CA2448120A1 - Muscle-specific expression vectors - Google Patents
Muscle-specific expression vectors Download PDFInfo
- Publication number
- CA2448120A1 CA2448120A1 CA002448120A CA2448120A CA2448120A1 CA 2448120 A1 CA2448120 A1 CA 2448120A1 CA 002448120 A CA002448120 A CA 002448120A CA 2448120 A CA2448120 A CA 2448120A CA 2448120 A1 CA2448120 A1 CA 2448120A1
- Authority
- CA
- Canada
- Prior art keywords
- promoter
- enhancer
- muscle
- mck
- regulatory element
- 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
- 210000003205 muscle Anatomy 0.000 title claims abstract description 58
- 239000013604 expression vector Substances 0.000 title description 13
- 239000003623 enhancer Substances 0.000 claims abstract description 113
- 108010044052 Desmin Proteins 0.000 claims abstract description 75
- 102100036912 Desmin Human genes 0.000 claims abstract description 75
- 210000005045 desmin Anatomy 0.000 claims abstract description 75
- 230000001105 regulatory effect Effects 0.000 claims abstract description 73
- 108010059343 MM Form Creatine Kinase Proteins 0.000 claims abstract description 69
- 230000014509 gene expression Effects 0.000 claims abstract description 58
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims abstract description 30
- 102000013394 Troponin I Human genes 0.000 claims abstract description 24
- 108010065729 Troponin I Proteins 0.000 claims abstract description 24
- 210000000107 myocyte Anatomy 0.000 claims abstract description 19
- 108010085238 Actins Proteins 0.000 claims abstract description 17
- 102000007469 Actins Human genes 0.000 claims abstract description 16
- 210000004027 cell Anatomy 0.000 claims description 53
- 108700019146 Transgenes Proteins 0.000 claims description 45
- 239000013598 vector Substances 0.000 claims description 41
- 241000282414 Homo sapiens Species 0.000 claims description 38
- 108090000623 proteins and genes Proteins 0.000 claims description 38
- 239000013612 plasmid Substances 0.000 claims description 25
- 238000000034 method Methods 0.000 claims description 14
- 241000894007 species Species 0.000 claims description 12
- 241000271566 Aves Species 0.000 claims description 10
- 239000013603 viral vector Substances 0.000 claims description 9
- 241000286209 Phasianidae Species 0.000 claims description 8
- 241000251539 Vertebrata <Metazoa> Species 0.000 claims description 6
- 241001430294 unidentified retrovirus Species 0.000 claims description 6
- 108091026890 Coding region Proteins 0.000 claims description 5
- 241000700605 Viruses Species 0.000 claims description 5
- 241000702421 Dependoparvovirus Species 0.000 claims description 4
- 241000713666 Lentivirus Species 0.000 claims description 4
- 241001529936 Murinae Species 0.000 claims description 4
- 241000701161 unidentified adenovirus Species 0.000 claims description 4
- 210000001236 prokaryotic cell Anatomy 0.000 claims description 3
- 108091006107 transcriptional repressors Proteins 0.000 claims description 3
- 101100499108 Homo sapiens DES gene Proteins 0.000 claims 2
- 210000004962 mammalian cell Anatomy 0.000 claims 2
- 230000002085 persistent effect Effects 0.000 abstract description 2
- 108091000080 Phosphotransferase Proteins 0.000 abstract 1
- CVSVTCORWBXHQV-UHFFFAOYSA-N creatine Chemical compound NC(=[NH2+])N(C)CC([O-])=O CVSVTCORWBXHQV-UHFFFAOYSA-N 0.000 abstract 1
- 102000020233 phosphotransferase Human genes 0.000 abstract 1
- 108020004414 DNA Proteins 0.000 description 32
- 210000001519 tissue Anatomy 0.000 description 29
- 241000699666 Mus <mouse, genus> Species 0.000 description 19
- 238000001890 transfection Methods 0.000 description 16
- 238000001415 gene therapy Methods 0.000 description 13
- 102000004169 proteins and genes Human genes 0.000 description 13
- 238000013518 transcription Methods 0.000 description 12
