CA3042197A1 - Synp198, a promoter for the specific expression of genes in direction selective retinal ganglion cells - Google Patents
Synp198, a promoter for the specific expression of genes in direction selective retinal ganglion cells Download PDFInfo
- Publication number
- CA3042197A1 CA3042197A1 CA3042197A CA3042197A CA3042197A1 CA 3042197 A1 CA3042197 A1 CA 3042197A1 CA 3042197 A CA3042197 A CA 3042197A CA 3042197 A CA3042197 A CA 3042197A CA 3042197 A1 CA3042197 A1 CA 3042197A1
- Authority
- CA
- Canada
- Prior art keywords
- nucleic acid
- gene
- sequence
- cell
- acid sequence
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Abandoned
Links
- 108090000623 proteins and genes Proteins 0.000 title claims abstract description 69
- 230000014509 gene expression Effects 0.000 title claims abstract description 55
- 210000003994 retinal ganglion cell Anatomy 0.000 title claims abstract description 14
- 150000007523 nucleic acids Chemical class 0.000 claims abstract description 90
- 108091028043 Nucleic acid sequence Proteins 0.000 claims abstract description 62
- 102000039446 nucleic acids Human genes 0.000 claims abstract description 35
- 108020004707 nucleic acids Proteins 0.000 claims abstract description 35
- 210000004027 cell Anatomy 0.000 claims description 98
- 208000003098 Ganglion Cysts Diseases 0.000 claims description 34
- 238000000034 method Methods 0.000 claims description 30
- 230000002207 retinal effect Effects 0.000 claims description 28
- 239000013598 vector Substances 0.000 claims description 24
- 108010050754 Halorhodopsins Proteins 0.000 claims description 13
- 208000005400 Synovial Cyst Diseases 0.000 claims description 12
- 108010035848 Channelrhodopsins Proteins 0.000 claims description 7
- 239000013603 viral vector Substances 0.000 claims description 5
- 230000001737 promoting effect Effects 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 description 34
- 102000004196 processed proteins & peptides Human genes 0.000 description 32
- 229920001184 polypeptide Polymers 0.000 description 31
- NCYCYZXNIZJOKI-UHFFFAOYSA-N vitamin A aldehyde Natural products O=CC=C(C)C=CC=C(C)C=CC1=C(C)CCCC1(C)C NCYCYZXNIZJOKI-UHFFFAOYSA-N 0.000 description 28
- 108020004414 DNA Proteins 0.000 description 25
- 210000001525 retina Anatomy 0.000 description 22
- 241001465754 Metazoa Species 0.000 description 17
- 239000000203 mixture Substances 0.000 description 16
- 241000282414 Homo sapiens Species 0.000 description 14
- 102000040430 polynucleotide Human genes 0.000 description 14
- 108091033319 polynucleotide Proteins 0.000 description 14
- 239000002157 polynucleotide Substances 0.000 description 14
- 241000700605 Viruses Species 0.000 description 12
- 125000003275 alpha amino acid group Chemical group 0.000 description 12
- 102000004169 proteins and genes Human genes 0.000 description 12
- 239000005090 green fluorescent protein Substances 0.000 description 11
- 235000018102 proteins Nutrition 0.000 description 11
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 10
- 150000001875 compounds Chemical class 0.000 description 10
- 239000000243 solution Substances 0.000 description 10
- 108700019146 Transgenes Proteins 0.000 description 9
- 235000001014 amino acid Nutrition 0.000 description 9
- 150000001413 amino acids Chemical class 0.000 description 9
- 230000002068 genetic effect Effects 0.000 description 9
- 238000002360 preparation method Methods 0.000 description 9
- ZHNUHDYFZUAESO-UHFFFAOYSA-N Formamide Chemical compound NC=O ZHNUHDYFZUAESO-UHFFFAOYSA-N 0.000 description 8
- 230000004075 alteration Effects 0.000 description 8
- 230000004048 modification Effects 0.000 description 8
- 238000012986 modification Methods 0.000 description 8
- 239000003795 chemical substances by application Substances 0.000 description 7
- 238000012217 deletion Methods 0.000 description 7
- 230000037430 deletion Effects 0.000 description 7
- 230000000694 effects Effects 0.000 description 7
- 210000002569 neuron Anatomy 0.000 description 7
- 230000003612 virological effect Effects 0.000 description 7
- 208000002267 Anti-neutrophil cytoplasmic antibody-associated vasculitis Diseases 0.000 description 6
- 230000015572 biosynthetic process Effects 0.000 description 6
- 230000001413 cellular effect Effects 0.000 description 6
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 6
- 238000002347 injection Methods 0.000 description 6
- 239000007924 injection Substances 0.000 description 6
- 230000033001 locomotion Effects 0.000 description 6
- 230000001105 regulatory effect Effects 0.000 description 6
- 241000894007 species Species 0.000 description 6
- 210000001519 tissue Anatomy 0.000 description 6
- 230000000007 visual effect Effects 0.000 description 6
- 241000702421 Dependoparvovirus Species 0.000 description 5
- 108091023040 Transcription factor Proteins 0.000 description 5
- 102000040945 Transcription factor Human genes 0.000 description 5
- 241000251539 Vertebrata <Metazoa> Species 0.000 description 5
- 230000000903 blocking effect Effects 0.000 description 5
- 239000003153 chemical reaction reagent Substances 0.000 description 5
- 208000035475 disorder Diseases 0.000 description 5
- 108010048367 enhanced green fluorescent protein Proteins 0.000 description 5
- 238000009396 hybridization Methods 0.000 description 5
- 239000000463 material Substances 0.000 description 5
- 239000012528 membrane Substances 0.000 description 5
- 125000003729 nucleotide group Chemical group 0.000 description 5
- 239000002953 phosphate buffered saline Substances 0.000 description 5
- 230000008569 process Effects 0.000 description 5
- -1 silencers Substances 0.000 description 5
- 210000000225 synapse Anatomy 0.000 description 5
- 238000012360 testing method Methods 0.000 description 5
- 238000013518 transcription Methods 0.000 description 5
- 230000035897 transcription Effects 0.000 description 5
- 108091026890 Coding region Proteins 0.000 description 4
- 102000053602 DNA Human genes 0.000 description 4
- 210000004899 c-terminal region Anatomy 0.000 description 4
- 210000000349 chromosome Anatomy 0.000 description 4
- 238000004590 computer program Methods 0.000 description 4
- 238000001514 detection method Methods 0.000 description 4
- 230000005284 excitation Effects 0.000 description 4
