description 1
• 101001027128 Homo sapiens Fibronectin Proteins 0.000 description 1
• 101000700475 Homo sapiens Glycogen phosphorylase, muscle form Proteins 0.000 description 1
• 101000959820 Homo sapiens Interferon alpha-1/13 Proteins 0.000 description 1
• 101001128634 Homo sapiens NADH dehydrogenase [ubiquinone] 1 beta subcomplex subunit 2, mitochondrial Proteins 0.000 description 1
• 101000724418 Homo sapiens Neutral amino acid transporter B(0) Proteins 0.000 description 1
• 101100351819 Homo sapiens PGAM2 gene Proteins 0.000 description 1
• 101001059454 Homo sapiens Serine/threonine-protein kinase MARK2 Proteins 0.000 description 1
• 206010020850 Hyperthyroidism Diseases 0.000 description 1
• UGQMRVRMYYASKQ-UHFFFAOYSA-N Hypoxanthine nucleoside Natural products OC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 UGQMRVRMYYASKQ-UHFFFAOYSA-N 0.000 description 1
• 102000043333 Immunoglobulin I-set Human genes 0.000 description 1
• 108700038397 Immunoglobulin I-set Proteins 0.000 description 1
• 101000668058 Infectious salmon anemia virus (isolate Atlantic salmon/Norway/810/9/99) RNA-directed RNA polymerase catalytic subunit Proteins 0.000 description 1
• 208000022559 Inflammatory bowel disease Diseases 0.000 description 1
• 102100037872 Intercellular adhesion molecule 2 Human genes 0.000 description 1
• 101710148794 Intercellular adhesion molecule 2 Proteins 0.000 description 1
• 102100040019 Interferon alpha-1/13 Human genes 0.000 description 1
• 102000003815 Interleukin-11 Human genes 0.000 description 1
• 108090000177 Interleukin-11 Proteins 0.000 description 1
• 108010063738 Interleukins Proteins 0.000 description 1
• 102000015696 Interleukins Human genes 0.000 description 1
• 108091092195 Intron Proteins 0.000 description 1
• PIWKPBJCKXDKJR-UHFFFAOYSA-N Isoflurane Chemical compound FC(F)OC(Cl)C(F)(F)F PIWKPBJCKXDKJR-UHFFFAOYSA-N 0.000 description 1
• 102000003855 L-lactate dehydrogenase Human genes 0.000 description 1
• 108700023483 L-lactate dehydrogenases Proteins 0.000 description 1
• FBOZXECLQNJBKD-ZDUSSCGKSA-N L-methotrexate Chemical compound C=1N=C2N=C(N)N=C(N)C2=NC=1CN(C)C1=CC=C(C(=O)N[C@@H](CCC(O)=O)C(O)=O)C=C1 FBOZXECLQNJBKD-ZDUSSCGKSA-N 0.000 description 1
• 108700006394 Lactate Dehydrogenase Deficiency Proteins 0.000 description 1
• GUBGYTABKSRVRQ-QKKXKWKRSA-N Lactose Natural products OC[C@H]1O[C@@H](O[C@H]2[C@H](O)[C@@H](O)C(O)O[C@@H]2CO)[C@H](O)[C@@H](O)[C@H]1O GUBGYTABKSRVRQ-QKKXKWKRSA-N 0.000 description 1
• 201000010743 Lambert-Eaton myasthenic syndrome Diseases 0.000 description 1
• 241000713666 Lentivirus Species 0.000 description 1
• 108010006444 Leucine-Rich Repeat Proteins Proteins 0.000 description 1
• NNJVILVZKWQKPM-UHFFFAOYSA-N Lidocaine Chemical compound CCN(CC)CC(=O)NC1=C(C)C=CC=C1C NNJVILVZKWQKPM-UHFFFAOYSA-N 0.000 description 1
• 208000017170 Lipid metabolism disease Diseases 0.000 description 1
• 102000019298 Lipocalin Human genes 0.000 description 1
• 108050006654 Lipocalin Proteins 0.000 description 1
• 208000019693 Lung disease Diseases 0.000 description 1
• 229940122696 MAP kinase inhibitor Drugs 0.000 description 1
• 239000007993 MOPS buffer Substances 0.000 description 1
• 241000282567 Macaca fascicularis Species 0.000 description 1
• GUBGYTABKSRVRQ-PICCSMPSSA-N Maltose Natural products O[C@@H]1[C@@H](O)[C@H](O)[C@@H](CO)O[C@@H]1O[C@@H]1[C@@H](CO)OC(O)[C@H](O)[C@H]1O GUBGYTABKSRVRQ-PICCSMPSSA-N 0.000 description 1
• 229930195725 Mannitol Natural products 0.000 description 1
• 102000002274 Matrix Metalloproteinases Human genes 0.000 description 1
• 108010000684 Matrix Metalloproteinases Proteins 0.000 description 1
• 108700011259 MicroRNAs Proteins 0.000 description 1
• VFKZTMPDYBFSTM-KVTDHHQDSA-N Mitobronitol Chemical compound BrC[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CBr VFKZTMPDYBFSTM-KVTDHHQDSA-N 0.000 description 1
• 102000004232 Mitogen-Activated Protein Kinase Kinases Human genes 0.000 description 1
• 108090000744 Mitogen-Activated Protein Kinase Kinases Proteins 0.000 description 1
• 229930192392 Mitomycin Natural products 0.000 description 1
• 244000302512 Momordica charantia Species 0.000 description 1
• 235000009811 Momordica charantia Nutrition 0.000 description 1
• WPNJAUFVNXKLIM-UHFFFAOYSA-N Moxonidine Chemical compound COC1=NC(C)=NC(Cl)=C1NC1=NCCN1 WPNJAUFVNXKLIM-UHFFFAOYSA-N 0.000 description 1
• 101500024857 Mus musculus Ubiquitin Proteins 0.000 description 1
• 101500025138 Mus musculus Ubiquitin Proteins 0.000 description 1
• 101500026511 Mus musculus Ubiquitin Proteins 0.000 description 1
• 101500027538 Mus musculus Ubiquitin Proteins 0.000 description 1
• 208000029578 Muscle disease Diseases 0.000 description 1
• 208000010316 Myotonia congenita Diseases 0.000 description 1
• SGSSKEDGVONRGC-UHFFFAOYSA-N N(2)-methylguanine Chemical compound O=C1NC(NC)=NC2=C1N=CN2 SGSSKEDGVONRGC-UHFFFAOYSA-N 0.000 description 1
• 102100032194 NADH dehydrogenase [ubiquinone] 1 beta subcomplex subunit 2, mitochondrial Human genes 0.000 description 1
• 108700019961 Neoplasm Genes Proteins 0.000 description 1
• 102000048850 Neoplasm Genes Human genes 0.000 description 1
• 241000221960 Neurospora Species 0.000 description 1
• 102100028267 Neutral amino acid transporter B(0) Human genes 0.000 description 1
• 239000000020 Nitrocellulose Substances 0.000 description 1
• 108091092724 Noncoding DNA Proteins 0.000 description 1
• QBRJRFULDLIDKL-UHFFFAOYSA-N OC1=C(C(C(C2=CC=3C=CC4=NC5=C6C=C7C=CC=CC7=CC6=CC=C5N=C4C=3C=C12)=O)=O)O Chemical compound OC1=C(C(C(C2=CC=3C=CC4=NC5=C6C=C7C=CC=CC7=CC6=CC=C5N=C4C=3C=C12)=O)=O)O QBRJRFULDLIDKL-UHFFFAOYSA-N 0.000 description 1
• 108700026244 Open Reading Frames Proteins 0.000 description 1
• 229930012538 Paclitaxel Natural products 0.000 description 1
• 102000003992 Peroxidases Human genes 0.000 description 1
• 208000037581 Persistent Infection Diseases 0.000 description 1
• 241000286209 Phasianidae Species 0.000 description 1
• 229940122907 Phosphatase inhibitor Drugs 0.000 description 1
• 102100037385 Phosphoglycerate mutase 2 Human genes 0.000 description 1
• 108010073135 Phosphorylases Proteins 0.000 description 1
• 102000009097 Phosphorylases Human genes 0.000 description 1
• 101100413173 Phytolacca americana PAP2 gene Proteins 0.000 description 1
