CA2942716C - Molecules for targeting compounds to various selected organs or tissues - Google Patents
Molecules for targeting compounds to various selected organs or tissues Download PDFInfo
- Publication number
- CA2942716C CA2942716C CA2942716A CA2942716A CA2942716C CA 2942716 C CA2942716 C CA 2942716C CA 2942716 A CA2942716 A CA 2942716A CA 2942716 A CA2942716 A CA 2942716A CA 2942716 C CA2942716 C CA 2942716C
- Authority
- CA
- Canada
- Prior art keywords
- peptide
- muscle
- moiety
- peptidomimetic
- biologically active
- 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.)
- Expired - Fee Related
Links
- 150000001875 compounds Chemical class 0.000 title claims description 34
- 230000008685 targeting Effects 0.000 title claims description 18
- 210000000056 organ Anatomy 0.000 title abstract description 13
- 108090000623 proteins and genes Proteins 0.000 claims abstract description 36
- 210000000663 muscle cell Anatomy 0.000 claims abstract description 27
- 102000004169 proteins and genes Human genes 0.000 claims abstract description 24
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 103
- 239000000816 peptidomimetic Substances 0.000 claims description 31
- 239000003814 drug Substances 0.000 claims description 15
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 14
- 238000011282 treatment Methods 0.000 claims description 10
- 208000021642 Muscular disease Diseases 0.000 claims description 7
- 201000009623 Myopathy Diseases 0.000 claims description 7
- 208000035475 disorder Diseases 0.000 claims description 7
- 108020004707 nucleic acids Proteins 0.000 claims description 7
- 102000039446 nucleic acids Human genes 0.000 claims description 7
- 150000007523 nucleic acids Chemical class 0.000 claims description 7
- 239000002773 nucleotide Substances 0.000 claims description 5
- 108091093037 Peptide nucleic acid Proteins 0.000 claims description 4
- 125000003729 nucleotide group Chemical group 0.000 claims description 4
- 238000002360 preparation method Methods 0.000 claims description 4
- 208000020446 Cardiac disease Diseases 0.000 claims description 3
- 239000005977 Ethylene Substances 0.000 claims description 3
- 206010028289 Muscle atrophy Diseases 0.000 claims description 3
- 208000008589 Obesity Diseases 0.000 claims description 3
- 206010012601 diabetes mellitus Diseases 0.000 claims description 3
- 108020001507 fusion proteins Proteins 0.000 claims description 3
- 102000037865 fusion proteins Human genes 0.000 claims description 3
- 208000019622 heart disease Diseases 0.000 claims description 3
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 3
- 201000000585 muscular atrophy Diseases 0.000 claims description 3
- 201000006938 muscular dystrophy Diseases 0.000 claims description 3
- 235000020824 obesity Nutrition 0.000 claims description 3
- VGGSQFUCUMXWEO-UHFFFAOYSA-N Ethene Chemical compound C=C VGGSQFUCUMXWEO-UHFFFAOYSA-N 0.000 claims description 2
- 125000003903 2-propenyl group Chemical group [H]C([*])([H])C([H])=C([H])[H] 0.000 claims 1
- 230000004807 localization Effects 0.000 claims 1
- 210000003205 muscle Anatomy 0.000 abstract description 31
- 210000001519 tissue Anatomy 0.000 abstract description 14
- 238000000034 method Methods 0.000 abstract description 10
- 108091034117 Oligonucleotide Proteins 0.000 abstract description 9
- 108020004459 Small interfering RNA Proteins 0.000 abstract description 6
- 241000700605 Viruses Species 0.000 abstract description 6
- 239000004055 small Interfering RNA Substances 0.000 abstract description 5
- 239000003795 chemical substances by application Substances 0.000 abstract description 2
- 210000002064 heart cell Anatomy 0.000 abstract description 2
- 230000007170 pathology Effects 0.000 abstract 2
- 210000004881 tumor cell Anatomy 0.000 abstract 1
- 102000004196 processed proteins & peptides Human genes 0.000 description 42
- 239000000562 conjugate Substances 0.000 description 29
- 241000699670 Mus sp. Species 0.000 description 26
- 235000018102 proteins Nutrition 0.000 description 21
- 150000001413 amino acids Chemical class 0.000 description 20
- 235000001014 amino acid Nutrition 0.000 description 19
- 238000001727 in vivo Methods 0.000 description 18
- 108010077850 Nuclear Localization Signals Proteins 0.000 description 14
- 210000004027 cell Anatomy 0.000 description 14
- 241000699666 Mus <mouse, genus> Species 0.000 description 12
- 230000001225 therapeutic effect Effects 0.000 description 11
- 210000004165 myocardium Anatomy 0.000 description 8
- 210000002027 skeletal muscle Anatomy 0.000 description 7
- 108020004414 DNA Proteins 0.000 description 6
- 102000001039 Dystrophin Human genes 0.000 description 6
- 108010069091 Dystrophin Proteins 0.000 description 6
- 206010068871 Myotonic dystrophy Diseases 0.000 description 6
- 230000000692 anti-sense effect Effects 0.000 description 6
- 230000008878 coupling Effects 0.000 description 6
- 238000010168 coupling process Methods 0.000 description 6
- 238000005859 coupling reaction Methods 0.000 description 6
- 238000000338 in vitro Methods 0.000 description 6
- 208000002320 spinal muscular atrophy Diseases 0.000 description 6
- 230000003612 virological effect Effects 0.000 description 6
- 102000004190 Enzymes Human genes 0.000 description 5
- 108090000790 Enzymes Proteins 0.000 description 5
- 229940088598 enzyme Drugs 0.000 description 5
- 239000007850 fluorescent dye Substances 0.000 description 5
- 238000002347 injection Methods 0.000 description 5
- 239000007924 injection Substances 0.000 description 5
- 238000012385 systemic delivery Methods 0.000 description 5
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 4
- 206010013801 Duchenne Muscular Dystrophy Diseases 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
- 230000001010 compromised effect Effects 0.000 description 4
- 201000010099 disease Diseases 0.000 description 4
- 229940079593 drug Drugs 0.000 description 4
- 239000005090 green fluorescent protein Substances 0.000 description 4
- 238000010255 intramuscular injection Methods 0.000 description 4