- 238000009472 formulation Methods 0.000 description 4
- 210000004602 germ cell Anatomy 0.000 description 4
- 238000000338 in vitro Methods 0.000 description 4
- 230000003834 intracellular effect Effects 0.000 description 4
- 230000035772 mutation Effects 0.000 description 4
- 239000002773 nucleotide Substances 0.000 description 4
- 238000002864 sequence alignment Methods 0.000 description 4
- 230000003363 transsynaptic effect Effects 0.000 description 4
- 238000011144 upstream manufacturing Methods 0.000 description 4
- 241000242764 Aequorea victoria Species 0.000 description 3
- 241000972773 Aulopiformes Species 0.000 description 3
- 108090000862 Ion Channels Proteins 0.000 description 3
- 102000004310 Ion Channels Human genes 0.000 description 3
- 206010028980 Neoplasm Diseases 0.000 description 3
- 108020004511 Recombinant DNA Proteins 0.000 description 3
- DBMJMQXJHONAFJ-UHFFFAOYSA-M Sodium laurylsulphate Chemical compound [Na+].CCCCCCCCCCCCOS([O-])(=O)=O DBMJMQXJHONAFJ-UHFFFAOYSA-M 0.000 description 3
- 241000701093 Suid alphaherpesvirus 1 Species 0.000 description 3
- 108700009124 Transcription Initiation Site Proteins 0.000 description 3
- 239000004480 active ingredient Substances 0.000 description 3
- 230000004071 biological effect Effects 0.000 description 3
- 239000002299 complementary DNA Substances 0.000 description 3
- 238000012937 correction Methods 0.000 description 3
- 125000004122 cyclic group Chemical group 0.000 description 3
- 210000001787 dendrite Anatomy 0.000 description 3
- 238000009826 distribution Methods 0.000 description 3
- 239000003814 drug Substances 0.000 description 3
- 239000000839 emulsion Substances 0.000 description 3
- 239000003623 enhancer Substances 0.000 description 3
- 239000012634 fragment Substances 0.000 description 3
- 238000002873 global sequence alignment Methods 0.000 description 3
- 108020004999 messenger RNA Proteins 0.000 description 3
- 238000013508 migration Methods 0.000 description 3
- 230000005012 migration Effects 0.000 description 3
- 239000003068 molecular probe Substances 0.000 description 3
- 230000003287 optical effect Effects 0.000 description 3
- 108091008695 photoreceptors Proteins 0.000 description 3
- 235000019515 salmon Nutrition 0.000 description 3
- 150000003839 salts Chemical class 0.000 description 3
- 235000019333 sodium laurylsulphate Nutrition 0.000 description 3
- 238000001228 spectrum Methods 0.000 description 3
- 239000000725 suspension Substances 0.000 description 3
- 239000003981 vehicle Substances 0.000 description 3
- 108700028369 Alleles Proteins 0.000 description 2
- 241000272517 Anseriformes Species 0.000 description 2
- 108010082845 Bacteriorhodopsins Proteins 0.000 description 2
- 241000283690 Bos taurus Species 0.000 description 2
- 108010058699 Choline O-acetyltransferase Proteins 0.000 description 2
- 102100023460 Choline O-acetyltransferase Human genes 0.000 description 2
- 241000701022 Cytomegalovirus Species 0.000 description 2
- 241000283074 Equus asinus Species 0.000 description 2
- 108700028146 Genetic Enhancer Elements Proteins 0.000 description 2
- WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Chemical compound OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 description 2
- 108010043121 Green Fluorescent Proteins Proteins 0.000 description 2
- 102000004144 Green Fluorescent Proteins Human genes 0.000 description 2
- 241001074968 Halobacteria Species 0.000 description 2
- 108091092195 Intron Proteins 0.000 description 2
- 241000713666 Lentivirus Species 0.000 description 2
- 241000699666 Mus <mouse, genus> Species 0.000 description 2
- 241000699670 Mus sp. Species 0.000 description 2
- 102000010175 Opsin Human genes 0.000 description 2
- 108050001704 Opsin Proteins 0.000 description 2
- 241000283973 Oryctolagus cuniculus Species 0.000 description 2
- 241000700159 Rattus Species 0.000 description 2
- 241000242743 Renilla reniformis Species 0.000 description 2
- 241000714474 Rous sarcoma virus Species 0.000 description 2
- VYPSYNLAJGMNEJ-UHFFFAOYSA-N Silicium dioxide Chemical compound O=[Si]=O VYPSYNLAJGMNEJ-UHFFFAOYSA-N 0.000 description 2
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 2
- 208000037065 Subacute sclerosing leukoencephalitis Diseases 0.000 description 2
- 206010042297 Subacute sclerosing panencephalitis Diseases 0.000 description 2
- 239000013504 Triton X-100 Substances 0.000 description 2
- 229920004890 Triton X-100 Polymers 0.000 description 2
- 230000004913 activation Effects 0.000 description 2
- 210000000411 amacrine cell Anatomy 0.000 description 2
- 210000003484 anatomy Anatomy 0.000 description 2
- 210000002459 blastocyst Anatomy 0.000 description 2
- 210000004556 brain Anatomy 0.000 description 2
- 210000000234 capsid Anatomy 0.000 description 2
- 125000003178 carboxy group Chemical group [H]OC(*)=O 0.000 description 2
- 238000003776 cleavage reaction Methods 0.000 description 2
- 210000004087 cornea Anatomy 0.000 description 2
- 230000001419 dependent effect Effects 0.000 description 2
- 239000002552 dosage form Substances 0.000 description 2
- 239000003937 drug carrier Substances 0.000 description 2
- 238000010291 electrical method Methods 0.000 description 2
- 238000005755 formation reaction Methods 0.000 description 2
- 238000001727 in vivo Methods 0.000 description 2
- 238000011534 incubation Methods 0.000 description 2
- 230000000977 initiatory effect Effects 0.000 description 2
- 239000012212 insulator Substances 0.000 description 2
- 238000005304 joining Methods 0.000 description 2
- 150000002632 lipids Chemical class 0.000 description 2
- 239000007788 liquid Substances 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
- 239000003550 marker Substances 0.000 description 2
- 239000011159 matrix material Substances 0.000 description 2
- 230000001404 mediated effect Effects 0.000 description 2
- 239000002245 particle Substances 0.000 description 2
- 239000000825 pharmaceutical preparation Substances 0.000 description 2
- 230000035479 physiological effects, processes and functions Effects 0.000 description 2
- 230000008488 polyadenylation Effects 0.000 description 2
- 230000001242 postsynaptic effect Effects 0.000 description 2
- 230000001323 posttranslational effect Effects 0.000 description 2