• 206010035664 Pneumonia Diseases 0.000 description 1
• 239000004698 Polyethylene Substances 0.000 description 1
• 229920002685 Polyoxyl 35CastorOil Polymers 0.000 description 1
• 101710130420 Probable capsid assembly scaffolding protein Proteins 0.000 description 1
• 206010036790 Productive cough Diseases 0.000 description 1
• 102000007327 Protamines Human genes 0.000 description 1
• 108010007568 Protamines Proteins 0.000 description 1
• 101800004937 Protein C Proteins 0.000 description 1
• 102000017975 Protein C Human genes 0.000 description 1
• 102000002727 Protein Tyrosine Phosphatase Human genes 0.000 description 1
• 229940123573 Protein synthesis inhibitor Drugs 0.000 description 1
• 102000055251 Proto-Oncogene Proteins c-cbl Human genes 0.000 description 1
• 241000589517 Pseudomonas aeruginosa Species 0.000 description 1
• 101000902592 Pyrococcus furiosus (strain ATCC 43587 / DSM 3638 / JCM 8422 / Vc1) DNA polymerase Proteins 0.000 description 1
• 108091008103 RNA aptamers Proteins 0.000 description 1
• 241000269435 Rana <genus> Species 0.000 description 1
• 108020004511 Recombinant DNA Proteins 0.000 description 1
• 102000003661 Ribonuclease III Human genes 0.000 description 1
• 108010057163 Ribonuclease III Proteins 0.000 description 1
• 108010083644 Ribonucleases Proteins 0.000 description 1
• 102000006382 Ribonucleases Human genes 0.000 description 1
• CQXADFVORZEARL-UHFFFAOYSA-N Rilmenidine Chemical compound C1CC1C(C1CC1)NC1=NCCO1 CQXADFVORZEARL-UHFFFAOYSA-N 0.000 description 1
• 102000014400 SH2 domains Human genes 0.000 description 1
• 108050003452 SH2 domains Proteins 0.000 description 1
• 101800001700 Saposin-D Proteins 0.000 description 1
• 101710204410 Scaffold protein Proteins 0.000 description 1
• 102100028904 Serine/threonine-protein kinase MARK2 Human genes 0.000 description 1
• 108020004682 Single-Stranded DNA Proteins 0.000 description 1
• 241000251131 Sphyrna Species 0.000 description 1
• 208000003954 Spinal Muscular Atrophies of Childhood Diseases 0.000 description 1
• 108010088160 Staphylococcal Protein A Proteins 0.000 description 1
• 229920002472 Starch Polymers 0.000 description 1
• 108091081024 Start codon Proteins 0.000 description 1
• ZSJLQEPLLKMAKR-UHFFFAOYSA-N Streptozotocin Natural products O=NN(C)C(=O)NC1C(O)OC(CO)C(O)C1O ZSJLQEPLLKMAKR-UHFFFAOYSA-N 0.000 description 1
• 208000032978 Structural Congenital Myopathies Diseases 0.000 description 1
• 241000272534 Struthio camelus Species 0.000 description 1
• 238000000692 Student's t-test Methods 0.000 description 1
• CZMRCDWAGMRECN-UGDNZRGBSA-N Sucrose Chemical compound O[C@H]1[C@H](O)[C@@H](CO)O[C@@]1(CO)O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 CZMRCDWAGMRECN-UGDNZRGBSA-N 0.000 description 1
• 229930006000 Sucrose Natural products 0.000 description 1
• NINIDFKCEFEMDL-UHFFFAOYSA-N Sulfur Chemical group [S] NINIDFKCEFEMDL-UHFFFAOYSA-N 0.000 description 1
• 102000019197 Superoxide Dismutase Human genes 0.000 description 1
• 108010012715 Superoxide dismutase Proteins 0.000 description 1
• 101710137500 T7 RNA polymerase Proteins 0.000 description 1
• 102100038126 Tenascin Human genes 0.000 description 1
• 108010008125 Tenascin Proteins 0.000 description 1
• 108010000499 Thromboplastin Proteins 0.000 description 1
• 102100030859 Tissue factor Human genes 0.000 description 1
• 102000004357 Transferases Human genes 0.000 description 1
• 108090000992 Transferases Proteins 0.000 description 1
• HDTRYLNUVZCQOY-WSWWMNSNSA-N Trehalose Natural products O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)O[C@@H]1O[C@@H]1[C@H](O)[C@@H](O)[C@@H](O)[C@@H](CO)O1 HDTRYLNUVZCQOY-WSWWMNSNSA-N 0.000 description 1
• 102000004243 Tubulin Human genes 0.000 description 1
• 108090000704 Tubulin Proteins 0.000 description 1
• GBOGMAARMMDZGR-UHFFFAOYSA-N UNPD149280 Natural products N1C(=O)C23OC(=O)C=CC(O)CCCC(C)CC=CC3C(O)C(=C)C(C)C2C1CC1=CC=CC=C1 GBOGMAARMMDZGR-UHFFFAOYSA-N 0.000 description 1
• 102100023543 Vascular cell adhesion protein 1 Human genes 0.000 description 1
• 240000001866 Vernicia fordii Species 0.000 description 1
• 241000282840 Vicugna vicugna Species 0.000 description 1
• 241000700605 Viruses Species 0.000 description 1
• 208000026481 Werdnig-Hoffmann disease Diseases 0.000 description 1
• 208000006269 X-Linked Bulbo-Spinal Atrophy Diseases 0.000 description 1
• CHKFLBOLYREYDO-SHYZEUOFSA-N [[(2s,4r,5r)-5-(4-amino-2-oxopyrimidin-1-yl)-4-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl] phosphono hydrogen phosphate Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@H](O)C[C@@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)O1 CHKFLBOLYREYDO-SHYZEUOFSA-N 0.000 description 1
• 230000001594 aberrant effect Effects 0.000 description 1
• 230000005856 abnormality Effects 0.000 description 1
• 229960002122 acebutolol Drugs 0.000 description 1
• GOEMGAFJFRBGGG-UHFFFAOYSA-N acebutolol Chemical compound CCCC(=O)NC1=CC=C(OCC(O)CNC(C)C)C(C(C)=O)=C1 GOEMGAFJFRBGGG-UHFFFAOYSA-N 0.000 description 1
• DPXJVFZANSGRMM-UHFFFAOYSA-N acetic acid;2,3,4,5,6-pentahydroxyhexanal;sodium Chemical compound [Na].CC(O)=O.OCC(O)C(O)C(O)C(O)C=O DPXJVFZANSGRMM-UHFFFAOYSA-N 0.000 description 1
• 239000000061 acid fraction Substances 0.000 description 1
• 230000002378 acidificating effect Effects 0.000 description 1
• 125000000641 acridinyl group Chemical group C1(=CC=CC2=NC3=CC=CC=C3C=C12)* 0.000 description 1
• 229930183665 actinomycin Natural products 0.000 description 1
• 230000003213 activating effect Effects 0.000 description 1
• 239000013543 active substance Substances 0.000 description 1
• 230000001154 acute effect Effects 0.000 description 1
• 229960000643 adenine Drugs 0.000 description 1
• 201000006960 adult spinal muscular atrophy Diseases 0.000 description 1
• 230000002411 adverse Effects 0.000 description 1
• 239000011543 agarose gel Substances 0.000 description 1
• 230000032683 aging Effects 0.000 description 1
• 150000001299 aldehydes Chemical class 0.000 description 1
• 229940062527 alendronate Drugs 0.000 description 1
• 229940100198 alkylating agent Drugs 0.000 description 1
• 239000002168 alkylating agent Substances 0.000 description 1
• HDTRYLNUVZCQOY-LIZSDCNHSA-N alpha,alpha-trehalose Chemical compound O[C@@H]1[C@@H](O)[C@H](O)[C@@H](CO)O[C@@H]1O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 HDTRYLNUVZCQOY-LIZSDCNHSA-N 0.000 description 1