- 239000007927 intramuscular injection Substances 0.000 description 4
- 238000004519 manufacturing process Methods 0.000 description 4
- 239000012528 membrane Substances 0.000 description 4
- 210000003098 myoblast Anatomy 0.000 description 4
- 239000002245 particle Substances 0.000 description 4
- 210000003314 quadriceps muscle Anatomy 0.000 description 4
- 238000002560 therapeutic procedure Methods 0.000 description 4
- 238000012384 transportation and delivery Methods 0.000 description 4
- CSCPPACGZOOCGX-UHFFFAOYSA-N Acetone Chemical compound CC(C)=O CSCPPACGZOOCGX-UHFFFAOYSA-N 0.000 description 3
- 206010053185 Glycogen storage disease type II Diseases 0.000 description 3
- 108010043121 Green Fluorescent Proteins Proteins 0.000 description 3
- 102000004144 Green Fluorescent Proteins Human genes 0.000 description 3
- 208000029578 Muscle disease Diseases 0.000 description 3
- 108091028043 Nucleic acid sequence Proteins 0.000 description 3
- 230000008827 biological function Effects 0.000 description 3
- 230000006870 function Effects 0.000 description 3
- 238000001415 gene therapy Methods 0.000 description 3
- 201000004502 glycogen storage disease II Diseases 0.000 description 3
- 210000001087 myotubule Anatomy 0.000 description 3
- 238000010186 staining Methods 0.000 description 3
- 201000006935 Becker muscular dystrophy Diseases 0.000 description 2
- 201000003728 Centronuclear myopathy Diseases 0.000 description 2
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 description 2
- 101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 description 2
- 102000004472 Myostatin Human genes 0.000 description 2
- 108010056852 Myostatin Proteins 0.000 description 2
- 208000010316 Myotonia congenita Diseases 0.000 description 2
- 108010067902 Peptide Library Proteins 0.000 description 2
- 208000032978 Structural Congenital Myopathies Diseases 0.000 description 2
- AYFVYJQAPQTCCC-UHFFFAOYSA-N THREONINE Chemical compound CC(O)C(N)C(O)=O AYFVYJQAPQTCCC-UHFFFAOYSA-N 0.000 description 2
- 108700005077 Viral Genes Proteins 0.000 description 2
- 210000001015 abdomen Anatomy 0.000 description 2
- 210000003489 abdominal muscle Anatomy 0.000 description 2
- 239000002253 acid Substances 0.000 description 2
- 230000015572 biosynthetic process Effects 0.000 description 2
- 210000004899 c-terminal region Anatomy 0.000 description 2
- 210000004413 cardiac myocyte Anatomy 0.000 description 2
- 238000004113 cell culture Methods 0.000 description 2
- 230000021615 conjugation Effects 0.000 description 2
- 238000012937 correction Methods 0.000 description 2
- 238000009826 distribution Methods 0.000 description 2
- 230000000694 effects Effects 0.000 description 2
- 238000002641 enzyme replacement therapy Methods 0.000 description 2
- 238000002474 experimental method Methods 0.000 description 2
- 238000000799 fluorescence microscopy Methods 0.000 description 2
- 102000045921 human GAA Human genes 0.000 description 2
- 238000003384 imaging method Methods 0.000 description 2
- 238000010348 incorporation Methods 0.000 description 2
- 239000006166 lysate Substances 0.000 description 2
- 108020004999 messenger RNA Proteins 0.000 description 2
- 238000012986 modification Methods 0.000 description 2
- 230000004048 modification Effects 0.000 description 2
- -1 poly(oxypropylene) Polymers 0.000 description 2
- 229920001184 polypeptide Polymers 0.000 description 2
- 238000003259 recombinant expression Methods 0.000 description 2
- 238000012216 screening Methods 0.000 description 2
- 210000002363 skeletal muscle cell Anatomy 0.000 description 2
- 238000003860 storage Methods 0.000 description 2
- 230000032258 transport Effects 0.000 description 2
- 210000003462 vein Anatomy 0.000 description 2
- VJLXSTXGGXYQCT-BKLSDQPFSA-N (2s)-3-aminopyrrolidine-2-carboxylic acid Chemical compound NC1CCN[C@@H]1C(O)=O VJLXSTXGGXYQCT-BKLSDQPFSA-N 0.000 description 1
- PHIQHXFUZVPYII-ZCFIWIBFSA-O (R)-carnitinium Chemical compound C[N+](C)(C)C[C@H](O)CC(O)=O PHIQHXFUZVPYII-ZCFIWIBFSA-O 0.000 description 1
- RCZUDMZUWULMID-UHFFFAOYSA-N 2-nitrotyrosine Chemical compound OC(=O)C(N)CC1=CC=C(O)C=C1[N+]([O-])=O RCZUDMZUWULMID-UHFFFAOYSA-N 0.000 description 1
- XWHHYOYVRVGJJY-QMMMGPOBSA-N 4-fluoro-L-phenylalanine Chemical compound OC(=O)[C@@H](N)CC1=CC=C(F)C=C1 XWHHYOYVRVGJJY-QMMMGPOBSA-N 0.000 description 1
- ASYBZHICIMVQII-UHFFFAOYSA-N 4-hydroxylysine Chemical compound NCCC(O)CC(N)C(O)=O ASYBZHICIMVQII-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
- 239000013607 AAV vector Substances 0.000 description 1
- 102100040894 Amylo-alpha-1,6-glucosidase Human genes 0.000 description 1
- 235000002198 Annona diversifolia Nutrition 0.000 description 1
- 208000029402 Bulbospinal muscular atrophy Diseases 0.000 description 1
- 208000015374 Central core disease Diseases 0.000 description 1
- 208000010693 Charcot-Marie-Tooth Disease Diseases 0.000 description 1
- 201000006868 Charcot-Marie-Tooth disease type 3 Diseases 0.000 description 1
- 102100022641 Coagulation factor IX Human genes 0.000 description 1
- 102100023804 Coagulation factor VII Human genes 0.000 description 1
- 108020004635 Complementary DNA Proteins 0.000 description 1
- 108020004394 Complementary RNA Proteins 0.000 description 1
- 206010010356 Congenital anomaly Diseases 0.000 description 1
- 150000008574 D-amino acids Chemical class 0.000 description 1
- 238000001712 DNA sequencing Methods 0.000 description 1
- BWGNESOTFCXPMA-UHFFFAOYSA-N Dihydrogen disulfide Chemical compound SS BWGNESOTFCXPMA-UHFFFAOYSA-N 0.000 description 1
- 101100075747 Drosophila melanogaster Lztr1 gene Proteins 0.000 description 1
- 238000002965 ELISA Methods 0.000 description 1
- 102100034239 Emerin Human genes 0.000 description 1
- 201000009344 Emery-Dreifuss muscular dystrophy Diseases 0.000 description 1
- 208000037149 Facioscapulohumeral dystrophy Diseases 0.000 description 1
- 108010076282 Factor IX Proteins 0.000 description 1
- 108010023321 Factor VII Proteins 0.000 description 1
- 108010054218 Factor VIII Proteins 0.000 description 1
- 102000001690 Factor VIII Human genes 0.000 description 1