- 230000035897 transcription Effects 0.000 description 12
- 241000701022 Cytomegalovirus Species 0.000 description 11
- 238000012360 testing method Methods 0.000 description 11
- 238000002347 injection Methods 0.000 description 10
- 239000007924 injection Substances 0.000 description 10
- 210000003098 myoblast Anatomy 0.000 description 10
- 230000003612 virological effect Effects 0.000 description 9
- 241000700159 Rattus Species 0.000 description 8
- 239000003795 chemical substances by application Substances 0.000 description 8
- 108020004707 nucleic acids Proteins 0.000 description 7
- 102000039446 nucleic acids Human genes 0.000 description 7
- 150000007523 nucleic acids Chemical class 0.000 description 7
- 210000002966 serum Anatomy 0.000 description 7
- 210000002027 skeletal muscle Anatomy 0.000 description 7
- 238000011144 upstream manufacturing Methods 0.000 description 7
- 241001465754 Metazoa Species 0.000 description 5
- 241000699670 Mus sp. Species 0.000 description 5
- 108010029485 Protein Isoforms Proteins 0.000 description 5
- 102000001708 Protein Isoforms Human genes 0.000 description 5
- 230000000694 effects Effects 0.000 description 5
- 238000001727 in vivo Methods 0.000 description 5
- 230000002459 sustained effect Effects 0.000 description 5
- 230000001225 therapeutic effect Effects 0.000 description 5
- 238000012546 transfer Methods 0.000 description 5
- 102000002260 Alkaline Phosphatase Human genes 0.000 description 4
- 108020004774 Alkaline Phosphatase Proteins 0.000 description 4
- 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 4
- 210000000663 muscle cell Anatomy 0.000 description 4
- 210000004165 myocardium Anatomy 0.000 description 4
- 239000002773 nucleotide Substances 0.000 description 4
- 125000003729 nucleotide group Chemical group 0.000 description 4
- 239000002953 phosphate buffered saline Substances 0.000 description 4
- 108091033319 polynucleotide Proteins 0.000 description 4
- 102000040430 polynucleotide Human genes 0.000 description 4
- 239000002157 polynucleotide Substances 0.000 description 4
- 239000000047 product Substances 0.000 description 4
- 210000003699 striated muscle Anatomy 0.000 description 4
- 238000012384 transportation and delivery Methods 0.000 description 4
- 108020005029 5' Flanking Region Proteins 0.000 description 3
- 102000004190 Enzymes Human genes 0.000 description 3
- 108090000790 Enzymes Proteins 0.000 description 3
- 101000928044 Homo sapiens Desmin Proteins 0.000 description 3
- 102100024640 Low-density lipoprotein receptor Human genes 0.000 description 3
- 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 description 3
- 108091028043 Nucleic acid sequence Proteins 0.000 description 3
- 108700026226 TATA Box Proteins 0.000 description 3
- 108700009124 Transcription Initiation Site Proteins 0.000 description 3
- 230000008901 benefit Effects 0.000 description 3
- 230000001413 cellular effect Effects 0.000 description 3
- 201000010099 disease Diseases 0.000 description 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 3
- 239000012634 fragment Substances 0.000 description 3
- 230000003834 intracellular effect Effects 0.000 description 3
- 230000001114 myogenic effect Effects 0.000 description 3
- 210000001087 myotubule Anatomy 0.000 description 3
- 230000002688 persistence Effects 0.000 description 3
- 230000008685 targeting Effects 0.000 description 3
- 230000002103 transcriptional effect Effects 0.000 description 3
- 241000894006 Bacteria Species 0.000 description 2
- OYPRJOBELJOOCE-UHFFFAOYSA-N Calcium Chemical compound [Ca] OYPRJOBELJOOCE-UHFFFAOYSA-N 0.000 description 2
- 108010035563 Chloramphenicol O-acetyltransferase Proteins 0.000 description 2
- 102000004420 Creatine Kinase Human genes 0.000 description 2
- 108010042126 Creatine kinase Proteins 0.000 description 2