• WQZGKKKJIJFFOK-PHYPRBDBSA-N alpha-D-galactose Chemical compound OC[C@H]1O[C@H](O)[C@H](O)[C@@H](O)[C@H]1O WQZGKKKJIJFFOK-PHYPRBDBSA-N 0.000 description 1
• CJCSPKMFHVPWAR-JTQLQIEISA-N alpha-methyl-L-dopa Chemical compound OC(=O)[C@](N)(C)CC1=CC=C(O)C(O)=C1 CJCSPKMFHVPWAR-JTQLQIEISA-N 0.000 description 1
• 229960002213 alprenolol Drugs 0.000 description 1
• PAZJSJFMUHDSTF-UHFFFAOYSA-N alprenolol Chemical compound CC(C)NCC(O)COC1=CC=CC=C1CC=C PAZJSJFMUHDSTF-UHFFFAOYSA-N 0.000 description 1
• 229950006323 angiotensin ii Drugs 0.000 description 1
• 229940045799 anthracyclines and related substance Drugs 0.000 description 1
• 230000000844 anti-bacterial effect Effects 0.000 description 1
• 229940121363 anti-inflammatory agent Drugs 0.000 description 1
• 239000002260 anti-inflammatory agent Substances 0.000 description 1
• 230000000340 anti-metabolite Effects 0.000 description 1
• 229940088710 antibiotic agent Drugs 0.000 description 1
• 230000009830 antibody antigen interaction Effects 0.000 description 1
• 229940121375 antifungal agent Drugs 0.000 description 1
• 239000003429 antifungal agent Substances 0.000 description 1
• 229940100197 antimetabolite Drugs 0.000 description 1
• 239000002256 antimetabolite Substances 0.000 description 1
• 239000003080 antimitotic agent Substances 0.000 description 1
• 239000003443 antiviral agent Substances 0.000 description 1
• 229960004405 aprotinin Drugs 0.000 description 1
• 238000009360 aquaculture Methods 0.000 description 1
• 244000144974 aquaculture Species 0.000 description 1
• 238000003491 array Methods 0.000 description 1
• 229960002274 atenolol Drugs 0.000 description 1
• 230000008267 autocrine signaling Effects 0.000 description 1
• 230000001363 autoimmune Effects 0.000 description 1
• 230000001580 bacterial effect Effects 0.000 description 1
• 108010028263 bacteriophage T3 RNA polymerase Proteins 0.000 description 1
• UHOVQNZJYSORNB-UHFFFAOYSA-N benzene Substances C1=CC=CC=C1 UHOVQNZJYSORNB-UHFFFAOYSA-N 0.000 description 1
• WPYMKLBDIGXBTP-UHFFFAOYSA-N benzoic acid group Chemical group C(C1=CC=CC=C1)(=O)O WPYMKLBDIGXBTP-UHFFFAOYSA-N 0.000 description 1
• DZBUGLKDJFMEHC-UHFFFAOYSA-N benzoquinolinylidene Natural products C1=CC=CC2=CC3=CC=CC=C3N=C21 DZBUGLKDJFMEHC-UHFFFAOYSA-N 0.000 description 1
• 208000036815 beta tubulin Diseases 0.000 description 1
• IQFYYKKMVGJFEH-UHFFFAOYSA-N beta-L-thymidine Natural products O=C1NC(=O)C(C)=CN1C1OC(CO)C(O)C1 IQFYYKKMVGJFEH-UHFFFAOYSA-N 0.000 description 1
• DRTQHJPVMGBUCF-PSQAKQOGSA-N beta-L-uridine Natural products O[C@H]1[C@@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-PSQAKQOGSA-N 0.000 description 1
• 229960004324 betaxolol Drugs 0.000 description 1
• CHDPSNLJFOQTRK-UHFFFAOYSA-N betaxolol hydrochloride Chemical compound [Cl-].C1=CC(OCC(O)C[NH2+]C(C)C)=CC=C1CCOCC1CC1 CHDPSNLJFOQTRK-UHFFFAOYSA-N 0.000 description 1
• 108091008324 binding proteins Proteins 0.000 description 1
• 238000004166 bioassay Methods 0.000 description 1
• 239000003181 biological factor Substances 0.000 description 1
• 229920001222 biopolymer Polymers 0.000 description 1
• 238000001574 biopsy Methods 0.000 description 1
• 229960002685 biotin Drugs 0.000 description 1
• 235000020958 biotin Nutrition 0.000 description 1
• 239000011616 biotin Substances 0.000 description 1
• 229960002781 bisoprolol Drugs 0.000 description 1
• VHYCDWMUTMEGQY-UHFFFAOYSA-N bisoprolol Chemical compound CC(C)NCC(O)COC1=CC=C(COCCOC(C)C)C=C1 VHYCDWMUTMEGQY-UHFFFAOYSA-N 0.000 description 1
• 229960001561 bleomycin Drugs 0.000 description 1
• OYVAGSVQBOHSSS-UAPAGMARSA-O bleomycin A2 Chemical compound N([C@H](C(=O)N[C@H](C)[C@@H](O)[C@H](C)C(=O)N[C@@H]([C@H](O)C)C(=O)NCCC=1SC=C(N=1)C=1SC=C(N=1)C(=O)NCCC[S+](C)C)[C@@H](O[C@H]1[C@H]([C@@H](O)[C@H](O)[C@H](CO)O1)O[C@@H]1[C@H]([C@@H](OC(N)=O)[C@H](O)[C@@H](CO)O1)O)C=1N=CNC=1)C(=O)C1=NC([C@H](CC(N)=O)NC[C@H](N)C(N)=O)=NC(N)=C1C OYVAGSVQBOHSSS-UAPAGMARSA-O 0.000 description 1
• 239000010839 body fluid Substances 0.000 description 1
• 210000000476 body water Anatomy 0.000 description 1
• 238000009835 boiling Methods 0.000 description 1
• RSIHSRDYCUFFLA-DYKIIFRCSA-N boldenone Chemical compound O=C1C=C[C@]2(C)[C@H]3CC[C@](C)([C@H](CC4)O)[C@@H]4[C@@H]3CCC2=C1 RSIHSRDYCUFFLA-DYKIIFRCSA-N 0.000 description 1
• 210000000988 bone and bone Anatomy 0.000 description 1
• 229950005341 bucindolol Drugs 0.000 description 1
• 244000309464 bull Species 0.000 description 1
• 229960002092 busulfan Drugs 0.000 description 1
• 239000001273 butane Substances 0.000 description 1
• 239000001506 calcium phosphate Substances 0.000 description 1
• 229910000389 calcium phosphate Inorganic materials 0.000 description 1
• 235000011010 calcium phosphates Nutrition 0.000 description 1
• 244000309466 calf Species 0.000 description 1
• 239000003560 cancer drug Substances 0.000 description 1
• 229940086673 canrenoate Drugs 0.000 description 1
• PBKZPPIHUVSDNM-WNHSNXHDSA-N canrenoic acid Chemical compound O=C1CC[C@]2(C)[C@H]3CC[C@](C)([C@](CC4)(O)CCC(O)=O)[C@@H]4[C@@H]3C=CC2=C1 PBKZPPIHUVSDNM-WNHSNXHDSA-N 0.000 description 1
• 239000002775 capsule Substances 0.000 description 1
• 208000020450 carbohydrate metabolism disease Diseases 0.000 description 1
• 235000014633 carbohydrates Nutrition 0.000 description 1
• 125000004432 carbon atom Chemical group C* 0.000 description 1
• 239000001768 carboxy methyl cellulose Substances 0.000 description 1
• 150000001735 carboxylic acids Chemical class 0.000 description 1
• 229960005243 carmustine Drugs 0.000 description 1
• 229960004203 carnitine Drugs 0.000 description 1
• 229960001222 carteolol Drugs 0.000 description 1
• LWAFSWPYPHEXKX-UHFFFAOYSA-N carteolol Chemical compound N1C(=O)CCC2=C1C=CC=C2OCC(O)CNC(C)(C)C LWAFSWPYPHEXKX-UHFFFAOYSA-N 0.000 description 1
• 229960004195 carvedilol Drugs 0.000 description 1
• NPAKNKYSJIDKMW-UHFFFAOYSA-N carvedilol Chemical compound COC1=CC=CC=C1OCCNCC(O)COC1=CC=CC2=NC3=CC=C[CH]C3=C12 NPAKNKYSJIDKMW-UHFFFAOYSA-N 0.000 description 1
• 238000005266 casting Methods 0.000 description 1
• 229920006317 cationic polymer Polymers 0.000 description 1
• 229960002320 celiprolol Drugs 0.000 description 1
• 238000004113 cell culture Methods 0.000 description 1