- 208000024412 Friedreich ataxia Diseases 0.000 description 1
- WHUUTDBJXJRKMK-UHFFFAOYSA-N Glutamic acid Natural products OC(=O)C(N)CCC(O)=O WHUUTDBJXJRKMK-UHFFFAOYSA-N 0.000 description 1
- 239000004471 Glycine Substances 0.000 description 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 description 1
- 208000032008 Glycogen storage disease due to glycogen debranching enzyme deficiency Diseases 0.000 description 1
- 206010053250 Glycogen storage disease type III Diseases 0.000 description 1
- 208000006411 Hereditary Sensory and Motor Neuropathy Diseases 0.000 description 1
- 206010020850 Hyperthyroidism Diseases 0.000 description 1
- 102000017727 Immunoglobulin Variable Region Human genes 0.000 description 1
- 108010067060 Immunoglobulin Variable Region Proteins 0.000 description 1
- 208000025814 Inflammatory myopathy with abundant macrophages Diseases 0.000 description 1
- 208000027747 Kennedy disease Diseases 0.000 description 1
- 150000008575 L-amino acids Chemical class 0.000 description 1
- WHUUTDBJXJRKMK-VKHMYHEASA-N L-glutamic acid Chemical compound OC(=O)[C@@H](N)CCC(O)=O WHUUTDBJXJRKMK-VKHMYHEASA-N 0.000 description 1
- JTTHKOPSMAVJFE-VIFPVBQESA-N L-homophenylalanine Chemical compound OC(=O)[C@@H](N)CCC1=CC=CC=C1 JTTHKOPSMAVJFE-VIFPVBQESA-N 0.000 description 1
- 102000003855 L-lactate dehydrogenase Human genes 0.000 description 1
- 108700023483 L-lactate dehydrogenases Proteins 0.000 description 1
- ROHFNLRQFUQHCH-YFKPBYRVSA-N L-leucine Chemical compound CC(C)C[C@H](N)C(O)=O ROHFNLRQFUQHCH-YFKPBYRVSA-N 0.000 description 1
- KDXKERNSBIXSRK-YFKPBYRVSA-N L-lysine Chemical compound NCCCC[C@H](N)C(O)=O KDXKERNSBIXSRK-YFKPBYRVSA-N 0.000 description 1
- FFEARJCKVFRZRR-BYPYZUCNSA-N L-methionine Chemical compound CSCC[C@H](N)C(O)=O FFEARJCKVFRZRR-BYPYZUCNSA-N 0.000 description 1
- LRQKBLKVPFOOQJ-YFKPBYRVSA-N L-norleucine Chemical compound CCCC[C@H]([NH3+])C([O-])=O LRQKBLKVPFOOQJ-YFKPBYRVSA-N 0.000 description 1
- COLNVLDHVKWLRT-QMMMGPOBSA-N L-phenylalanine Chemical compound OC(=O)[C@@H](N)CC1=CC=CC=C1 COLNVLDHVKWLRT-QMMMGPOBSA-N 0.000 description 1
- 241000282842 Lama glama Species 0.000 description 1
- 201000010743 Lambert-Eaton myasthenic syndrome Diseases 0.000 description 1
- 102100022745 Laminin subunit alpha-2 Human genes 0.000 description 1
- ROHFNLRQFUQHCH-UHFFFAOYSA-N Leucine Natural products CC(C)CC(N)C(O)=O ROHFNLRQFUQHCH-UHFFFAOYSA-N 0.000 description 1
- 201000009342 Limb-girdle muscular dystrophy Diseases 0.000 description 1
- 239000004472 Lysine Substances 0.000 description 1
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 description 1
- 201000002169 Mitochondrial myopathy Diseases 0.000 description 1
- 241001529936 Murinae Species 0.000 description 1
- 206010028424 Myasthenic syndrome Diseases 0.000 description 1
- 208000012905 Myotonic disease Diseases 0.000 description 1
- 108091061960 Naked DNA Proteins 0.000 description 1
- 201000009110 Oculopharyngeal muscular dystrophy Diseases 0.000 description 1
- 102000001105 Phosphofructokinases Human genes 0.000 description 1
- 108010069341 Phosphofructokinases Proteins 0.000 description 1
- 102000011755 Phosphoglycerate Kinase Human genes 0.000 description 1
- 108700023219 Phosphoglycerate kinases Proteins 0.000 description 1
- 102000009097 Phosphorylases Human genes 0.000 description 1
- 108010073135 Phosphorylases Proteins 0.000 description 1
- RVGRUAULSDPKGF-UHFFFAOYSA-N Poloxamer Chemical compound C1CO1.CC1CO1 RVGRUAULSDPKGF-UHFFFAOYSA-N 0.000 description 1
- 239000002202 Polyethylene glycol Substances 0.000 description 1
- 229920002873 Polyethylenimine Polymers 0.000 description 1
- 208000032225 Proximal spinal muscular atrophy type 1 Diseases 0.000 description 1
- ODHCTXKNWHHXJC-GSVOUGTGSA-N Pyroglutamic acid Natural products OC(=O)[C@H]1CCC(=O)N1 ODHCTXKNWHHXJC-GSVOUGTGSA-N 0.000 description 1
- 238000010240 RT-PCR analysis Methods 0.000 description 1
- 108020004511 Recombinant DNA Proteins 0.000 description 1
- 208000003954 Spinal Muscular Atrophies of Childhood Diseases 0.000 description 1
- 208000035954 Thomsen and Becker disease Diseases 0.000 description 1
- 108090000992 Transferases Proteins 0.000 description 1
- 102000004357 Transferases Human genes 0.000 description 1
- 108091026823 U7 small nuclear RNA Proteins 0.000 description 1
- ODHCTXKNWHHXJC-UHFFFAOYSA-N acide pyroglutamique Natural products OC(=O)C1CCC(=O)N1 ODHCTXKNWHHXJC-UHFFFAOYSA-N 0.000 description 1
- 150000007513 acids Chemical class 0.000 description 1
- 108010056760 agalsidase beta Proteins 0.000 description 1
- 102000016679 alpha-Glucosidases Human genes 0.000 description 1
- 108010028144 alpha-Glucosidases Proteins 0.000 description 1
- 150000001408 amides Chemical class 0.000 description 1
- 125000003277 amino group Chemical group 0.000 description 1
- 125000002344 aminooxy group Chemical group [H]N([H])O[*] 0.000 description 1
- 108010006759 amylo-1,6-glucosidase Proteins 0.000 description 1
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 description 1
- 238000004458 analytical method Methods 0.000 description 1
- 239000000074 antisense oligonucleotide Substances 0.000 description 1
- 238000012230 antisense oligonucleotides Methods 0.000 description 1
- 239000003443 antiviral agent Substances 0.000 description 1
- 235000009697 arginine Nutrition 0.000 description 1
- 150000001484 arginines Chemical class 0.000 description 1
- 208000031375 autosomal dominant myotonia congenita Diseases 0.000 description 1
- 230000001580 bacterial effect Effects 0.000 description 1
- 230000003115 biocidal effect Effects 0.000 description 1
- 230000004071 biological effect Effects 0.000 description 1
- 230000000903 blocking effect Effects 0.000 description 1
- 238000006664 bond formation reaction Methods 0.000 description 1
- 238000010804 cDNA synthesis Methods 0.000 description 1
- 244000309466 calf Species 0.000 description 1
- 229910052799 carbon Inorganic materials 0.000 description 1
- 125000004432 carbon atom Chemical group C* 0.000 description 1
- 150000003857 carboxamides Chemical class 0.000 description 1