- 102100022785 Creatine kinase B-type Human genes 0.000 description 2
- 241000287828 Gallus gallus Species 0.000 description 2
- 108700039691 Genetic Promoter Regions Proteins 0.000 description 2
- 241000282412 Homo Species 0.000 description 2
- 208000000563 Hyperlipoproteinemia Type II Diseases 0.000 description 2
- 206010061598 Immunodeficiency Diseases 0.000 description 2
- 208000029462 Immunodeficiency disease Diseases 0.000 description 2
- DRBBFCLWYRJSJZ-UHFFFAOYSA-N N-phosphocreatine Chemical compound OC(=O)CN(C)C(=N)NP(O)(O)=O DRBBFCLWYRJSJZ-UHFFFAOYSA-N 0.000 description 2
- 108091034117 Oligonucleotide Proteins 0.000 description 2
- 108700008625 Reporter Genes Proteins 0.000 description 2
- 108700029229 Transcriptional Regulatory Elements Proteins 0.000 description 2
- 102000004903 Troponin Human genes 0.000 description 2
- 108090001027 Troponin Proteins 0.000 description 2
- 206010045261 Type IIa hyperlipidaemia Diseases 0.000 description 2
- 108010073929 Vascular Endothelial Growth Factor A Proteins 0.000 description 2
- 102000005789 Vascular Endothelial Growth Factors Human genes 0.000 description 2
- 108010019530 Vascular Endothelial Growth Factors Proteins 0.000 description 2
- 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 2
- 230000001580 bacterial effect Effects 0.000 description 2
- 230000009286 beneficial effect Effects 0.000 description 2
- 210000004369 blood Anatomy 0.000 description 2
- 239000008280 blood Substances 0.000 description 2
- 239000006227 byproduct Substances 0.000 description 2
- 229910052791 calcium Inorganic materials 0.000 description 2
- 239000011575 calcium Substances 0.000 description 2
- 230000000747 cardiac effect Effects 0.000 description 2
- 210000004413 cardiac myocyte Anatomy 0.000 description 2
- 210000002421 cell wall Anatomy 0.000 description 2
- 150000001875 compounds Chemical class 0.000 description 2
- 230000007547 defect Effects 0.000 description 2
- 238000012217 deletion Methods 0.000 description 2
- 230000037430 deletion Effects 0.000 description 2
- 238000002474 experimental method Methods 0.000 description 2
- 201000001386 familial hypercholesterolemia Diseases 0.000 description 2
- 230000001605 fetal effect Effects 0.000 description 2
- 210000002950 fibroblast Anatomy 0.000 description 2
- 229940088597 hormone Drugs 0.000 description 2
- 239000005556 hormone Substances 0.000 description 2
- 230000007813 immunodeficiency Effects 0.000 description 2
- 230000001965 increasing effect Effects 0.000 description 2
- 230000001939 inductive effect Effects 0.000 description 2
- NOESYZHRGYRDHS-UHFFFAOYSA-N insulin Chemical compound N1C(=O)C(NC(=O)C(CCC(N)=O)NC(=O)C(CCC(O)=O)NC(=O)C(C(C)C)NC(=O)C(NC(=O)CN)C(C)CC)CSSCC(C(NC(CO)C(=O)NC(CC(C)C)C(=O)NC(CC=2C=CC(O)=CC=2)C(=O)NC(CCC(N)=O)C(=O)NC(CC(C)C)C(=O)NC(CCC(O)=O)C(=O)NC(CC(N)=O)C(=O)NC(CC=2C=CC(O)=CC=2)C(=O)NC(CSSCC(NC(=O)C(C(C)C)NC(=O)C(CC(C)C)NC(=O)C(CC=2C=CC(O)=CC=2)NC(=O)C(CC(C)C)NC(=O)C(C)NC(=O)C(CCC(O)=O)NC(=O)C(C(C)C)NC(=O)C(CC(C)C)NC(=O)C(CC=2NC=NC=2)NC(=O)C(CO)NC(=O)CNC2=O)C(=O)NCC(=O)NC(CCC(O)=O)C(=O)NC(CCCNC(N)=N)C(=O)NCC(=O)NC(CC=3C=CC=CC=3)C(=O)NC(CC=3C=CC=CC=3)C(=O)NC(CC=3C=CC(O)=CC=3)C(=O)NC(C(C)O)C(=O)N3C(CCC3)C(=O)NC(CCCCN)C(=O)NC(C)C(O)=O)C(=O)NC(CC(N)=O)C(O)=O)=O)NC(=O)C(C(C)CC)NC(=O)C(CO)NC(=O)C(C(C)O)NC(=O)C1CSSCC2NC(=O)C(CC(C)C)NC(=O)C(NC(=O)C(CCC(N)=O)NC(=O)C(CC(N)=O)NC(=O)C(NC(=O)C(N)CC=1C=CC=CC=1)C(C)C)CC1=CN=CN1 NOESYZHRGYRDHS-UHFFFAOYSA-N 0.000 description 2
- 210000003963 intermediate filament Anatomy 0.000 description 2
- 238000010255 intramuscular injection Methods 0.000 description 2
- 239000007927 intramuscular injection Substances 0.000 description 2
- 230000007774 longterm Effects 0.000 description 2
- 208000030159 metabolic disease Diseases 0.000 description 2
- 210000003470 mitochondria Anatomy 0.000 description 2