• 230000005889 cellular cytotoxicity Effects 0.000 description 1
• 230000001413 cellular effect Effects 0.000 description 1
• 239000001913 cellulose Substances 0.000 description 1
• 229920002678 cellulose Polymers 0.000 description 1
• 235000010980 cellulose Nutrition 0.000 description 1
• 229940083181 centrally acting adntiadrenergic agent methyldopa Drugs 0.000 description 1
• 210000001175 cerebrospinal fluid Anatomy 0.000 description 1
• 239000002738 chelating agent Substances 0.000 description 1
• NDAYQJDHGXTBJL-MWWSRJDJSA-N chembl557217 Chemical compound C1=CC=C2C(C[C@H](NC(=O)[C@@H](CC(C)C)NC(=O)[C@H](CC=3C4=CC=CC=C4NC=3)NC(=O)[C@@H](CC(C)C)NC(=O)[C@H](CC=3C4=CC=CC=C4NC=3)NC(=O)[C@@H](CC(C)C)NC(=O)[C@H](CC=3C4=CC=CC=C4NC=3)NC(=O)[C@@H](C(C)C)NC(=O)[C@H](C(C)C)NC(=O)[C@@H](C(C)C)NC(=O)[C@H](C)NC(=O)[C@H](NC(=O)CNC(=O)[C@@H](NC=O)C(C)C)CC(C)C)C(=O)NCCO)=CNC2=C1 NDAYQJDHGXTBJL-MWWSRJDJSA-N 0.000 description 1
• 230000000973 chemotherapeutic effect Effects 0.000 description 1
• 229960004630 chlorambucil Drugs 0.000 description 1
• 229960004926 chlorobutanol Drugs 0.000 description 1
• 235000012000 cholesterol Nutrition 0.000 description 1
• 208000037976 chronic inflammation Diseases 0.000 description 1
• 230000006020 chronic inflammation Effects 0.000 description 1
• 229960004316 cisplatin Drugs 0.000 description 1
• 238000003776 cleavage reaction Methods 0.000 description 1
• 229960002896 clonidine Drugs 0.000 description 1
• 229940110456 cocoa butter Drugs 0.000 description 1
• 235000019868 cocoa butter Nutrition 0.000 description 1
• 229960001338 colchicine Drugs 0.000 description 1
• 239000003086 colorant Substances 0.000 description 1
• 229940000425 combination drug Drugs 0.000 description 1
• 230000004154 complement system Effects 0.000 description 1
• 239000003184 complementary RNA Substances 0.000 description 1
• 239000002131 composite material Substances 0.000 description 1
• 238000009833 condensation Methods 0.000 description 1
• 230000005494 condensation Effects 0.000 description 1
• 108091036078 conserved sequence Proteins 0.000 description 1
• 239000000470 constituent Substances 0.000 description 1
• 230000008602 contraction Effects 0.000 description 1
• 238000007796 conventional method Methods 0.000 description 1
• 239000002285 corn oil Substances 0.000 description 1
• 235000005687 corn oil Nutrition 0.000 description 1
• 239000007822 coupling agent Substances 0.000 description 1
• 230000009260 cross reactivity Effects 0.000 description 1
• WZHCOOQXZCIUNC-UHFFFAOYSA-N cyclandelate Chemical compound C1C(C)(C)CC(C)CC1OC(=O)C(O)C1=CC=CC=C1 WZHCOOQXZCIUNC-UHFFFAOYSA-N 0.000 description 1
• 229960004397 cyclophosphamide Drugs 0.000 description 1
• 235000018417 cysteine Nutrition 0.000 description 1
• 229960000684 cytarabine Drugs 0.000 description 1
• UHDGCWIWMRVCDJ-ZAKLUEHWSA-N cytidine Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-ZAKLUEHWSA-N 0.000 description 1
• GBOGMAARMMDZGR-TYHYBEHESA-N cytochalasin B Chemical compound C([C@H]1[C@@H]2[C@@H](C([C@@H](O)[C@@H]3/C=C/C[C@H](C)CCC[C@@H](O)/C=C/C(=O)O[C@@]23C(=O)N1)=C)C)C1=CC=CC=C1 GBOGMAARMMDZGR-TYHYBEHESA-N 0.000 description 1
• GBOGMAARMMDZGR-JREHFAHYSA-N cytochalasin B Natural products C[C@H]1CCC[C@@H](O)C=CC(=O)O[C@@]23[C@H](C=CC1)[C@H](O)C(=C)[C@@H](C)[C@@H]2[C@H](Cc4ccccc4)NC3=O GBOGMAARMMDZGR-JREHFAHYSA-N 0.000 description 1
• 230000009089 cytolysis Effects 0.000 description 1
• 210000000805 cytoplasm Anatomy 0.000 description 1
• 229940104302 cytosine Drugs 0.000 description 1
• 230000007850 degeneration Effects 0.000 description 1
• RSIHSRDYCUFFLA-UHFFFAOYSA-N dehydrotestosterone Natural products O=C1C=CC2(C)C3CCC(C)(C(CC4)O)C4C3CCC2=C1 RSIHSRDYCUFFLA-UHFFFAOYSA-N 0.000 description 1
• 230000003111 delayed effect Effects 0.000 description 1
• 229960001251 denosumab Drugs 0.000 description 1
• 238000000326 densiometry Methods 0.000 description 1
• 239000007933 dermal patch Substances 0.000 description 1
• 201000001981 dermatomyositis Diseases 0.000 description 1
• 230000001627 detrimental effect Effects 0.000 description 1
• 235000019425 dextrin Nutrition 0.000 description 1
• 239000008121 dextrose Substances 0.000 description 1
• 238000003745 diagnosis Methods 0.000 description 1
• 238000010586 diagram Methods 0.000 description 1
• 230000029087 digestion Effects 0.000 description 1
• ZLFRJHOBQVVTOJ-UHFFFAOYSA-N dimethyl hexanediimidate Chemical compound COC(=N)CCCCC(=N)OC ZLFRJHOBQVVTOJ-UHFFFAOYSA-N 0.000 description 1
• 206010013023 diphtheria Diseases 0.000 description 1
• 231100000676 disease causative agent Toxicity 0.000 description 1
• 238000009826 distribution Methods 0.000 description 1
• ZWIBGKZDAWNIFC-UHFFFAOYSA-N disuccinimidyl suberate Chemical compound O=C1CCC(=O)N1OC(=O)CCCCCCC(=O)ON1C(=O)CCC1=O ZWIBGKZDAWNIFC-UHFFFAOYSA-N 0.000 description 1
• 125000002228 disulfide group Chemical group 0.000 description 1
• 229960001389 doxazosin Drugs 0.000 description 1
• RUZYUOTYCVRMRZ-UHFFFAOYSA-N doxazosin Chemical compound C1OC2=CC=CC=C2OC1C(=O)N(CC1)CCN1C1=NC(N)=C(C=C(C(OC)=C2)OC)C2=N1 RUZYUOTYCVRMRZ-UHFFFAOYSA-N 0.000 description 1
• 238000009509 drug development Methods 0.000 description 1
• 239000012893 effector ligand Substances 0.000 description 1
• 230000005672 electromagnetic field Effects 0.000 description 1
• 238000010828 elution Methods 0.000 description 1
• 239000002895 emetic Substances 0.000 description 1
• AUVVAXYIELKVAI-UWBTVBNJSA-N emetine Natural products N1CCC2=CC(OC)=C(OC)C=C2[C@H]1C[C@H]1C[C@H]2C3=CC(OC)=C(OC)C=C3CCN2C[C@H]1CC AUVVAXYIELKVAI-UWBTVBNJSA-N 0.000 description 1
• 239000003995 emulsifying agent Substances 0.000 description 1
• 239000000839 emulsion Substances 0.000 description 1
• 230000002124 endocrine Effects 0.000 description 1
• 108010048367 enhanced green fluorescent protein Proteins 0.000 description 1
• 239000003623 enhancer Substances 0.000 description 1
• 230000002708 enhancing effect Effects 0.000 description 1
• 108010028531 enomycin Proteins 0.000 description 1
• 239000002702 enteric coating Substances 0.000 description 1
• 238000009505 enteric coating Methods 0.000 description 1
• 229960003745 esmolol Drugs 0.000 description 1
• AQNDDEOPVVGCPG-UHFFFAOYSA-N esmolol Chemical compound COC(=O)CCC1=CC=C(OCC(O)CNC(C)C)C=C1 AQNDDEOPVVGCPG-UHFFFAOYSA-N 0.000 description 1