- 150000001732 carboxylic acid derivatives Chemical class 0.000 description 1
- 125000002843 carboxylic acid group Chemical group 0.000 description 1
- 229960004203 carnitine Drugs 0.000 description 1
- 125000002091 cationic group Chemical group 0.000 description 1
- 210000003855 cell nucleus Anatomy 0.000 description 1
- 201000007303 central core myopathy Diseases 0.000 description 1
- 229940049197 cerezyme Drugs 0.000 description 1
- 239000002299 complementary DNA Substances 0.000 description 1
- 239000003184 complementary RNA Substances 0.000 description 1
- 201000006815 congenital muscular dystrophy Diseases 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
- 210000000805 cytoplasm Anatomy 0.000 description 1
- 230000001086 cytosolic effect Effects 0.000 description 1
- 239000000824 cytostatic agent Substances 0.000 description 1
- 230000007812 deficiency Effects 0.000 description 1
- 201000001981 dermatomyositis Diseases 0.000 description 1
- 238000001514 detection method Methods 0.000 description 1
- 201000009338 distal myopathy Diseases 0.000 description 1
- 239000003937 drug carrier Substances 0.000 description 1
- 230000009881 electrostatic interaction Effects 0.000 description 1
- 201000010048 endomyocardial fibrosis Diseases 0.000 description 1
- 238000005516 engineering process Methods 0.000 description 1
- 229940125532 enzyme inhibitor Drugs 0.000 description 1
- 239000002532 enzyme inhibitor Substances 0.000 description 1
- 150000002148 esters Chemical class 0.000 description 1
- 239000013604 expression vector Substances 0.000 description 1
- 229940014516 fabrazyme Drugs 0.000 description 1
- 208000008570 facioscapulohumeral muscular dystrophy Diseases 0.000 description 1
- 229960004222 factor ix Drugs 0.000 description 1
- 229940012413 factor vii Drugs 0.000 description 1
- 229960000301 factor viii Drugs 0.000 description 1
- 239000000835 fiber Substances 0.000 description 1
- 210000002950 fibroblast Anatomy 0.000 description 1
- 239000012634 fragment Substances 0.000 description 1
- 238000001476 gene delivery Methods 0.000 description 1
- 230000002068 genetic effect Effects 0.000 description 1
- 235000013922 glutamic acid Nutrition 0.000 description 1
- 239000004220 glutamic acid Substances 0.000 description 1
- 208000007345 glycogen storage disease Diseases 0.000 description 1
- 201000004543 glycogen storage disease III Diseases 0.000 description 1
- 210000004209 hair Anatomy 0.000 description 1
- 125000002887 hydroxy group Chemical group [H]O* 0.000 description 1
- 230000000396 hypokalemic effect Effects 0.000 description 1
- 108010039650 imiglucerase Proteins 0.000 description 1
- 238000012750 in vivo screening Methods 0.000 description 1
- 208000023692 inborn mitochondrial myopathy Diseases 0.000 description 1
- 201000008319 inclusion body myositis Diseases 0.000 description 1
- 230000006698 induction Effects 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 231100001231 less toxic Toxicity 0.000 description 1
- 210000003141 lower extremity Anatomy 0.000 description 1
- 230000007246 mechanism Effects 0.000 description 1
- 229930182817 methionine Natural products 0.000 description 1
- 238000012544 monitoring process Methods 0.000 description 1
- 238000010172 mouse model Methods 0.000 description 1
- 206010028417 myasthenia gravis Diseases 0.000 description 1
- 210000005036 nerve Anatomy 0.000 description 1
- 230000025308 nuclear transport Effects 0.000 description 1
- 210000004940 nucleus Anatomy 0.000 description 1
- 230000030648 nucleus localization Effects 0.000 description 1
- 125000000913 palmityl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 description 1
- 238000004091 panning Methods 0.000 description 1
- 208000027838 paramyotonia congenita of Von Eulenburg Diseases 0.000 description 1
- 239000000863 peptide conjugate Substances 0.000 description 1
- 238000005897 peptide coupling reaction Methods 0.000 description 1
- 208000029308 periodic paralysis Diseases 0.000 description 1
- 238000002823 phage display Methods 0.000 description 1
- 239000008194 pharmaceutical composition Substances 0.000 description 1
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Natural products OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 description 1
- 229960000502 poloxamer Drugs 0.000 description 1
- 229920001983 poloxamer Polymers 0.000 description 1
- 229920000729 poly(L-lysine) polymer Polymers 0.000 description 1
- 229920001223 polyethylene glycol Polymers 0.000 description 1
- 208000005987 polymyositis Diseases 0.000 description 1
- 230000008569 process Effects 0.000 description 1
- 238000011160 research Methods 0.000 description 1
- 230000029058 respiratory gaseous exchange Effects 0.000 description 1
- 230000035945 sensitivity Effects 0.000 description 1
- 210000002966 serum Anatomy 0.000 description 1
- 125000006850 spacer group Chemical group 0.000 description 1
- DFVFTMTWCUHJBL-BQBZGAKWSA-N statine Chemical class CC(C)C[C@H](N)[C@@H](O)CC(O)=O DFVFTMTWCUHJBL-BQBZGAKWSA-N 0.000 description 1
- 238000007920 subcutaneous administration Methods 0.000 description 1
- 238000010254 subcutaneous injection Methods 0.000 description 1
- 239000007929 subcutaneous injection Substances 0.000 description 1
- 238000006467 substitution reaction Methods 0.000 description 1
- 229940124530 sulfonamide Drugs 0.000 description 1
- 150000003456 sulfonamides Chemical class 0.000 description 1
- 208000011580 syndromic disease Diseases 0.000 description 1
- 238000003786 synthesis reaction Methods 0.000 description 1
- 230000009885 systemic effect Effects 0.000 description 1
- 125000003396 thiol group Chemical group [H]S* 0.000 description 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 description 1
- 231100000331 toxic Toxicity 0.000 description 1
- 230000002588 toxic effect Effects 0.000 description 1
- 238000013518 transcription Methods 0.000 description 1
- 230000035897 transcription Effects 0.000 description 1
- 230000009261 transgenic effect Effects 0.000 description 1
- 230000014616 translation Effects 0.000 description 1
- 238000002255 vaccination Methods 0.000 description 1