- 239000003755 preservative agent Substances 0.000 description 2
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 description 2
- 238000012545 processing Methods 0.000 description 2
- 238000011160 research Methods 0.000 description 2
- 230000007017 scission Effects 0.000 description 2
- 238000012216 screening Methods 0.000 description 2
- 239000011780 sodium chloride Substances 0.000 description 2
- 229910000162 sodium phosphate Inorganic materials 0.000 description 2
- 239000000375 suspending agent Substances 0.000 description 2
- 238000003786 synthesis reaction Methods 0.000 description 2
- 229940124597 therapeutic agent Drugs 0.000 description 2
- 238000001890 transfection Methods 0.000 description 2
- 238000010798 ubiquitination Methods 0.000 description 2
- 230000034512 ubiquitination Effects 0.000 description 2
- 238000005406 washing Methods 0.000 description 2
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 2
- 108091005957 yellow fluorescent proteins Proteins 0.000 description 2
- 102000040650 (ribonucleotides)n+m Human genes 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
- FWBHETKCLVMNFS-UHFFFAOYSA-N 4',6-Diamino-2-phenylindol Chemical compound C1=CC(C(=N)N)=CC=C1C1=CC2=CC=C(C(N)=N)C=C2N1 FWBHETKCLVMNFS-UHFFFAOYSA-N 0.000 description 1
- ODHCTXKNWHHXJC-VKHMYHEASA-N 5-oxo-L-proline Chemical compound OC(=O)[C@@H]1CCC(=O)N1 ODHCTXKNWHHXJC-VKHMYHEASA-N 0.000 description 1
- 230000005730 ADP ribosylation Effects 0.000 description 1
- 102000007469 Actins Human genes 0.000 description 1
- 108010085238 Actins Proteins 0.000 description 1
- 241000243290 Aequorea Species 0.000 description 1
- 241000426851 Aequorea aequorea Species 0.000 description 1
- 229920001817 Agar Polymers 0.000 description 1
- 206010001497 Agitation Diseases 0.000 description 1
- 239000012103 Alexa Fluor 488 Substances 0.000 description 1
- 239000012112 Alexa Fluor 633 Substances 0.000 description 1
- 235000019489 Almond oil Nutrition 0.000 description 1
- 241000203069 Archaea Species 0.000 description 1
- 108050003620 Arrestin-C Proteins 0.000 description 1
- 102100026440 Arrestin-C Human genes 0.000 description 1
- 241000894006 Bacteria Species 0.000 description 1
- 125000001433 C-terminal amino-acid group Chemical group 0.000 description 1
- 238000011740 C57BL/6 mouse Methods 0.000 description 1
- 241000282472 Canis lupus familiaris Species 0.000 description 1
- 108091005462 Cation channels Proteins 0.000 description 1
- VEXZGXHMUGYJMC-UHFFFAOYSA-M Chloride anion Chemical compound [Cl-] VEXZGXHMUGYJMC-UHFFFAOYSA-M 0.000 description 1
- 241000195628 Chlorophyta Species 0.000 description 1
- 108010077544 Chromatin Proteins 0.000 description 1
- 108020004635 Complementary DNA Proteins 0.000 description 1
- 229920002261 Corn starch Polymers 0.000 description 1
- 230000006429 DNA hypomethylation Effects 0.000 description 1
- 230000007067 DNA methylation Effects 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
- 241000196324 Embryophyta Species 0.000 description 1
- 108090000790 Enzymes Proteins 0.000 description 1
- 102000004190 Enzymes Human genes 0.000 description 1
- 241000283086 Equidae Species 0.000 description 1
- 108700039887 Essential Genes Proteins 0.000 description 1
- LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 description 1
- 108700024394 Exon Proteins 0.000 description 1
- 241000282326 Felis catus Species 0.000 description 1
- 241000287828 Gallus gallus Species 0.000 description 1
- 241000204946 Halobacterium salinarum Species 0.000 description 1
- 102000001554 Hemoglobins Human genes 0.000 description 1
- 108010054147 Hemoglobins Proteins 0.000 description 1
- HTTJABKRGRZYRN-UHFFFAOYSA-N Heparin Chemical compound OC1C(NC(=O)C)C(O)OC(COS(O)(=O)=O)C1OC1C(OS(O)(=O)=O)C(O)C(OC2C(C(OS(O)(=O)=O)C(OC3C(C(O)C(O)C(O3)C(O)=O)OS(O)(=O)=O)C(CO)O2)NS(O)(=O)=O)C(C(O)=O)O1 HTTJABKRGRZYRN-UHFFFAOYSA-N 0.000 description 1
- 108010033040 Histones Proteins 0.000 description 1
- 229930010555 Inosine Natural products 0.000 description 1
- 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 1
- 108010083687 Ion Pumps 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
- 235000010643 Leucaena leucocephala Nutrition 0.000 description 1
- 240000007472 Leucaena leucocephala Species 0.000 description 1
- 235000019759 Maize starch Nutrition 0.000 description 1
- 241000124008 Mammalia Species 0.000 description 1
- 108010052285 Membrane Proteins Proteins 0.000 description 1
- 229920000168 Microcrystalline cellulose Polymers 0.000 description 1
- 241000204971 Natronomonas pharaonis Species 0.000 description 1
- 208000012902 Nervous system disease Diseases 0.000 description 1
- 108010047956 Nucleosomes Proteins 0.000 description 1
- 102000010292 Peptide Elongation Factor 1 Human genes 0.000 description 1
- 108010077524 Peptide Elongation Factor 1 Proteins 0.000 description 1
- 239000002202 Polyethylene glycol Substances 0.000 description 1
- 108010029485 Protein Isoforms Proteins 0.000 description 1
- 102000001708 Protein Isoforms Human genes 0.000 description 1
- 241000711798 Rabies lyssavirus Species 0.000 description 1
- 102000007056 Recombinant Fusion Proteins Human genes 0.000 description 1
- 108010008281 Recombinant Fusion Proteins Proteins 0.000 description 1
- 108700008625 Reporter Genes Proteins 0.000 description 1
- NCYCYZXNIZJOKI-OVSJKPMPSA-N Retinaldehyde Chemical compound O=C\C=C(/C)\C=C\C=C(/C)\C=C\C1=C(C)CCCC1(C)C NCYCYZXNIZJOKI-OVSJKPMPSA-N 0.000 description 1
- 102000004330 Rhodopsin Human genes 0.000 description 1
- 108090000820 Rhodopsin Proteins 0.000 description 1
- AUNGANRZJHBGPY-SCRDCRAPSA-N Riboflavin Chemical compound OC[C@@H](O)[C@@H](O)[C@@H](O)CN1C=2C=C(C)C(C)=CC=2N=C2C1=NC(=O)NC2=O AUNGANRZJHBGPY-SCRDCRAPSA-N 0.000 description 1
- 241000283984 Rodentia Species 0.000 description 1
- 241000242583 Scyphozoa Species 0.000 description 1
- 108010022999 Serine Proteases Proteins 0.000 description 1
- 102000012479 Serine Proteases Human genes 0.000 description 1
- 241000700584 Simplexvirus Species 0.000 description 1
- 108020004682 Single-Stranded DNA Proteins 0.000 description 1
- 239000004141 Sodium laurylsulphate Substances 0.000 description 1
- 108090000787 Subtilisin Proteins 0.000 description 1