• 150000002148 esters Chemical class 0.000 description 1
• BEFDCLMNVWHSGT-UHFFFAOYSA-N ethenylcyclopentane Chemical compound C=CC1CCCC1 BEFDCLMNVWHSGT-UHFFFAOYSA-N 0.000 description 1
• ZMMJGEGLRURXTF-UHFFFAOYSA-N ethidium bromide Chemical compound [Br-].C12=CC(N)=CC=C2C2=CC=C(N)C=C2[N+](CC)=C1C1=CC=CC=C1 ZMMJGEGLRURXTF-UHFFFAOYSA-N 0.000 description 1
• 229960005542 ethidium bromide Drugs 0.000 description 1
• LVGKNOAMLMIIKO-QXMHVHEDSA-N ethyl oleate Chemical compound CCCCCCCC\C=C/CCCCCCCC(=O)OCC LVGKNOAMLMIIKO-QXMHVHEDSA-N 0.000 description 1
• 229940093471 ethyl oleate Drugs 0.000 description 1
• VJJPUSNTGOMMGY-MRVIYFEKSA-N etoposide Chemical compound COC1=C(O)C(OC)=CC([C@@H]2C3=CC=4OCOC=4C=C3[C@@H](O[C@H]3[C@@H]([C@@H](O)[C@@H]4O[C@H](C)OC[C@H]4O3)O)[C@@H]3[C@@H]2C(OC3)=O)=C1 VJJPUSNTGOMMGY-MRVIYFEKSA-N 0.000 description 1
• 229960005420 etoposide Drugs 0.000 description 1
• 230000029142 excretion Effects 0.000 description 1
• 238000000605 extraction Methods 0.000 description 1
• 210000003414 extremity Anatomy 0.000 description 1
• 235000013861 fat-free Nutrition 0.000 description 1
• MHMNJMPURVTYEJ-UHFFFAOYSA-N fluorescein-5-isothiocyanate Chemical compound O1C(=O)C2=CC(N=C=S)=CC=C2C21C1=CC=C(O)C=C1OC1=CC(O)=CC=C21 MHMNJMPURVTYEJ-UHFFFAOYSA-N 0.000 description 1
• 229910052731 fluorine Inorganic materials 0.000 description 1
• 239000011737 fluorine Substances 0.000 description 1
• 150000002222 fluorine compounds Chemical class 0.000 description 1
• 230000004907 flux Effects 0.000 description 1
• 229940014144 folate Drugs 0.000 description 1
• OVBPIULPVIDEAO-LBPRGKRZSA-N folic acid Chemical compound C=1N=C2NC(N)=NC(=O)C2=NC=1CNC1=CC=C(C(=O)N[C@@H](CCC(O)=O)C(O)=O)C=C1 OVBPIULPVIDEAO-LBPRGKRZSA-N 0.000 description 1
• 235000019152 folic acid Nutrition 0.000 description 1
• 239000011724 folic acid Substances 0.000 description 1
• 239000012458 free base Substances 0.000 description 1
• 229930182830 galactose Natural products 0.000 description 1
• 239000008273 gelatin Substances 0.000 description 1
• 229920000159 gelatin Polymers 0.000 description 1
• 239000007903 gelatin capsule Substances 0.000 description 1
• 235000019322 gelatine Nutrition 0.000 description 1
• 235000011852 gelatine desserts Nutrition 0.000 description 1
• 230000004034 genetic regulation Effects 0.000 description 1
• 201000009339 glycogen storage disease VII Diseases 0.000 description 1
• 150000002334 glycols Chemical class 0.000 description 1
• 229940029575 guanosine Drugs 0.000 description 1
• 238000010438 heat treatment Methods 0.000 description 1
• 238000010842 high-capacity cDNA reverse transcription kit Methods 0.000 description 1
• 230000003054 hormonal effect Effects 0.000 description 1
• 210000004408 hybridoma Anatomy 0.000 description 1
• 239000001257 hydrogen Substances 0.000 description 1
• 229910052739 hydrogen Inorganic materials 0.000 description 1
• XMBWDFGMSWQBCA-UHFFFAOYSA-N hydrogen iodide Chemical compound I XMBWDFGMSWQBCA-UHFFFAOYSA-N 0.000 description 1
• UWYVPFMHMJIBHE-OWOJBTEDSA-N hydroxymaleic acid group Chemical group O/C(/C(=O)O)=C/C(=O)O UWYVPFMHMJIBHE-OWOJBTEDSA-N 0.000 description 1
• 238000005213 imbibition Methods 0.000 description 1
• 150000002463 imidates Chemical class 0.000 description 1
• 230000036737 immune function Effects 0.000 description 1
• 210000000987 immune system Anatomy 0.000 description 1
• 229940072221 immunoglobulins Drugs 0.000 description 1
• 230000002637 immunotoxin Effects 0.000 description 1
• 239000002596 immunotoxin Substances 0.000 description 1
• 229940051026 immunotoxin Drugs 0.000 description 1
• 231100000608 immunotoxin Toxicity 0.000 description 1
• 230000003116 impacting effect Effects 0.000 description 1
• 238000007901 in situ hybridization Methods 0.000 description 1
• 238000000099 in vitro assay Methods 0.000 description 1
• 238000005462 in vivo assay Methods 0.000 description 1
• 238000011065 in-situ storage Methods 0.000 description 1
• 208000017745 inborn carbohydrate metabolic disease Diseases 0.000 description 1
• 208000033065 inborn errors of immunity Diseases 0.000 description 1
• 230000001939 inductive effect Effects 0.000 description 1
• 230000004968 inflammatory condition Effects 0.000 description 1
• 230000002757 inflammatory effect Effects 0.000 description 1
• ZPNFWUPYTFPOJU-LPYSRVMUSA-N iniprol Chemical compound C([C@H]1C(=O)NCC(=O)NCC(=O)N[C@H]2CSSC[C@H]3C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](C)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@H](C(N[C@H](C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CC=4C=CC(O)=CC=4)C(=O)N[C@@H](CC=4C=CC=CC=4)C(=O)N[C@@H](CC=4C=CC(O)=CC=4)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](C)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](C)C(=O)NCC(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CSSC[C@H](NC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](C)NC(=O)[C@H](CO)NC(=O)[C@H](CCCCN)NC(=O)[C@H](CC=4C=CC=CC=4)NC(=O)[C@H](CC(N)=O)NC(=O)[C@H](CC(N)=O)NC(=O)[C@H](CCCNC(N)=N)NC(=O)[C@H](CCCCN)NC(=O)[C@H](C)NC(=O)[C@H](CCCNC(N)=N)NC2=O)C(=O)N[C@@H](CCSC)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H](CSSC[C@H](NC(=O)[C@H](CC=2C=CC=CC=2)NC(=O)[C@H](CC(O)=O)NC(=O)[C@H]2N(CCC2)C(=O)[C@@H](N)CCCNC(N)=N)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCC(O)=O)C(=O)N2[C@@H](CCC2)C(=O)N2[C@@H](CCC2)C(=O)N[C@@H](CC=2C=CC(O)=CC=2)C(=O)N[C@@H]([C@@H](C)O)C(=O)NCC(=O)N2[C@@H](CCC2)C(=O)N3)C(=O)NCC(=O)NCC(=O)N[C@@H](C)C(O)=O)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@H](C(=O)N[C@@H](CC=2C=CC=CC=2)C(=O)N[C@H](C(=O)N1)C(C)C)[C@@H](C)O)[C@@H](C)CC)=O)[C@@H](C)CC)C1=CC=C(O)C=C1 ZPNFWUPYTFPOJU-LPYSRVMUSA-N 0.000 description 1
• CDAISMWEOUEBRE-GPIVLXJGSA-N inositol Chemical group O[C@H]1[C@H](O)[C@@H](O)[C@H](O)[C@H](O)[C@@H]1O CDAISMWEOUEBRE-GPIVLXJGSA-N 0.000 description 1
• 201000006913 intermediate spinal muscular atrophy Diseases 0.000 description 1
• 230000009878 intermolecular interaction Effects 0.000 description 1
• 210000001596 intra-abdominal fat Anatomy 0.000 description 1
• 238000001361 intraarterial administration Methods 0.000 description 1
• 238000000185 intracerebroventricular administration Methods 0.000 description 1