- 108700026220 vif Genes Proteins 0.000 description 1
- 238000005406 washing Methods 0.000 description 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K49/00—Preparations for testing in vivo
- A61K49/001—Preparation for luminescence or biological staining
- A61K49/0013—Luminescence
- A61K49/0017—Fluorescence in vivo
- A61K49/005—Fluorescence in vivo characterised by the carrier molecule carrying the fluorescent agent
- A61K49/0056—Peptides, proteins, polyamino acids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K49/00—Preparations for testing in vivo
- A61K49/001—Preparation for luminescence or biological staining
- A61K49/0013—Luminescence
- A61K49/0017—Fluorescence in vivo
- A61K49/0019—Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules
- A61K49/0021—Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules the fluorescent group being a small organic molecule
- A61K49/0032—Methine dyes, e.g. cyanine dyes
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K49/00—Preparations for testing in vivo
- A61K49/001—Preparation for luminescence or biological staining
- A61K49/0013—Luminescence
- A61K49/0017—Fluorescence in vivo
- A61K49/0019—Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules
- A61K49/0045—Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules the fluorescent agent being a peptide or protein used for imaging or diagnosis in vivo
- A61K49/0047—Green fluorescent protein [GFP]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/04—Anorexiants; Antiobesity agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P5/00—Drugs for disorders of the endocrine system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- Public Health (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Diabetes (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Physical Education & Sports Medicine (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Endocrinology (AREA)
- Pathology (AREA)
- Radiology & Medical Imaging (AREA)
- Heart & Thoracic Surgery (AREA)
- Child & Adolescent Psychology (AREA)
- Emergency Medicine (AREA)
- Cardiology (AREA)
- Pain & Pain Management (AREA)
- Peptides Or Proteins (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP07112323 | 2007-07-12 | ||
| EP07112323.6 | 2007-07-12 | ||
| CA2693048A CA2693048C (en) | 2007-07-12 | 2008-07-14 | Molecules for targeting compounds to various selected organs or tissues |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2693048A Division CA2693048C (en) | 2007-07-12 | 2008-07-14 | Molecules for targeting compounds to various selected organs or tissues |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CA2942716A1 CA2942716A1 (en) | 2009-01-15 |
| CA2942716C true CA2942716C (en) | 2019-07-09 |
Family
ID=39471746
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2693048A Expired - Fee Related CA2693048C (en) | 2007-07-12 | 2008-07-14 | Molecules for targeting compounds to various selected organs or tissues |
| CA2942716A Expired - Fee Related CA2942716C (en) | 2007-07-12 | 2008-07-14 | Molecules for targeting compounds to various selected organs or tissues |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2693048A Expired - Fee Related CA2693048C (en) | 2007-07-12 | 2008-07-14 | Molecules for targeting compounds to various selected organs or tissues |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US8268962B2 (enExample) |
| EP (2) | EP2505211B1 (enExample) |
| JP (1) | JP5706157B2 (enExample) |
| CN (2) | CN101815535B (enExample) |
| AU (1) | AU2008273096B2 (enExample) |
| CA (2) | CA2693048C (enExample) |
| CY (1) | CY1117713T1 (enExample) |
| DK (1) | DK2167136T3 (enExample) |
| IL (2) | IL203273A (enExample) |
| NZ (1) | NZ582521A (enExample) |
| WO (1) | WO2009008727A2 (enExample) |
Families Citing this family (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2003225410A1 (en) | 2003-03-21 | 2004-10-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| US8168181B2 (en) | 2006-02-13 | 2012-05-01 | Alethia Biotherapeutics, Inc. | Methods of impairing osteoclast differentiation using antibodies that bind siglec-15 |
| DK1994155T4 (da) | 2006-02-13 | 2022-07-25 | Daiichi Sankyo Co Ltd | Polynukleotid- og polypeptidsekvenser involveret i fremgangsmåden med knogleremodellering |
| DK2049664T3 (da) | 2006-08-11 | 2012-01-02 | Prosensa Technologies Bv | Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser |
| CN101896186A (zh) | 2007-10-26 | 2010-11-24 | 莱顿教学医院 | 对抗肌肉病症的方式和方法 |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| CA2759899A1 (en) | 2009-04-24 | 2010-10-28 | Prosensa Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
| CN102048694B (zh) * | 2009-11-06 | 2013-03-13 | 复旦大学 | 一种多肽修饰的肝肿瘤靶向纳米给药系统及其制备方法 |
| JP2014513946A (ja) * | 2011-04-22 | 2014-06-19 | プロセンサ テクノロジーズ ビー．ブイ． | 筋強直性ジストロフィー１型（ｄｍ１）などのヒト遺伝性疾患の治療、遅延及び／又は予防のための新規の化合物 |
| WO2013103614A1 (en) * | 2011-12-30 | 2013-07-11 | Stc.Unm | Crlf-2 binding peptides, protocells and viral-like particles useful in the treatment of cancer, including acute lymphoblastic leukemia (all) |
| BR112014018427B1 (pt) | 2012-01-27 | 2021-11-03 | Biomarin Technologies B.V. | Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker |
| EP2841578B1 (en) | 2012-04-23 | 2017-06-07 | BioMarin Technologies B.V. | RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders |
| US9493562B2 (en) | 2012-07-19 | 2016-11-15 | Alethia Biotherapeutics Inc. | Anti-Siglec-15 antibodies |
| CN103705465B (zh) * | 2012-10-09 | 2016-01-13 | 复旦大学 | 一种微酸环境靶向多肽修饰的肿瘤靶向纳米给药系统及其制备方法 |
| HK1231425A1 (zh) | 2014-01-13 | 2017-12-22 | Berg Llc | 烯醇酶1(eno1)組合物及其用途 |
| CN107073065B (zh) * | 2014-08-22 | 2021-07-02 | 密歇根大学董事会 | 用于发育异常、早期癌症和癌症的检测和靶向的肽试剂和方法 |
| CN104800858B (zh) * | 2015-04-27 | 2017-11-21 | 中国医学科学院基础医学研究所 | Hsp90抑制肽偶联物及其在肿瘤治疗中的应用 |
| WO2016205397A2 (en) * | 2015-06-15 | 2016-12-22 | Klibanov Alexander L | Target-specific delivery of therapeutic agents |
| CA2992611A1 (en) * | 2015-07-16 | 2017-01-19 | Berg Llc | Enolase 1 (eno1) compositions and uses thereof |