- 241000282887 Suidae Species 0.000 description 1
- 101150006914 TRP1 gene Proteins 0.000 description 1
- 108020004566 Transfer RNA Proteins 0.000 description 1
- LVTKHGUGBGNBPL-UHFFFAOYSA-N Trp-P-1 Chemical compound N1C2=CC=CC=C2C2=C1C(C)=C(N)N=C2C LVTKHGUGBGNBPL-UHFFFAOYSA-N 0.000 description 1
- 108090000848 Ubiquitin Proteins 0.000 description 1
- 102000044159 Ubiquitin Human genes 0.000 description 1
- 241000195615 Volvox Species 0.000 description 1
- 241001492404 Woodchuck hepatitis virus Species 0.000 description 1
- 238000002835 absorbance Methods 0.000 description 1
- 230000021736 acetylation Effects 0.000 description 1
- 238000006640 acetylation reaction Methods 0.000 description 1
- 230000003213 activating effect Effects 0.000 description 1
- 230000010933 acylation Effects 0.000 description 1
- 238000005917 acylation reaction Methods 0.000 description 1
- 239000000654 additive Substances 0.000 description 1
- 239000008272 agar Substances 0.000 description 1
- 230000002776 aggregation Effects 0.000 description 1
- 238000004220 aggregation Methods 0.000 description 1
- 150000004347 all-trans-retinol derivatives Chemical class 0.000 description 1
- 239000008168 almond oil Substances 0.000 description 1
- 230000009435 amidation Effects 0.000 description 1
- 238000007112 amidation reaction Methods 0.000 description 1
- 125000000539 amino acid group Chemical group 0.000 description 1
- 238000004458 analytical method Methods 0.000 description 1
- 239000003125 aqueous solvent Substances 0.000 description 1
- 239000008135 aqueous vehicle Substances 0.000 description 1
- 230000010516 arginylation Effects 0.000 description 1
- 238000003556 assay Methods 0.000 description 1
- 230000001580 bacterial effect Effects 0.000 description 1
- 239000011230 binding agent Substances 0.000 description 1
- 238000004166 bioassay Methods 0.000 description 1
- 230000008033 biological extinction Effects 0.000 description 1
- 230000008827 biological function Effects 0.000 description 1
- 230000033228 biological regulation Effects 0.000 description 1
- 230000006287 biotinylation Effects 0.000 description 1
- 238000007413 biotinylation Methods 0.000 description 1
- 238000006664 bond formation reaction Methods 0.000 description 1
- 239000000872 buffer Substances 0.000 description 1
- 239000008366 buffered solution Substances 0.000 description 1
- FUFJGUQYACFECW-UHFFFAOYSA-L calcium hydrogenphosphate Chemical compound [Ca+2].OP([O-])([O-])=O FUFJGUQYACFECW-UHFFFAOYSA-L 0.000 description 1
- 230000009460 calcium influx Effects 0.000 description 1
- 239000002775 capsule Substances 0.000 description 1
- 239000000969 carrier Substances 0.000 description 1
- 239000013592 cell lysate Substances 0.000 description 1
- 230000033077 cellular process Effects 0.000 description 1
- 239000001913 cellulose Substances 0.000 description 1
- 235000010980 cellulose Nutrition 0.000 description 1
- 229920002678 cellulose Polymers 0.000 description 1
- 238000012512 characterization method Methods 0.000 description 1
- 238000007385 chemical modification Methods 0.000 description 1
- 235000013330 chicken meat Nutrition 0.000 description 1
- 210000003483 chromatin Anatomy 0.000 description 1
- 230000002759 chromosomal effect Effects 0.000 description 1
- 238000010367 cloning Methods 0.000 description 1
- 238000004891 communication Methods 0.000 description 1
- 238000013270 controlled release Methods 0.000 description 1
- 230000001276 controlling effect Effects 0.000 description 1
- 239000006071 cream Substances 0.000 description 1
- 238000004132 cross linking Methods 0.000 description 1
- 238000009402 cross-breeding Methods 0.000 description 1
- 239000013078 crystal Substances 0.000 description 1
- 235000018417 cysteine Nutrition 0.000 description 1
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 description 1
- 239000003405 delayed action preparation Substances 0.000 description 1
- 230000017858 demethylation Effects 0.000 description 1
- 238000010520 demethylation reaction Methods 0.000 description 1
- 238000011161 development Methods 0.000 description 1
- 229960000633 dextran sulfate Drugs 0.000 description 1
- 238000003745 diagnosis Methods 0.000 description 1
- 235000019700 dicalcium phosphate Nutrition 0.000 description 1
- 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 1
- 201000010099 disease Diseases 0.000 description 1
- 239000007884 disintegrant Substances 0.000 description 1
- 239000002270 dispersing agent Substances 0.000 description 1
- 238000002224 dissection Methods 0.000 description 1
- 229940079593 drug Drugs 0.000 description 1
- 238000007876 drug discovery Methods 0.000 description 1
- 229940126534 drug product Drugs 0.000 description 1
- 239000000975 dye Substances 0.000 description 1
- 238000001962 electrophoresis Methods 0.000 description 1
- 238000004520 electroporation Methods 0.000 description 1
- 210000002257 embryonic structure Anatomy 0.000 description 1
- 239000003995 emulsifying agent Substances 0.000 description 1
- 238000005516 engineering process Methods 0.000 description 1
- 230000001973 epigenetic effect Effects 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
- 230000001747 exhibiting effect Effects 0.000 description 1
- 238000002474 experimental method Methods 0.000 description 1
- 239000003925 fat Substances 0.000 description 1
- 230000004720 fertilization Effects 0.000 description 1
- 230000001605 fetal effect Effects 0.000 description 1
- 239000000945 filler Substances 0.000 description 1
- 238000010304 firing Methods 0.000 description 1
- 239000012530 fluid Substances 0.000 description 1
- GNBHRKFJIUUOQI-UHFFFAOYSA-N fluorescein Chemical compound O1C(=O)C2=CC=CC=C2C21C1=CC=C(O)C=C1OC1=CC(O)=CC=C21 GNBHRKFJIUUOQI-UHFFFAOYSA-N 0.000 description 1
- 239000007850 fluorescent dye Substances 0.000 description 1
- 239000011888 foil Substances 0.000 description 1
- 230000022244 formylation Effects 0.000 description 1
- 238000006170 formylation reaction Methods 0.000 description 1
- 230000006870 function Effects 0.000 description 1
- 230000006251 gamma-carboxylation Effects 0.000 description 1
- 230000004545 gene duplication Effects 0.000 description 1
- 230000030279 gene silencing Effects 0.000 description 1
- 230000004077 genetic alteration Effects 0.000 description 1