• 238000007917 intracranial administration Methods 0.000 description 1
• 238000007918 intramuscular administration Methods 0.000 description 1
• 238000007912 intraperitoneal administration Methods 0.000 description 1
• 238000007913 intrathecal administration Methods 0.000 description 1
• 238000001990 intravenous administration Methods 0.000 description 1
• 229950003599 ipsapirone Drugs 0.000 description 1
• 230000002427 irreversible effect Effects 0.000 description 1
• 239000012948 isocyanate Substances 0.000 description 1
• 229960002725 isoflurane Drugs 0.000 description 1
• 239000007951 isotonicity adjuster Substances 0.000 description 1
• 238000005304 joining Methods 0.000 description 1
• 239000003350 kerosene Substances 0.000 description 1
• 210000003292 kidney cell Anatomy 0.000 description 1
• 210000003127 knee Anatomy 0.000 description 1
• 239000008101 lactose Substances 0.000 description 1
• 239000000787 lecithin Substances 0.000 description 1
• 229940067606 lecithin Drugs 0.000 description 1
• 235000010445 lecithin Nutrition 0.000 description 1
• 229960004194 lidocaine Drugs 0.000 description 1
• 238000007834 ligase chain reaction Methods 0.000 description 1
• 210000005229 liver cell Anatomy 0.000 description 1
• 244000144972 livestock Species 0.000 description 1
• 230000033001 locomotion Effects 0.000 description 1
• 229960002247 lomustine Drugs 0.000 description 1
• 210000002751 lymph Anatomy 0.000 description 1
• 239000006166 lysate Substances 0.000 description 1
• 230000002132 lysosomal effect Effects 0.000 description 1
• 108010026228 mRNA guanylyltransferase Proteins 0.000 description 1
• 235000019359 magnesium stearate Nutrition 0.000 description 1
• 230000005415 magnetization Effects 0.000 description 1
• 230000014759 maintenance of location Effects 0.000 description 1
• 239000000594 mannitol Substances 0.000 description 1
• 235000010355 mannitol Nutrition 0.000 description 1
• 150000008146 mannosides Chemical class 0.000 description 1
• 239000003550 marker Substances 0.000 description 1
• 235000013372 meat Nutrition 0.000 description 1
• 230000007246 mechanism Effects 0.000 description 1
• 229960004961 mechlorethamine Drugs 0.000 description 1
• HAWPXGHAZFHHAD-UHFFFAOYSA-N mechlorethamine Chemical compound ClCCN(C)CCCl HAWPXGHAZFHHAD-UHFFFAOYSA-N 0.000 description 1
• 238000002483 medication Methods 0.000 description 1
• 229960001924 melphalan Drugs 0.000 description 1
• SGDBTWWWUNNDEQ-LBPRGKRZSA-N melphalan Chemical compound OC(=O)[C@@H](N)CC1=CC=C(N(CCCl)CCCl)C=C1 SGDBTWWWUNNDEQ-LBPRGKRZSA-N 0.000 description 1
• 229960001428 mercaptopurine Drugs 0.000 description 1
• CPHJTSJQUQZOLJ-ISIDMKFXSA-N mespirenone Chemical compound C([C@]12[C@H]3C[C@H]3[C@@H]3[C@]1(C)CC[C@@H]1[C@@]4(C)C=CC(=O)C=C4C[C@H]([C@@H]31)SC(=O)C)CC(=O)O2 CPHJTSJQUQZOLJ-ISIDMKFXSA-N 0.000 description 1
• 229950008481 mespirenone Drugs 0.000 description 1
• 230000002503 metabolic effect Effects 0.000 description 1
• 239000002207 metabolite Substances 0.000 description 1
• 229960000485 methotrexate Drugs 0.000 description 1
• 229920000609 methyl cellulose Polymers 0.000 description 1
• 150000004702 methyl esters Chemical class 0.000 description 1
• 239000001923 methylcellulose Substances 0.000 description 1
• 235000010981 methylcellulose Nutrition 0.000 description 1
• 229960002900 methylcellulose Drugs 0.000 description 1
• 229960002704 metipranolol Drugs 0.000 description 1
• BLWNYSZZZWQCKO-UHFFFAOYSA-N metipranolol hydrochloride Chemical compound [Cl-].CC(C)[NH2+]CC(O)COC1=CC(C)=C(OC(C)=O)C(C)=C1C BLWNYSZZZWQCKO-UHFFFAOYSA-N 0.000 description 1
• 229960002237 metoprolol Drugs 0.000 description 1
• IUBSYMUCCVWXPE-UHFFFAOYSA-N metoprolol Chemical compound COCCC1=CC=C(OCC(O)CNC(C)C)C=C1 IUBSYMUCCVWXPE-UHFFFAOYSA-N 0.000 description 1
• 108091070501 miRNA Proteins 0.000 description 1
• 235000013336 milk Nutrition 0.000 description 1
• 239000008267 milk Substances 0.000 description 1
• 210000004080 milk Anatomy 0.000 description 1
• 230000003278 mimic effect Effects 0.000 description 1
• 229960005485 mitobronitol Drugs 0.000 description 1
• 210000003470 mitochondria Anatomy 0.000 description 1
• KKZJGLLVHKMTCM-UHFFFAOYSA-N mitoxantrone Chemical compound O=C1C2=C(O)C=CC(O)=C2C(=O)C2=C1C(NCCNCCO)=CC=C2NCCNCCO KKZJGLLVHKMTCM-UHFFFAOYSA-N 0.000 description 1
• 229960001156 mitoxantrone Drugs 0.000 description 1
• 238000002156 mixing Methods 0.000 description 1
• 238000009126 molecular therapy Methods 0.000 description 1
• 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 description 1
• 210000002161 motor neuron Anatomy 0.000 description 1
• 208000005264 motor neuron disease Diseases 0.000 description 1
• 210000000214 mouth Anatomy 0.000 description 1
• 229960003938 moxonidine Drugs 0.000 description 1
• 210000000663 muscle cell Anatomy 0.000 description 1
• 210000003365 myofibril Anatomy 0.000 description 1
• 108010059725 myosin-binding protein C Proteins 0.000 description 1
• 229960004255 nadolol Drugs 0.000 description 1
• VWPOSFSPZNDTMJ-UCWKZMIHSA-N nadolol Chemical compound C1[C@@H](O)[C@@H](O)CC2=C1C=CC=C2OCC(O)CNC(C)(C)C VWPOSFSPZNDTMJ-UCWKZMIHSA-N 0.000 description 1
• 229930014626 natural product Natural products 0.000 description 1
• 229960000619 nebivolol Drugs 0.000 description 1
• 210000000944 nerve tissue Anatomy 0.000 description 1
• 208000018360 neuromuscular disease Diseases 0.000 description 1
• 230000003472 neutralizing effect Effects 0.000 description 1
• 229920001220 nitrocellulos Polymers 0.000 description 1
• 239000000041 non-steroidal anti-inflammatory agent Substances 0.000 description 1
• 229940021182 non-steroidal anti-inflammatory drug Drugs 0.000 description 1
• 239000012457 nonaqueous media Substances 0.000 description 1
• 150000003833 nucleoside derivatives Chemical class 0.000 description 1
• 239000003921 oil Substances 0.000 description 1
• 235000019198 oils Nutrition 0.000 description 1
• 238000002966 oligonucleotide array Methods 0.000 description 1
• 239000004006 olive oil Substances 0.000 description 1
• 235000008390 olive oil Nutrition 0.000 description 1
• 238000001543 one-way ANOVA Methods 0.000 description 1
• 150000002894 organic compounds Chemical class 0.000 description 1
• 150000002895 organic esters Chemical class 0.000 description 1