| US11672866B2 (en) | 2016-01-08 | 2023-06-13 | Paul N. DURFEE | Osteotropic nanoparticles for prevention or treatment of bone metastases |
| WO2017136435A1 (en) | 2016-02-01 | 2017-08-10 | The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health | Compounds for modulating fc-epsilon-ri-beta expression and uses thereof |
| AU2017361784B2 (en) | 2016-11-16 | 2021-07-15 | Academisch Ziekenhuis Leiden | Substances for targeting various selected organs or tissues |
| US11344629B2 (en) | 2017-03-01 | 2022-05-31 | Charles Jeffrey Brinker | Active targeting of cells by monosized protocells |
| GB2563875B (en) * | 2017-06-28 | 2020-08-19 | Sutura Therapeutics Ltd | Improvements in drug delivery |
| WO2020068798A1 (en) | 2018-09-24 | 2020-04-02 | Guo Jimin | Living mammalian cells modified with functional modular nanoparticles |
| JP7661218B2 (ja) | 2018-11-02 | 2025-04-14 | バイオマリン テクノロジーズ ベー．フェー． | ジストロフィンエクソンスキッピングのための二重特異性アンチセンスオリゴヌクレオチド |
| WO2020176716A1 (en) | 2019-02-28 | 2020-09-03 | Brinker C Jeffrey | Modular metal–organic polyhedra superassembly compositions |
| WO2021113290A1 (en) | 2019-12-03 | 2021-06-10 | Alamar Biosciences, Inc. | Nucleic acid linked immune-sandwich assay (nulisa) |
Family Cites Families (104)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5034506A (en) * | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5541308A (en) * | 1986-11-24 | 1996-07-30 | Gen-Probe Incorporated | Nucleic acid probes for detection and/or quantitation of non-viral organisms |
| US5766847A (en) * | 1988-10-11 | 1998-06-16 | Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. | Process for analyzing length polymorphisms in DNA regions |
| DE3834636A1 (de) | 1988-10-11 | 1990-04-19 | Max Planck Gesellschaft | Verfahren zur analyse von laengenpolymorphismen in dna-bereichen |
| US6867195B1 (en) * | 1989-03-21 | 2005-03-15 | Vical Incorporated | Lipid-mediated polynucleotide administration to reduce likelihood of subject's becoming infected |
| US5608046A (en) * | 1990-07-27 | 1997-03-04 | Isis Pharmaceuticals, Inc. | Conjugated 4'-desmethyl nucleoside analog compounds |
| FR2675803B1 (fr) * | 1991-04-25 | 1996-09-06 | Genset Sa | Oligonucleotides fermes, antisens et sens et leurs applications. |
| CA2082411A1 (en) * | 1991-06-28 | 1992-12-29 | Robert D. Rosenberg | Localized oligonucleotide therapy |
| WO1993001286A2 (en) | 1991-06-28 | 1993-01-21 | Massachusetts Institute Of Technology | Localized oligonucleotide therapy |
| US6200747B1 (en) * | 1992-01-28 | 2001-03-13 | North Shore University Hospital Research Corp. | Method and kits for detection of fragile X specific, GC-rich DNA sequences |
| US5869252A (en) * | 1992-03-31 | 1999-02-09 | Abbott Laboratories | Method of multiplex ligase chain reaction |
| US5418139A (en) * | 1993-02-10 | 1995-05-23 | University Of Iowa Research Foundation | Method for screening for cardiomyopathy |
| CA2116280A1 (en) | 1993-03-05 | 1994-09-06 | Marcy E. Macdonald | Huntingtin dna, protein and uses thereof |
| US5741645A (en) * | 1993-06-29 | 1998-04-21 | Regents Of The University Of Minnesota | Gene sequence for spinocerebellar ataxia type 1 and method for diagnosis |
| US5627263A (en) * | 1993-11-24 | 1997-05-06 | La Jolla Cancer Research Foundation | Integrin-binding peptides |
| DE4342605A1 (de) | 1993-12-14 | 1995-06-22 | Buna Gmbh | Funktionalisierte Olefinhomo- und -copolymere |
| US5962332A (en) * | 1994-03-17 | 1999-10-05 | University Of Massachusetts | Detection of trinucleotide repeats by in situ hybridization |
| WO1995030774A1 (en) | 1994-05-05 | 1995-11-16 | Beckman Instruments, Inc. | Oligonucleotide repeat arrays |
| US5968909A (en) * | 1995-08-04 | 1999-10-19 | Hybridon, Inc. | Method of modulating gene expression with reduced immunostimulatory response |
| US5854223A (en) | 1995-10-06 | 1998-12-29 | The Trustees Of Columbia University In The City Of New York | S-DC28 as an antirestenosis agent after balloon injury |
| US6300060B1 (en) * | 1995-11-09 | 2001-10-09 | Dana-Farber Cancer Institute, Inc. | Method for predicting the risk of prostate cancer morbidity and mortality |
| ATE267207T1 (de) | 1996-02-14 | 2004-06-15 | Isis Pharmaceuticals Inc | Kohlenhydratmodifizierte luckenhafte oligonukleotide |
| WO1998003679A1 (fr) * | 1996-07-18 | 1998-01-29 | Srl, Inc. | Procede de diagnostic de l'ataxie spino-cerebelleuse de type 2 et amorces destinees a ce procede |
| WO1998018920A1 (fr) | 1996-10-30 | 1998-05-07 | Srl, Inc. | Fragments d'adnc du gene responsable de l'ataxie spino-cerebelleuse de type 2 |
| US5853995A (en) | 1997-01-07 | 1998-12-29 | Research Development Foundation | Large scale genotyping of diseases and a diagnostic test for spinocerebellar ataxia type 6 |
| US20020137890A1 (en) * | 1997-03-31 | 2002-09-26 | Genentech, Inc. | Secreted and transmembrane polypeptides and nucleic acids encoding the same |
| WO1998049345A1 (en) | 1997-04-29 | 1998-11-05 | Trustees Of Boston University | Methods and compositions for targeted dna differential display |
| US6329501B1 (en) * | 1997-05-29 | 2001-12-11 | Auburn University | Methods and compositions for targeting compounds to muscle |
| US6280938B1 (en) * | 1997-08-19 | 2001-08-28 | Regents Of The University Of Minnesota | SCA7 gene and method of use |
| US6514755B1 (en) * | 1998-08-18 | 2003-02-04 | Regents Of The University Of Minnesota | SCA7 gene and methods of use |
| US6794499B2 (en) * | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| US6130207A (en) * | 1997-11-05 | 2000-10-10 | South Alabama Medical Science Foundation | Cell-specific molecule and method for importing DNA into a nucleus |
| JP3012923B2 (ja) * | 1998-01-26 | 2000-02-28 | 新潟大学長 | Ｃａｇリピート病の治療薬 |
| KR100280219B1 (ko) * | 1998-02-26 | 2001-04-02 | 이수빈 | 삼핵산 반복 서열을 이용한 신경정신 질환의 진단 방법 및 진단 시약 |
| US6322978B1 (en) * | 1998-04-20 | 2001-11-27 | Joslin Diabetes Center, Inc. | Repeat polymorphism in the frataxin gene and uses therefore |
| US6924355B2 (en) * | 1998-09-01 | 2005-08-02 | Genentech, Inc. | PRO1343 polypeptides |
| AU6059099A (en) * | 1998-09-25 | 2000-04-17 | Children's Medical Center Corporation | Short peptides which selectively modulate the activity of protein kinases |