- 231100000118 genetic alteration Toxicity 0.000 description 1
- 238000010353 genetic engineering Methods 0.000 description 1
- 239000011521 glass Substances 0.000 description 1
- 230000013595 glycosylation Effects 0.000 description 1
- 238000006206 glycosylation reaction Methods 0.000 description 1
- PCHJSUWPFVWCPO-UHFFFAOYSA-N gold Chemical compound [Au] PCHJSUWPFVWCPO-UHFFFAOYSA-N 0.000 description 1
- 239000010931 gold Substances 0.000 description 1
- 229910052737 gold Inorganic materials 0.000 description 1
- 150000003278 haem Chemical group 0.000 description 1
- 229960002897 heparin Drugs 0.000 description 1
- 229920000669 heparin Polymers 0.000 description 1
- 239000012456 homogeneous solution Substances 0.000 description 1
- 238000002744 homologous recombination Methods 0.000 description 1
- 230000006801 homologous recombination Effects 0.000 description 1
- 230000002209 hydrophobic effect Effects 0.000 description 1
- 230000033444 hydroxylation Effects 0.000 description 1
- 238000005805 hydroxylation reaction Methods 0.000 description 1
- 239000001866 hydroxypropyl methyl cellulose Substances 0.000 description 1
- 235000010979 hydroxypropyl methyl cellulose Nutrition 0.000 description 1
- 229920003088 hydroxypropyl methyl cellulose Polymers 0.000 description 1
- UFVKGYZPFZQRLF-UHFFFAOYSA-N hydroxypropyl methyl cellulose Chemical compound OC1C(O)C(OC)OC(CO)C1OC1C(O)C(O)C(OC2C(C(O)C(OC3C(C(O)C(O)C(CO)O3)O)C(CO)O2)O)C(CO)O1 UFVKGYZPFZQRLF-UHFFFAOYSA-N 0.000 description 1
- 210000003016 hypothalamus Anatomy 0.000 description 1
- 238000010191 image analysis Methods 0.000 description 1
- 210000002865 immune cell Anatomy 0.000 description 1
- 230000001771 impaired effect Effects 0.000 description 1
- 238000002513 implantation Methods 0.000 description 1
- 230000006872 improvement Effects 0.000 description 1
- 239000012678 infectious agent Substances 0.000 description 1
- 208000015181 infectious disease Diseases 0.000 description 1
- 238000001802 infusion Methods 0.000 description 1
- 229960003786 inosine Drugs 0.000 description 1
- 230000003993 interaction Effects 0.000 description 1
- 238000007918 intramuscular administration Methods 0.000 description 1
- 230000026045 iodination Effects 0.000 description 1
- 238000006192 iodination reaction Methods 0.000 description 1
- 239000003456 ion exchange resin Substances 0.000 description 1
- 229920003303 ion-exchange polymer Polymers 0.000 description 1
- 238000002955 isolation Methods 0.000 description 1
- 239000008101 lactose Substances 0.000 description 1
- 239000000787 lecithin Substances 0.000 description 1
- 235000010445 lecithin Nutrition 0.000 description 1
- 229940067606 lecithin Drugs 0.000 description 1
- 239000003446 ligand Substances 0.000 description 1
- 239000002502 liposome Substances 0.000 description 1
- 239000006210 lotion Substances 0.000 description 1
- 239000000314 lubricant Substances 0.000 description 1
- 235000019359 magnesium stearate Nutrition 0.000 description 1
- 238000012423 maintenance Methods 0.000 description 1
- 210000004962 mammalian cell Anatomy 0.000 description 1
- 210000001161 mammalian embryo Anatomy 0.000 description 1
- 230000007246 mechanism Effects 0.000 description 1
- 210000001259 mesencephalon Anatomy 0.000 description 1
- 229910052751 metal Inorganic materials 0.000 description 1
- 239000002184 metal Substances 0.000 description 1
- MYWUZJCMWCOHBA-VIFPVBQESA-N methamphetamine Chemical compound CN[C@@H](C)CC1=CC=CC=C1 MYWUZJCMWCOHBA-VIFPVBQESA-N 0.000 description 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 description 1
- 235000010270 methyl p-hydroxybenzoate Nutrition 0.000 description 1
- 230000011987 methylation Effects 0.000 description 1
- 238000007069 methylation reaction Methods 0.000 description 1
- 230000000813 microbial effect Effects 0.000 description 1
- 235000019813 microcrystalline cellulose Nutrition 0.000 description 1
- 239000008108 microcrystalline cellulose Substances 0.000 description 1
- 229940016286 microcrystalline cellulose Drugs 0.000 description 1
- 238000001000 micrograph Methods 0.000 description 1
- 238000000520 microinjection Methods 0.000 description 1
- 238000002406 microsurgery Methods 0.000 description 1
- 235000019799 monosodium phosphate Nutrition 0.000 description 1
- 210000000472 morula Anatomy 0.000 description 1
- 210000000663 muscle cell Anatomy 0.000 description 1
- 238000002703 mutagenesis Methods 0.000 description 1
- 231100000350 mutagenesis Toxicity 0.000 description 1
- 230000007498 myristoylation Effects 0.000 description 1
- 230000001537 neural effect Effects 0.000 description 1
- 208000015122 neurodegenerative disease Diseases 0.000 description 1
- 239000002687 nonaqueous vehicle Substances 0.000 description 1
- 239000002736 nonionic surfactant Substances 0.000 description 1
- 231100000252 nontoxic Toxicity 0.000 description 1
- 230000003000 nontoxic effect Effects 0.000 description 1
- 210000001623 nucleosome Anatomy 0.000 description 1
- 239000003921 oil Substances 0.000 description 1
- 235000019198 oils Nutrition 0.000 description 1
- 230000003647 oxidation Effects 0.000 description 1
- 238000007254 oxidation reaction Methods 0.000 description 1
- 238000007911 parenteral administration Methods 0.000 description 1
- 230000001575 pathological effect Effects 0.000 description 1
- 230000006320 pegylation Effects 0.000 description 1
- 239000008194 pharmaceutical composition Substances 0.000 description 1
- 239000000546 pharmaceutical excipient Substances 0.000 description 1
- 230000026731 phosphorylation Effects 0.000 description 1
- 238000006366 phosphorylation reaction Methods 0.000 description 1
- 230000029264 phototaxis Effects 0.000 description 1
- 239000000049 pigment Substances 0.000 description 1
- 239000013600 plasmid vector Substances 0.000 description 1
- 239000004033 plastic Substances 0.000 description 1
- 229920003023 plastic Polymers 0.000 description 1
- 229920001223 polyethylene glycol Polymers 0.000 description 1
- 229920000136 polysorbate Polymers 0.000 description 1
- 239000001267 polyvinylpyrrolidone Substances 0.000 description 1
- 229920000036 polyvinylpyrrolidone Polymers 0.000 description 1
- 235000013855 polyvinylpyrrolidone Nutrition 0.000 description 1