• 230000010355 oscillation Effects 0.000 description 1
• QUANRIQJNFHVEU-UHFFFAOYSA-N oxirane;propane-1,2,3-triol Chemical compound C1CO1.OCC(O)CO QUANRIQJNFHVEU-UHFFFAOYSA-N 0.000 description 1
• 229960004570 oxprenolol Drugs 0.000 description 1
• 229960001592 paclitaxel Drugs 0.000 description 1
• 210000000496 pancreas Anatomy 0.000 description 1
• 230000001575 pathological effect Effects 0.000 description 1
• 230000007170 pathology Effects 0.000 description 1
• 239000013610 patient sample Substances 0.000 description 1
• 229960002035 penbutolol Drugs 0.000 description 1
• KQXKVJAGOJTNJS-HNNXBMFYSA-N penbutolol Chemical compound CC(C)(C)NC[C@H](O)COC1=CC=CC=C1C1CCCC1 KQXKVJAGOJTNJS-HNNXBMFYSA-N 0.000 description 1
• XDRYMKDFEDOLFX-UHFFFAOYSA-N pentamidine Chemical compound C1=CC(C(=N)N)=CC=C1OCCCCCOC1=CC=C(C(N)=N)C=C1 XDRYMKDFEDOLFX-UHFFFAOYSA-N 0.000 description 1
• 229960004448 pentamidine Drugs 0.000 description 1
• 229960003330 pentetic acid Drugs 0.000 description 1
• 239000000137 peptide hydrolase inhibitor Substances 0.000 description 1
• 239000002304 perfume Substances 0.000 description 1
• 208000027232 peripheral nervous system disease Diseases 0.000 description 1
• 108040007629 peroxidase activity proteins Proteins 0.000 description 1
• 108010076042 phenomycin Proteins 0.000 description 1
• WLJVXDMOQOGPHL-UHFFFAOYSA-N phenylacetic acid Chemical compound OC(=O)CC1=CC=CC=C1 WLJVXDMOQOGPHL-UHFFFAOYSA-N 0.000 description 1
• 230000037081 physical activity Effects 0.000 description 1
• 239000006187 pill Substances 0.000 description 1
• 229960002508 pindolol Drugs 0.000 description 1
• PHUTUTUABXHXLW-UHFFFAOYSA-N pindolol Chemical compound CC(C)NCC(O)COC1=CC=CC2=NC=C[C]12 PHUTUTUABXHXLW-UHFFFAOYSA-N 0.000 description 1
• BASFCYQUMIYNBI-UHFFFAOYSA-N platinum Substances [Pt] BASFCYQUMIYNBI-UHFFFAOYSA-N 0.000 description 1
• 239000008389 polyethoxylated castor oil Substances 0.000 description 1
• 229920000573 polyethylene Polymers 0.000 description 1
• 208000005987 polymyositis Diseases 0.000 description 1
• 229920005862 polyol Polymers 0.000 description 1
• 150000003077 polyols Chemical class 0.000 description 1
• 210000004896 polypeptide structure Anatomy 0.000 description 1
• 230000008092 positive effect Effects 0.000 description 1
• 238000010149 post-hoc-test Methods 0.000 description 1
• 230000003389 potentiating effect Effects 0.000 description 1
• 239000000843 powder Substances 0.000 description 1
• 229960001289 prazosin Drugs 0.000 description 1
• IENZQIKPVFGBNW-UHFFFAOYSA-N prazosin Chemical compound N=1C(N)=C2C=C(OC)C(OC)=CC2=NC=1N(CC1)CCN1C(=O)C1=CC=CO1 IENZQIKPVFGBNW-UHFFFAOYSA-N 0.000 description 1
• 239000003755 preservative agent Substances 0.000 description 1
• 208000028529 primary immunodeficiency disease Diseases 0.000 description 1
• 239000013615 primer Substances 0.000 description 1
• 229960004919 procaine Drugs 0.000 description 1
• MFDFERRIHVXMIY-UHFFFAOYSA-N procaine Chemical compound CCN(CC)CCOC(=O)C1=CC=C(N)C=C1 MFDFERRIHVXMIY-UHFFFAOYSA-N 0.000 description 1
• 230000000750 progressive effect Effects 0.000 description 1
• 230000002035 prolonged effect Effects 0.000 description 1
• 230000001737 promoting effect Effects 0.000 description 1
• 230000000069 prophylactic effect Effects 0.000 description 1
• 238000011321 prophylaxis Methods 0.000 description 1
• 229940048914 protamine Drugs 0.000 description 1
• 238000000159 protein binding assay Methods 0.000 description 1
• 229960000856 protein c Drugs 0.000 description 1
• 108020001580 protein domains Proteins 0.000 description 1
• 230000009145 protein modification Effects 0.000 description 1
• 239000000007 protein synthesis inhibitor Substances 0.000 description 1
• 108020000494 protein-tyrosine phosphatase Proteins 0.000 description 1
• 230000004850 protein–protein interaction Effects 0.000 description 1
• 230000002797 proteolythic effect Effects 0.000 description 1
• 230000004063 proteosomal degradation Effects 0.000 description 1
• 238000000746 purification Methods 0.000 description 1
• 229950010131 puromycin Drugs 0.000 description 1
• 238000003908 quality control method Methods 0.000 description 1
• 150000003242 quaternary ammonium salts Chemical class 0.000 description 1
• 238000011897 real-time detection Methods 0.000 description 1
• 102000027426 receptor tyrosine kinases Human genes 0.000 description 1
• 108091008598 receptor tyrosine kinases Proteins 0.000 description 1
• 238000010188 recombinant method Methods 0.000 description 1
• 230000008929 regeneration Effects 0.000 description 1
• 238000011069 regeneration method Methods 0.000 description 1
• 230000008439 repair process Effects 0.000 description 1
• 230000003252 repetitive effect Effects 0.000 description 1
• 230000010076 replication Effects 0.000 description 1
• 230000000717 retained effect Effects 0.000 description 1
• 230000002441 reversible effect Effects 0.000 description 1
• 125000002652 ribonucleotide group Chemical group 0.000 description 1
• 238000002702 ribosome display Methods 0.000 description 1
• 229960000764 rilmenidine Drugs 0.000 description 1
• 238000005096 rolling process Methods 0.000 description 1
• 238000013391 scatchard analysis Methods 0.000 description 1
• 230000007017 scission Effects 0.000 description 1
• 208000008864 scrapie Diseases 0.000 description 1
• 238000001338 self-assembly Methods 0.000 description 1
• 230000035945 sensitivity Effects 0.000 description 1
• 210000002460 smooth muscle Anatomy 0.000 description 1
• 235000019812 sodium carboxymethyl cellulose Nutrition 0.000 description 1
• 229920001027 sodium carboxymethylcellulose Polymers 0.000 description 1
• 238000002415 sodium dodecyl sulfate polyacrylamide gel electrophoresis Methods 0.000 description 1
• 238000002764 solid phase assay Methods 0.000 description 1
• 239000004334 sorbic acid Substances 0.000 description 1
• 229940075582 sorbic acid Drugs 0.000 description 1
• 235000010199 sorbic acid Nutrition 0.000 description 1
• 239000000600 sorbitol Substances 0.000 description 1
• 229960002370 sotalol Drugs 0.000 description 1
• ZBMZVLHSJCTVON-UHFFFAOYSA-N sotalol Chemical compound CC(C)NCC(O)C1=CC=C(NS(C)(=O)=O)C=C1 ZBMZVLHSJCTVON-UHFFFAOYSA-N 0.000 description 1