| US6172216B1 (en) * | 1998-10-07 | 2001-01-09 | Isis Pharmaceuticals Inc. | Antisense modulation of BCL-X expression |
| US6399575B1 (en) * | 1998-11-10 | 2002-06-04 | Auburn University | Methods and compositions for targeting compounds to the central nervous system |
| US6133031A (en) * | 1999-08-19 | 2000-10-17 | Isis Pharmaceuticals Inc. | Antisense inhibition of focal adhesion kinase expression |
| US20040226056A1 (en) * | 1998-12-22 | 2004-11-11 | Myriad Genetics, Incorporated | Compositions and methods for treating neurological disorders and diseases |
| US20020049173A1 (en) * | 1999-03-26 | 2002-04-25 | Bennett C. Frank | Alteration of cellular behavior by antisense modulation of mRNA processing |
| US6379698B1 (en) * | 1999-04-06 | 2002-04-30 | Isis Pharmaceuticals, Inc. | Fusogenic lipids and vesicles |
| JP2000325085A (ja) * | 1999-05-21 | 2000-11-28 | Masafumi Matsuo | デュシェンヌ型筋ジストロフィー治療剤 |
| US20030236214A1 (en) * | 1999-06-09 | 2003-12-25 | Wolff Jon A. | Charge reversal of polyion complexes and treatment of peripheral occlusive disease |
| AU5625500A (en) * | 1999-06-18 | 2001-01-09 | Emory University | Huntington disease cellular model: stably transfected pc12 cells expressing mutant huntingtin |
| EP1133993A1 (en) | 2000-03-10 | 2001-09-19 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | Substances for the treatment of spinal muscular atrophy |
| WO2001079283A1 (en) | 2000-04-13 | 2001-10-25 | University Of British Columbia | Modulating cell survival by modulating huntingtin function |
| US6653467B1 (en) | 2000-04-26 | 2003-11-25 | Jcr Pharmaceutical Co., Ltd. | Medicament for treatment of Duchenne muscular dystrophy |
| CA2407309C (en) | 2000-04-28 | 2011-08-02 | Xiao Xiao | Dna sequences encoding dystrophin minigenes and methods of use thereof |
| AU2001259706A1 (en) * | 2000-05-09 | 2001-11-20 | Reliable Biopharmaceutical, Inc. | Polymeric compounds useful as prodrugs |
| EP1294866B1 (en) * | 2000-05-31 | 2010-04-14 | Serono Genetics Institute S.A. | Use of acrp30 globular head to promote increases in muscle mass and muscle differentiation |
| US20030124523A1 (en) * | 2000-06-22 | 2003-07-03 | Asselbergs Fredericus Alphonsus Maria | Organic compounds |
| US6794192B2 (en) * | 2000-06-29 | 2004-09-21 | Pfizer Inc. | Target |
| RU2165149C1 (ru) | 2000-07-03 | 2001-04-20 | Шапошников Валерий Геннадьевич | Система формования и упаковки изделий из сахарной ваты |
| US6727355B2 (en) | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition for treatment of Duchenne muscular dystrophy |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| US6623927B1 (en) * | 2000-11-08 | 2003-09-23 | Council Of Scientific And Industrial Research | Method of detection of allelic variants of SCA2 gene |
| US6743893B2 (en) * | 2000-11-30 | 2004-06-01 | The Uab Research Foundation | Receptor-mediated uptake of peptides that bind the human transferrin receptor |
| TW200526779A (en) * | 2001-02-08 | 2005-08-16 | Wyeth Corp | Modified and stabilized GDF propeptides and uses thereof |
| US6902896B2 (en) * | 2001-05-11 | 2005-06-07 | Regents Of The University Of Minnesota | Intron associated with myotonic dystrophy type 2 and methods of use |
| US20050277133A1 (en) * | 2001-05-18 | 2005-12-15 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
| EP1627061B1 (en) | 2001-05-18 | 2009-08-12 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA) |
| AU2002326589B2 (en) * | 2001-08-07 | 2008-06-05 | University Of Delaware | Compositions and methods for the prevention and treatment of Huntington's disease |
| US20060074034A1 (en) * | 2001-09-17 | 2006-04-06 | Collins Douglas A | Cobalamin mediated delivery of nucleic acids, analogs and derivatives thereof |
| KR20030035047A (ko) | 2001-10-29 | 2003-05-09 | (주)바이오코돈 | Bmp-4 유전자 발현을 이용한 편평태선 질환의 치료 및진단방법 |
| WO2003037172A2 (en) * | 2001-11-01 | 2003-05-08 | Gpc Biotech Inc. | Endothelial-cell binding peptides for diagnosis and therapy |
| US20030134790A1 (en) * | 2002-01-11 | 2003-07-17 | University Of Medicine And Dentistry Of New Jersey | Bone Morphogenetic Protein-2 And Bone Morphogenetic Protein-4 In The Treatment And Diagnosis Of Cancer |
| WO2003069330A1 (en) * | 2002-02-11 | 2003-08-21 | The Trustees Of Columbia University In The City Of New York | System and method for identifying proteins involved in force-initiated signal transduction |
| US20050096284A1 (en) * | 2002-02-20 | 2005-05-05 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
| US7771727B2 (en) | 2002-03-01 | 2010-08-10 | The Administrators Of The Tulane Educational Fund | Conjugates of therapeutic or cytotoxic agents and biologically active peptides |
| US20040101852A1 (en) * | 2002-11-21 | 2004-05-27 | Isis Pharmaceuticals Inc. | Modulation of CGG triplet repeat binding protein 1 expression |
| ITRM20020253A1 (it) | 2002-05-08 | 2003-11-10 | Univ Roma | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
| EP1380644A1 (en) | 2002-07-08 | 2004-01-14 | Kylix B.V. | The use of specified TCF target genes to identify drugs for the treatment of cancer, in particular colorectal cancer, in which TCF/beta-catenin/WNT signalling plays a central role |
| US7589059B2 (en) | 2002-07-26 | 2009-09-15 | Roche Madison Inc. | Delivery of molecules and complexes to mammalian cells in vivo |
| US20050255086A1 (en) | 2002-08-05 | 2005-11-17 | Davidson Beverly L | Nucleic acid silencing of Huntington's Disease gene |
| EP1529105B1 (en) | 2002-08-12 | 2012-10-03 | Société de commercialisation des produits de la recherche appliquée SOCPRA Sciences Santé et Humaines s.e.c. | Methods to reprogram splice site selection in pre-messenger rnas |
| GB0219143D0 (en) | 2002-08-16 | 2002-09-25 | Univ Leicester | Modified tailed oligonucleotides |
| AU2002342850A1 (en) * | 2002-10-23 | 2004-05-13 | Centre For Research And Technology Hellas/Institute Of Agrobiotechnology In.A | Prion protein-binding peptide sequences |
| US7892793B2 (en) | 2002-11-04 | 2011-02-22 | University Of Massachusetts | Allele-specific RNA interference |