- 230000001124 posttranscriptional effect Effects 0.000 description 1
- 229920001592 potato starch Polymers 0.000 description 1
- 239000000843 powder Substances 0.000 description 1
- 244000062645 predators Species 0.000 description 1
- 230000013823 prenylation Effects 0.000 description 1
- 230000002335 preservative effect Effects 0.000 description 1
- 230000003518 presynaptic effect Effects 0.000 description 1
- 210000005215 presynaptic neuron Anatomy 0.000 description 1
- 230000002265 prevention Effects 0.000 description 1
- 235000010232 propyl p-hydroxybenzoate Nutrition 0.000 description 1
- QELSKZZBTMNZEB-UHFFFAOYSA-N propylparaben Chemical class CCCOC(=O)C1=CC=C(O)C=C1 QELSKZZBTMNZEB-UHFFFAOYSA-N 0.000 description 1
- 230000001681 protective effect Effects 0.000 description 1
- 210000004777 protein coat Anatomy 0.000 description 1
- 230000002797 proteolythic effect Effects 0.000 description 1
- 208000009305 pseudorabies Diseases 0.000 description 1
- 229940043131 pyroglutamate Drugs 0.000 description 1
- 238000004445 quantitative analysis Methods 0.000 description 1
- 230000006340 racemization Effects 0.000 description 1
- 230000006798 recombination Effects 0.000 description 1
- 238000005215 recombination Methods 0.000 description 1
- 230000022532 regulation of transcription, DNA-dependent Effects 0.000 description 1
- 230000010076 replication Effects 0.000 description 1
- 230000003362 replicative effect Effects 0.000 description 1
- 230000004044 response Effects 0.000 description 1
- 108091008146 restriction endonucleases Proteins 0.000 description 1
- 210000001116 retinal neuron Anatomy 0.000 description 1
- PYWVYCXTNDRMGF-UHFFFAOYSA-N rhodamine B Chemical compound [Cl-].C=12C=CC(=[N+](CC)CC)C=C2OC2=CC(N(CC)CC)=CC=C2C=1C1=CC=CC=C1C(O)=O PYWVYCXTNDRMGF-UHFFFAOYSA-N 0.000 description 1
- 238000007363 ring formation reaction Methods 0.000 description 1
- 239000000523 sample Substances 0.000 description 1
- 210000003786 sclera Anatomy 0.000 description 1
- 230000001953 sensory effect Effects 0.000 description 1
- 210000002966 serum Anatomy 0.000 description 1
- 239000000377 silicon dioxide Substances 0.000 description 1
- 239000001509 sodium citrate Substances 0.000 description 1
- AJPJDKMHJJGVTQ-UHFFFAOYSA-M sodium dihydrogen phosphate Chemical compound [Na+].OP(O)([O-])=O AJPJDKMHJJGVTQ-UHFFFAOYSA-M 0.000 description 1
- 239000001488 sodium phosphate Substances 0.000 description 1
- 235000011008 sodium phosphates Nutrition 0.000 description 1
- 229940079832 sodium starch glycolate Drugs 0.000 description 1
- 239000008109 sodium starch glycolate Substances 0.000 description 1
- 229920003109 sodium starch glycolate Polymers 0.000 description 1
- 239000012453 solvate Substances 0.000 description 1
- 235000010199 sorbic acid Nutrition 0.000 description 1
- 229940075582 sorbic acid Drugs 0.000 description 1
- 239000004334 sorbic acid Substances 0.000 description 1
- 239000000600 sorbitol Substances 0.000 description 1
- 235000010356 sorbitol Nutrition 0.000 description 1
- 230000003595 spectral effect Effects 0.000 description 1
- 239000003381 stabilizer Substances 0.000 description 1
- 230000000087 stabilizing effect Effects 0.000 description 1
- 238000010561 standard procedure Methods 0.000 description 1
- 238000007920 subcutaneous administration Methods 0.000 description 1
- 238000006467 substitution reaction Methods 0.000 description 1
- 230000019635 sulfation Effects 0.000 description 1
- 238000005670 sulfation reaction Methods 0.000 description 1
- 230000000946 synaptic effect Effects 0.000 description 1
- 238000010189 synthetic method Methods 0.000 description 1
- 239000006188 syrup Substances 0.000 description 1
- 235000020357 syrup Nutrition 0.000 description 1
- 239000000454 talc Substances 0.000 description 1
- 229910052623 talc Inorganic materials 0.000 description 1
- 210000001103 thalamus Anatomy 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
- 238000011285 therapeutic regimen Methods 0.000 description 1
- ANRHNWWPFJCPAZ-UHFFFAOYSA-M thionine Chemical compound [Cl-].C1=CC(N)=CC2=[S+]C3=CC(N)=CC=C3N=C21 ANRHNWWPFJCPAZ-UHFFFAOYSA-M 0.000 description 1
- 230000036962 time dependent Effects 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
- 230000002103 transcriptional effect Effects 0.000 description 1
- 238000010361 transduction Methods 0.000 description 1
- 230000026683 transduction Effects 0.000 description 1
- 238000012546 transfer Methods 0.000 description 1
- HRXKRNGNAMMEHJ-UHFFFAOYSA-K trisodium citrate Chemical compound [Na+].[Na+].[Na+].[O-]C(=O)CC(O)(CC([O-])=O)C([O-])=O HRXKRNGNAMMEHJ-UHFFFAOYSA-K 0.000 description 1
- 229940038773 trisodium citrate Drugs 0.000 description 1
- RYFMWSXOAZQYPI-UHFFFAOYSA-K trisodium phosphate Chemical compound [Na+].[Na+].[Na+].[O-]P([O-])([O-])=O RYFMWSXOAZQYPI-UHFFFAOYSA-K 0.000 description 1
- 230000001810 trypsinlike Effects 0.000 description 1
- 241001430294 unidentified retrovirus Species 0.000 description 1
- 235000015112 vegetable and seed oil Nutrition 0.000 description 1
- 239000008158 vegetable oil Substances 0.000 description 1
- 210000002845 virion Anatomy 0.000 description 1
- 238000001429 visible spectrum Methods 0.000 description 1
- 239000000080 wetting agent Substances 0.000 description 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0618—Cells of the nervous system
- C12N5/0619—Neurons
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0618—Cells of the nervous system
- C12N5/0621—Eye cells, e.g. cornea, iris pigmented cells
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
- C12N2015/8527—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic for producing animal models, e.g. for tests or diseases
- C12N2015/859—Animal models comprising reporter system for screening tests
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2510/00—Genetically modified cells
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/022—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from an adenovirus
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Neurology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Neurosurgery (AREA)
- Cell Biology (AREA)
- Ophthalmology & Optometry (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP16196818.5 | 2016-11-02 | ||