• 238000004611 spectroscopical analysis Methods 0.000 description 1
• LXMSZDCAJNLERA-ZHYRCANASA-N spironolactone Chemical compound C([C@@H]1[C@]2(C)CC[C@@H]3[C@@]4(C)CCC(=O)C=C4C[C@H]([C@@H]13)SC(=O)C)C[C@@]21CCC(=O)O1 LXMSZDCAJNLERA-ZHYRCANASA-N 0.000 description 1
• 229960002256 spironolactone Drugs 0.000 description 1
• 210000003802 sputum Anatomy 0.000 description 1
• 208000024794 sputum Diseases 0.000 description 1
• 239000008107 starch Substances 0.000 description 1
• 235000019698 starch Nutrition 0.000 description 1
• 239000007858 starting material Substances 0.000 description 1
• 239000002294 steroidal antiinflammatory agent Substances 0.000 description 1
• 230000003637 steroidlike Effects 0.000 description 1
• 238000003860 storage Methods 0.000 description 1
• ZSJLQEPLLKMAKR-GKHCUFPYSA-N streptozocin Chemical compound O=NN(C)C(=O)N[C@H]1[C@@H](O)O[C@H](CO)[C@@H](O)[C@@H]1O ZSJLQEPLLKMAKR-GKHCUFPYSA-N 0.000 description 1
• 229960001052 streptozocin Drugs 0.000 description 1
• 238000007920 subcutaneous administration Methods 0.000 description 1
• 239000005720 sucrose Substances 0.000 description 1
• 150000005846 sugar alcohols Polymers 0.000 description 1
• 150000008163 sugars Chemical class 0.000 description 1
• 239000013589 supplement Substances 0.000 description 1
• 230000001629 suppression Effects 0.000 description 1
• 238000002198 surface plasmon resonance spectroscopy Methods 0.000 description 1
• 239000004094 surface-active agent Substances 0.000 description 1
• 108010033090 surfactant protein A receptor Proteins 0.000 description 1
• 239000000725 suspension Substances 0.000 description 1
• 230000002459 sustained effect Effects 0.000 description 1
• 235000020357 syrup Nutrition 0.000 description 1
• 239000006188 syrup Substances 0.000 description 1
• 238000007910 systemic administration Methods 0.000 description 1
• 238000012385 systemic delivery Methods 0.000 description 1
• 229940037128 systemic glucocorticoids Drugs 0.000 description 1
• 239000000454 talc Substances 0.000 description 1
• 229910052623 talc Inorganic materials 0.000 description 1
• 230000008685 targeting Effects 0.000 description 1
• RCINICONZNJXQF-MZXODVADSA-N taxol Chemical compound O([C@@H]1[C@@]2(C[C@@H](C(C)=C(C2(C)C)[C@H](C([C@]2(C)[C@@H](O)C[C@H]3OC[C@]3([C@H]21)OC(C)=O)=O)OC(=O)C)OC(=O)[C@H](O)[C@@H](NC(=O)C=1C=CC=CC=1)C=1C=CC=CC=1)O)C(=O)C1=CC=CC=C1 RCINICONZNJXQF-MZXODVADSA-N 0.000 description 1
• 210000001138 tear Anatomy 0.000 description 1
• NRUKOCRGYNPUPR-QBPJDGROSA-N teniposide Chemical compound COC1=C(O)C(OC)=CC([C@@H]2C3=CC=4OCOC=4C=C3[C@@H](O[C@H]3[C@@H]([C@@H](O)[C@@H]4O[C@@H](OC[C@H]4O3)C=3SC=CC=3)O)[C@@H]3[C@@H]2C(OC3)=O)=C1 NRUKOCRGYNPUPR-QBPJDGROSA-N 0.000 description 1
• 229960001278 teniposide Drugs 0.000 description 1
• VCKUSRYTPJJLNI-UHFFFAOYSA-N terazosin Chemical compound N=1C(N)=C2C=C(OC)C(OC)=CC2=NC=1N(CC1)CCN1C(=O)C1CCCO1 VCKUSRYTPJJLNI-UHFFFAOYSA-N 0.000 description 1
• 229960001693 terazosin Drugs 0.000 description 1
• BPEWUONYVDABNZ-DZBHQSCQSA-N testolactone Chemical compound O=C1C=C[C@]2(C)[C@H]3CC[C@](C)(OC(=O)CC4)[C@@H]4[C@@H]3CCC2=C1 BPEWUONYVDABNZ-DZBHQSCQSA-N 0.000 description 1
• 229960005353 testolactone Drugs 0.000 description 1
• 229960002372 tetracaine Drugs 0.000 description 1
• GKCBAIGFKIBETG-UHFFFAOYSA-N tetracaine Chemical compound CCCCNC1=CC=C(C(=O)OCCN(C)C)C=C1 GKCBAIGFKIBETG-UHFFFAOYSA-N 0.000 description 1
• 239000000892 thaumatin Substances 0.000 description 1
• 235000010436 thaumatin Nutrition 0.000 description 1
• 229940124597 therapeutic agent Drugs 0.000 description 1
• CNHYKKNIIGEXAY-UHFFFAOYSA-N thiolan-2-imine Chemical compound N=C1CCCS1 CNHYKKNIIGEXAY-UHFFFAOYSA-N 0.000 description 1
• ZEMGGZBWXRYJHK-UHFFFAOYSA-N thiouracil Chemical compound O=C1C=CNC(=S)N1 ZEMGGZBWXRYJHK-UHFFFAOYSA-N 0.000 description 1
• 229940104230 thymidine Drugs 0.000 description 1
• 230000036962 time dependent Effects 0.000 description 1
• 229960004605 timolol Drugs 0.000 description 1
• 229960003087 tioguanine Drugs 0.000 description 1
• 238000004448 titration Methods 0.000 description 1
• RUELTTOHQODFPA-UHFFFAOYSA-N toluene 2,6-diisocyanate Chemical compound CC1=C(N=C=O)C=CC=C1N=C=O RUELTTOHQODFPA-UHFFFAOYSA-N 0.000 description 1
• 230000000699 topical effect Effects 0.000 description 1
• 231100000331 toxic Toxicity 0.000 description 1
• 230000002588 toxic effect Effects 0.000 description 1
• 230000001131 transforming effect Effects 0.000 description 1
• 230000014621 translational initiation Effects 0.000 description 1
• 230000000472 traumatic effect Effects 0.000 description 1
• QORWJWZARLRLPR-UHFFFAOYSA-H tricalcium bis(phosphate) Chemical compound [Ca+2].[Ca+2].[Ca+2].[O-]P([O-])([O-])=O.[O-]P([O-])([O-])=O QORWJWZARLRLPR-UHFFFAOYSA-H 0.000 description 1
• 229930013292 trichothecene Natural products 0.000 description 1
• 150000003327 trichothecene derivatives Chemical class 0.000 description 1
• UFTFJSFQGQCHQW-UHFFFAOYSA-N triformin Chemical compound O=COCC(OC=O)COC=O UFTFJSFQGQCHQW-UHFFFAOYSA-N 0.000 description 1
• PIEPQKCYPFFYMG-UHFFFAOYSA-N tris acetate Chemical compound CC(O)=O.OCC(N)(CO)CO PIEPQKCYPFFYMG-UHFFFAOYSA-N 0.000 description 1
• 208000005606 type IV spinal muscular atrophy Diseases 0.000 description 1
• 241000701447 unidentified baculovirus Species 0.000 description 1
• 241001430294 unidentified retrovirus Species 0.000 description 1
• 238000009827 uniform distribution Methods 0.000 description 1
• DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 description 1
• 229940045145 uridine Drugs 0.000 description 1
• 210000002700 urine Anatomy 0.000 description 1
• 238000010200 validation analysis Methods 0.000 description 1
• 235000013311 vegetables Nutrition 0.000 description 1
• 239000013603 viral vector Substances 0.000 description 1
• 230000003612 virological effect Effects 0.000 description 1
• 239000011782 vitamin Substances 0.000 description 1
• 229940088594 vitamin Drugs 0.000 description 1
• 235000013343 vitamin Nutrition 0.000 description 1
• 229930003231 vitamin Natural products 0.000 description 1
• 230000003442 weekly effect Effects 0.000 description 1
• 239000000080 wetting agent Substances 0.000 description 1
• 238000002424 x-ray crystallography Methods 0.000 description 1
• 229940075420 xanthine Drugs 0.000 description 1