| EP2213738B1 (en) * | 2002-11-14 | 2012-10-10 | Dharmacon, Inc. | siRNA molecules targeting Bcl-2 |
| CA3001404C (en) * | 2002-11-25 | 2020-07-28 | Masafumi Matsuo | Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors |
| GB0228079D0 (en) | 2002-12-02 | 2003-01-08 | Laxdale Ltd | Huntington's Disease |
| AU2003225410A1 (en) | 2003-03-21 | 2004-10-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| US7589189B2 (en) | 2003-05-14 | 2009-09-15 | Japan Science And Technology Agency | Inhibition of the expression of huntingtin gene |
| JP5110877B2 (ja) | 2003-06-02 | 2012-12-26 | ワイス・エルエルシー | 神経筋障害を処置するための、コルチコステロイドと組み合わせたミオスタチン（ｇｄｆ８）インヒビターの使用 |
| ATE387217T1 (de) | 2003-07-11 | 2008-03-15 | Lbr Medbiotech B V | Mannose-6-phosphat rezeptor vermittelter gentransfer zu muskelzellen |
| US20050054752A1 (en) * | 2003-09-08 | 2005-03-10 | O'brien John P. | Peptide-based diblock and triblock dispersants and diblock polymers |
| WO2005033134A2 (en) * | 2003-09-30 | 2005-04-14 | Regeneron Pharmaceuticals, Inc. | Secreted protein therapeutics and uses thereof |
| EP1680439A2 (en) * | 2003-10-14 | 2006-07-19 | Kernel Biopharma Inc. | Dual phase-pna conjugates for the delivery of pna through the blood brain barrier |
| US20050191636A1 (en) | 2004-03-01 | 2005-09-01 | Biocept, Inc. | Detection of STRP, such as fragile X syndrome |
| WO2005105995A2 (en) | 2004-04-14 | 2005-11-10 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| CA2566286A1 (en) | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same |
| PL1766010T3 (pl) | 2004-06-28 | 2011-07-29 | Univ Western Australia | Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania |
| EP1618881A1 (en) | 2004-07-20 | 2006-01-25 | Santhera Pharmaceuticals (Schweiz) GmbH | Use of non-glucocorticoid steroids for the treatment of muscular dystrophy |
| ITRM20040568A1 (it) | 2004-11-18 | 2005-02-18 | Uni Degli Studi Di Roma Tor Vergata | Uso della tecnica "phage display" per l'identificazione di peptidi con capacita' di legame a cellule staminali/progenitore, peptidi cosi' ottenuti e loro usi. |
| US20060148740A1 (en) * | 2005-01-05 | 2006-07-06 | Prosensa B.V. | Mannose-6-phosphate receptor mediated gene transfer into muscle cells |
| CA2596588C (en) | 2005-01-31 | 2017-06-27 | University Of Iowa Research Foundation | Nucleic acid silencing of huntington's disease gene |
| WO2006121960A2 (en) | 2005-05-06 | 2006-11-16 | Medtronic, Inc. | Methods and sequences to suppress primate huntington gene expression |
| CN100393320C (zh) * | 2005-06-24 | 2008-06-11 | 奥林格斯技术有限公司 | 治疗肌肉萎缩的寡核苷酸药物 |
| ATE522626T1 (de) | 2005-06-28 | 2011-09-15 | Medtronic Inc | Verfahren und nukleotid-sequenzen, die bevorzugt die expression eines mutierten huntingtin-genes unterdrücken |
| US7906617B2 (en) * | 2005-12-15 | 2011-03-15 | E. I. Du Pont De Nemours And Company | Polyethylene binding peptides and methods of use |
| US20100248239A1 (en) * | 2009-03-24 | 2010-09-30 | Mayo Foundation For Medical Education And Research | Methods and materials for detecting fragile x mutations |
| US20110166081A1 (en) * | 2009-12-03 | 2011-07-07 | University Of Iowa Research Foundation | Alpha-dystroglycan as a Protein Therapeutic |
-
2008
- 2008-07-14 AU AU2008273096A patent/AU2008273096B2/en not_active Ceased
- 2008-07-14 CA CA2693048A patent/CA2693048C/en not_active Expired - Fee Related
- 2008-07-14 DK DK08779023.4T patent/DK2167136T3/en active
- 2008-07-14 EP EP12174179.7A patent/EP2505211B1/en active Active
- 2008-07-14 JP JP2010515992A patent/JP5706157B2/ja not_active Expired - Fee Related
- 2008-07-14 WO PCT/NL2008/050475 patent/WO2009008727A2/en not_active Ceased
- 2008-07-14 CN CN2008801016669A patent/CN101815535B/zh not_active Expired - Fee Related
- 2008-07-14 EP EP08779023.4A patent/EP2167136B1/en active Active
- 2008-07-14 CA CA2942716A patent/CA2942716C/en not_active Expired - Fee Related
- 2008-07-14 NZ NZ582521A patent/NZ582521A/en not_active IP Right Cessation
- 2008-07-14 CN CN2013100912811A patent/CN103212085A/zh active Pending
-
2010
- 2010-01-11 US US12/685,369 patent/US8268962B2/en not_active Expired - Fee Related
- 2010-01-12 IL IL203273A patent/IL203273A/en active IP Right Grant
-
2012
- 2012-07-16 IL IL220979A patent/IL220979A0/en unknown
- 2012-08-07 US US13/568,866 patent/US8519097B2/en active Active
-
2016
- 2016-07-01 CY CY20161100611T patent/CY1117713T1/el unknown
Also Published As
| Publication number | Publication date |
|---|---|
| AU2008273096B2 (en) | 2013-05-02 |
| EP2167136A2 (en) | 2010-03-31 |
| JP2010533171A (ja) | 2010-10-21 |
| CA2942716A1 (en) | 2009-01-15 |
| JP5706157B2 (ja) | 2015-04-22 |
| EP2167136B1 (en) | 2016-04-20 |
| CA2693048C (en) | 2016-10-18 |
| CN103212085A (zh) | 2013-07-24 |
| US8519097B2 (en) | 2013-08-27 |
| US8268962B2 (en) | 2012-09-18 |
| WO2009008727A3 (en) | 2009-08-13 |
| HK1145635A1 (en) | 2011-04-29 |
| US20120322724A1 (en) | 2012-12-20 |
| IL203273A (en) | 2014-04-30 |
| IL220979A0 (en) | 2012-08-30 |
| CN101815535B (zh) | 2013-04-24 |
| WO2009008727A2 (en) | 2009-01-15 |
| AU2008273096A1 (en) | 2009-01-15 |
| DK2167136T3 (en) | 2016-07-25 |
| EP2505211A1 (en) | 2012-10-03 |
| CN101815535A (zh) | 2010-08-25 |
| US20100184948A1 (en) | 2010-07-22 |
| CA2693048A1 (en) | 2009-01-15 |
| NZ582521A (en) | 2011-09-30 |
| CY1117713T1 (el) | 2017-05-17 |
| EP2505211B1 (en) | 2020-04-08 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CA2942716C (en) | Molecules for targeting compounds to various selected organs or tissues | |
| US8609065B2 (en) | Molecules for targeting compounds to various selected organs, tissues or tumor cells | |
| US11459358B2 (en) | Substances for targeting various selected organs or tissues | |
| ES2577712T3 (es) | Moléculas para direccionar compuestos a varios órganos o tejidos seleccionados | |
| HK1176286A (en) | Molecules for targeting compounds to various selected organs or tissues | |
| HK1145635B (en) | Molecules for targeting compounds to various selected organs or tissues |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20160922 |
|
| MKLA | Lapsed |
Effective date: 20220714 |