| EP16196818 | 2016-11-02 | ||
| PCT/IB2017/056792 WO2018083607A1 (en) | 2016-11-02 | 2017-11-01 | Synp198, a promoter for the specific expression of genes in direction selective retinal ganglion cells |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3042197A1 true CA3042197A1 (en) | 2018-05-11 |
Family
ID=57223559
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3042197A Abandoned CA3042197A1 (en) | 2016-11-02 | 2017-11-01 | Synp198, a promoter for the specific expression of genes in direction selective retinal ganglion cells |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US11525145B2 (enExample) |
| EP (1) | EP3534966B1 (enExample) |
| JP (1) | JP7023279B2 (enExample) |
| KR (1) | KR20190077471A (enExample) |
| CN (1) | CN110072557B (enExample) |
| AU (1) | AU2017354086C1 (enExample) |
| BR (1) | BR112019008488A2 (enExample) |
| CA (1) | CA3042197A1 (enExample) |
| IL (1) | IL266115A (enExample) |
| RU (1) | RU2766353C2 (enExample) |
| WO (1) | WO2018083607A1 (enExample) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2679246A3 (en) | 2008-04-18 | 2014-02-26 | Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research | Novel therapeutical tools and methods for treating blindness |
| US10941417B2 (en) | 2015-04-30 | 2021-03-09 | Friedrich Miescher Institute For Biomedical Research | Promoter for the specific expression of genes in Müller cells |
| US10857241B2 (en) | 2015-09-15 | 2020-12-08 | Friedrich Miescher Institute For Biomedical Research | Therapeutical tools and methods for treating blindness by targeting photoreceptors |
| WO2017064642A1 (en) | 2015-10-14 | 2017-04-20 | Fredrich Miescher Institute For Biomedical Research | Promoter for the specific expression of genes in retinal endothelial cells |
| ES2886664T3 (es) | 2015-12-03 | 2021-12-20 | Friedrich Miescher Institute For Biomedical Res | SynP162, un promotor para la expresión específica de genes en fotorreceptores de bastón |
| ES2909444T3 (es) | 2015-12-03 | 2022-05-06 | Friedrich Miescher Institute For Biomedical Res | Synp159, un promotor para la expresión específica de genes en los fotorreceptores de los bastones |
| US10994026B2 (en) | 2015-12-03 | 2021-05-04 | Friedrich Miescher Institute For Biomedical Research | SynP160, a promoter for the specific expression of genes in rod photoreceptors |
| WO2017093935A1 (en) | 2015-12-03 | 2017-06-08 | Friedrich Miescher Institute For Biomedical Research | Synp161, a promoter for the specific expression of genes in rod photoreceptors |
| CA3042197A1 (en) | 2016-11-02 | 2018-05-11 | Friedrich Miescher Institute For Biomedical Research | Synp198, a promoter for the specific expression of genes in direction selective retinal ganglion cells |
| CN110392582A (zh) | 2017-02-08 | 2019-10-29 | 弗里德里克·米谢尔生物医学研究所 | 用于使基因在视网膜神经节细胞中特异性表达的启动子SynP88 |
| WO2019097454A1 (en) | 2017-11-15 | 2019-05-23 | Friedrich Miescher Institute For Biomedical Research | Primate retinal pigment epithelium cell-specific promoter |
| WO2019106035A1 (en) | 2017-11-30 | 2019-06-06 | Friedrich Miescher Institute For Biomedical Research | Synpiii, a promoter for the specific expression of genes in retinal pigment epithelium |
| EP3870242A1 (en) * | 2018-10-25 | 2021-09-01 | Friedrich Miescher Institute for Biomedical Research | Synp151 (proc29), a promoter for the specific expression of genes in retinal ganglion cells |
| KR20230148207A (ko) * | 2021-02-22 | 2023-10-24 | 더 리전츠 오브 더 유니버시티 오브 콜로라도, 어 바디 코퍼레이트 | 안구 병태에 대한 바이러스 벡터-기반 유전자 요법 |
Family Cites Families (38)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2019588A4 (en) * | 2006-05-04 | 2010-11-24 | Univ Wayne State | RESTORATION OF VISUAL RESPONSES BY IN VIVO ADMINISTRATION OF NUCLEIC ACIDS OF RHODOPSIN |
| EP1891976A1 (en) | 2006-08-23 | 2008-02-27 | Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute for Biomedical Research | Use of light sensitive genes |
| EP2679246A3 (en) | 2008-04-18 | 2014-02-26 | Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research | Novel therapeutical tools and methods for treating blindness |
| DK2350279T3 (en) * | 2008-10-22 | 2016-02-15 | Quark Pharmaceuticals Inc | METHODS OF TREATING EYE DISEASES |
| WO2013068413A1 (en) * | 2011-11-08 | 2013-05-16 | Novartis Forschungsstiftung, Zweigniederlassung, Friedrich Miescher Institute For Biomedical Research | Rod cell-specific promoter |
| US9579399B2 (en) | 2012-08-27 | 2017-02-28 | Friedrich Miescher Institute For Biomedical Research | Retinal OFF circuit-specific promoter |
| US9724429B2 (en) * | 2013-05-16 | 2017-08-08 | Applied Genetic Technologies Corporation | Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases |
| WO2014199299A1 (en) | 2013-06-11 | 2014-12-18 | Friedrich Miescher Institute For Biomedical Research | Retinal on bipolar cells-specific artificial promoter |
| EP3104895A1 (en) | 2014-02-10 | 2016-12-21 | Friedrich Miescher Institute for Biomedical Research | Aii retinal amacrine cell-specific promoter |
| WO2015121793A1 (en) | 2014-02-11 | 2015-08-20 | Friedrich Miescher Institute For Biomedical Research | Müller cell-specific promoter |
| US10941417B2 (en) | 2015-04-30 | 2021-03-09 | Friedrich Miescher Institute For Biomedical Research | Promoter for the specific expression of genes in Müller cells |
| US10857241B2 (en) | 2015-09-15 | 2020-12-08 | Friedrich Miescher Institute For Biomedical Research | Therapeutical tools and methods for treating blindness by targeting photoreceptors |
| WO2017064642A1 (en) | 2015-10-14 | 2017-04-20 | Fredrich Miescher Institute For Biomedical Research | Promoter for the specific expression of genes in retinal endothelial cells |
| US10994026B2 (en) | 2015-12-03 | 2021-05-04 | Friedrich Miescher Institute For Biomedical Research | SynP160, a promoter for the specific expression of genes in rod photoreceptors |
| ES2886664T3 (es) | 2015-12-03 | 2021-12-20 | Friedrich Miescher Institute For Biomedical Res | SynP162, un promotor para la expresión específica de genes en fotorreceptores de bastón |
| WO2017093935A1 (en) | 2015-12-03 | 2017-06-08 | Friedrich Miescher Institute For Biomedical Research | Synp161, a promoter for the specific expression of genes in rod photoreceptors |
| ES2909444T3 (es) | 2015-12-03 | 2022-05-06 | Friedrich Miescher Institute For Biomedical Res | Synp159, un promotor para la expresión específica de genes en los fotorreceptores de los bastones |
| AU2016363830C1 (en) | 2015-12-04 | 2022-03-24 | Centre National De La Recherche Scientifique | Promoters and uses thereof |
| US20190209708A1 (en) | 2016-05-17 | 2019-07-11 | Friedrich Miescher Institute For Biomedical Research | Novel therapeutic tools and methods for treating blindness |
| CA3042197A1 (en) | 2016-11-02 | 2018-05-11 | Friedrich Miescher Institute For Biomedical Research | Synp198, a promoter for the specific expression of genes in direction selective retinal ganglion cells |
| CN110072559A (zh) | 2016-12-01 | 2019-07-30 | 弗里德里克·米谢尔生物医学研究所 | 用于使基因在中间神经元中特异性表达的启动子SynP107 |
| US20190376083A1 (en) | 2016-12-01 | 2019-12-12 | Friedrich Miescher Institute For Biomedical Research | Synp1, a promoter for the specific expression of genes in interneurons |
| CN110392582A (zh) | 2017-02-08 | 2019-10-29 | 弗里德里克·米谢尔生物医学研究所 | 用于使基因在视网膜神经节细胞中特异性表达的启动子SynP88 |
| WO2019097454A1 (en) | 2017-11-15 | 2019-05-23 | Friedrich Miescher Institute For Biomedical Research | Primate retinal pigment epithelium cell-specific promoter |
| BR112020010775A2 (pt) | 2017-11-30 | 2020-11-24 | Friedrich Miescher Institute For Biomedical Research | synp61, promotor específico à célula de epitélio de pigmento retiniano de primata |
| WO2019106035A1 (en) | 2017-11-30 | 2019-06-06 | Friedrich Miescher Institute For Biomedical Research | Synpiii, a promoter for the specific expression of genes in retinal pigment epithelium |
| EP3870239A1 (en) | 2018-10-25 | 2021-09-01 | Friedrich Miescher Institute for Biomedical Research | Synp57 (proa14), a promoter for the specific expression of genes in photoreceptors |
| EP3870242A1 (en) | 2018-10-25 | 2021-09-01 | Friedrich Miescher Institute for Biomedical Research | Synp151 (proc29), a promoter for the specific expression of genes in retinal ganglion cells |
| EP3870709A1 (en) | 2018-10-25 | 2021-09-01 | Friedrich Miescher Institute for Biomedical Research | Synp27 (prob12), a promoter for the specific expression of genes in protoplasmic astrocytes |
| US20210353773A1 (en) | 2018-10-25 | 2021-11-18 | Friedrich Miescher Institute For Biomedical Research | Synp194 (prob15), a promoter for the specific expression of genes in retinal ganglion cells |
| WO2020084537A1 (en) | 2018-10-25 | 2020-04-30 | Friedrich Miescher Institute For Biomedical Research | Synp17 (prob1), a promoter for the specific expression of genes in retinal ganglion cells |
| US20210355505A1 (en) | 2018-10-25 | 2021-11-18 | Friedrich Miescher Institute For Biomedical Research | Synp78 (proa27), a promoter for the specific expression of genes in retinal ganglion cells |
| WO2020152624A1 (en) | 2019-01-24 | 2020-07-30 | Friedrich Miescher Institute For Biomedical Research | Synp35 (proc8), a promoter for the specific expression of genes in retinal ganglion cells |
| WO2020152626A1 (en) | 2019-01-24 | 2020-07-30 | Friedrich Miescher Institute For Biomedical Research | Synp166 (proa36), a promoter for the specific expression of genes in photoreceptors |
| EP3914300A1 (en) | 2019-01-24 | 2021-12-01 | Friedrich Miescher Institute for Biomedical Research | Synp66 (proa21), a promoter for the specific expression of genes in retinal ganglion cells |
| US20220119841A1 (en) | 2019-01-24 | 2022-04-21 | Friedrich Miescher Instit for Biomedical Research | Synp5 (proa9), a promoter for the specific expression of genes in retinal ganglion cells |
| JP2022521025A (ja) | 2019-02-25 | 2022-04-04 | ノバルティス アーゲー | Biettiクリスタリン網膜症を治療するための組成物及び方法 |
| US20220154211A1 (en) | 2019-02-25 | 2022-05-19 | Novartis Ag | Compositions and methods to treat bietti crystalline dystrophy |
-
2017
- 2017-11-01 CA CA3042197A patent/CA3042197A1/en not_active Abandoned
- 2017-11-01 RU RU2019115117A patent/RU2766353C2/ru active
- 2017-11-01 KR KR1020197015426A patent/KR20190077471A/ko not_active Ceased
- 2017-11-01 US US16/346,680 patent/US11525145B2/en active Active
- 2017-11-01 CN CN201780067090.8A patent/CN110072557B/zh not_active Expired - Fee Related
- 2017-11-01 EP EP17811378.3A patent/EP3534966B1/en active Active
- 2017-11-01 AU AU2017354086A patent/AU2017354086C1/en not_active Ceased
- 2017-11-01 JP JP2019522550A patent/JP7023279B2/ja not_active Expired - Fee Related
- 2017-11-01 WO PCT/IB2017/056792 patent/WO2018083607A1/en not_active Ceased
- 2017-11-01 BR BR112019008488A patent/BR112019008488A2/pt not_active Application Discontinuation
-
2019
- 2019-04-17 IL IL266115A patent/IL266115A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP3534966A1 (en) | 2019-09-11 |
| CN110072557B (zh) | 2022-11-11 |
| BR112019008488A2 (pt) | 2019-07-09 |
| JP2019537437A (ja) | 2019-12-26 |
| KR20190077471A (ko) | 2019-07-03 |
| RU2766353C2 (ru) | 2022-03-15 |
| RU2019115117A3 (enExample) | 2021-03-23 |
| AU2017354086A1 (en) | 2019-05-09 |
| AU2017354086B2 (en) | 2021-01-28 |
| JP7023279B2 (ja) | 2022-02-21 |
| IL266115A (en) | 2019-06-30 |
| RU2019115117A (ru) | 2020-12-03 |
| US11525145B2 (en) | 2022-12-13 |
| WO2018083607A1 (en) | 2018-05-11 |
| AU2017354086C1 (en) | 2021-05-06 |
| CN110072557A (zh) | 2019-07-30 |
| EP3534966B1 (en) | 2024-06-19 |
| US20190276847A1 (en) | 2019-09-12 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US11857642B2 (en) | SYNP161, a promoter for the expression of genes | |
| US11883507B2 (en) | Expression cassette with a SynP160 promoter | |
| US11591618B2 (en) | SynP159, a promoter for the expression of genes | |
| US11608365B2 (en) | SYNP162, a promoter for the expression of genes | |
| AU2017354086B2 (en) | SynP198, a promoter for the specific expression of genes in direction selective retinal ganglion cells | |
| US9999685B2 (en) | AII retinal amacrine cell-specific promoter | |
| CA3082080A1 (en) | Primate retinal pigment epithelium cell-specific promoter | |
| US20180298378A1 (en) | Promoter for the specific expression of genes in retinal endothelial cells | |
| EP3176177A1 (en) | Synp157, a promoter for the specific expression of genes in rod photoreceptors | |
| US9862970B2 (en) | Retinal on bipolar cells-specific artificial promoter |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FZDE | Discontinued |
Effective date: 20240213 |