- 238000010369 molecular cloning Methods 0.000 description 2
- 210000000056 organ Anatomy 0.000 description 2
- 230000035699 permeability Effects 0.000 description 2
- 230000008488 polyadenylation Effects 0.000 description 2
- 102000037983 regulatory factors Human genes 0.000 description 2
- 108091008025 regulatory factors Proteins 0.000 description 2
- 230000001177 retroviral effect Effects 0.000 description 2
- 230000028327 secretion Effects 0.000 description 2
- 101150061985 ska gene Proteins 0.000 description 2
- 210000002460 smooth muscle Anatomy 0.000 description 2
- 210000000130 stem cell Anatomy 0.000 description 2
- 238000007910 systemic administration Methods 0.000 description 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 1
- NHBKXEKEPDILRR-UHFFFAOYSA-N 2,3-bis(butanoylsulfanyl)propyl butanoate Chemical compound CCCC(=O)OCC(SC(=O)CCC)CSC(=O)CCC NHBKXEKEPDILRR-UHFFFAOYSA-N 0.000 description 1
- BFSVOASYOCHEOV-UHFFFAOYSA-N 2-diethylaminoethanol Chemical compound CCN(CC)CCO BFSVOASYOCHEOV-UHFFFAOYSA-N 0.000 description 1
- 108020005345 3' Untranslated Regions Proteins 0.000 description 1
- 108020003589 5' Untranslated Regions Proteins 0.000 description 1
- 229920000936 Agarose Polymers 0.000 description 1
- 102100024321 Alkaline phosphatase, placental type Human genes 0.000 description 1
- 108010077173 BB Form Creatine Kinase Proteins 0.000 description 1
- 102000015081 Blood Coagulation Factors Human genes 0.000 description 1
- 108010039209 Blood Coagulation Factors Proteins 0.000 description 1
- 241000283690 Bos taurus Species 0.000 description 1
- 241000282693 Cercopithecidae Species 0.000 description 1
- 241000699800 Cricetinae Species 0.000 description 1
- 102000012410 DNA Ligases Human genes 0.000 description 1
- 108010061982 DNA Ligases Proteins 0.000 description 1
- 102000004594 DNA Polymerase I Human genes 0.000 description 1
- 108010017826 DNA Polymerase I Proteins 0.000 description 1
- 230000007023 DNA restriction-modification system Effects 0.000 description 1
- 101100120663 Drosophila melanogaster fs(1)h gene Proteins 0.000 description 1
- 102100021238 Dynamin-2 Human genes 0.000 description 1
- 108010069091 Dystrophin Proteins 0.000 description 1
- 102000001039 Dystrophin Human genes 0.000 description 1
- 108091035710 E-box Proteins 0.000 description 1
- 241000283073 Equus caballus Species 0.000 description 1
- 241000588724 Escherichia coli Species 0.000 description 1
- 208000024720 Fabry Disease Diseases 0.000 description 1
- 241000282326 Felis catus Species 0.000 description 1
- 102000016970 Follistatin Human genes 0.000 description 1
- 108010014612 Follistatin Proteins 0.000 description 1
- 208000015872 Gaucher disease Diseases 0.000 description 1
- 102000053171 Glial Fibrillary Acidic Human genes 0.000 description 1
- 101710193519 Glial fibrillary acidic protein Proteins 0.000 description 1
- 108010088406 Glucagon-Like Peptides Proteins 0.000 description 1
- 102000004547 Glucosylceramidase Human genes 0.000 description 1
- 108010017544 Glucosylceramidase Proteins 0.000 description 1
- 102000053187 Glucuronidase Human genes 0.000 description 1
- 108010060309 Glucuronidase Proteins 0.000 description 1
- 208000009292 Hemophilia A Diseases 0.000 description 1
- 101000817607 Homo sapiens Dynamin-2 Proteins 0.000 description 1
- 101000599951 Homo sapiens Insulin-like growth factor I Proteins 0.000 description 1
- 101000803403 Homo sapiens Vimentin Proteins 0.000 description 1
- 241000701024 Human betaherpesvirus 5 Species 0.000 description 1
- 108090001061 Insulin Proteins 0.000 description 1
- 102000004877 Insulin Human genes 0.000 description 1
- 102000011782 Keratins Human genes 0.000 description 1
- 108010076876 Keratins Proteins 0.000 description 1
- AEMOLEFTQBMNLQ-HNFCZKTMSA-N L-idopyranuronic acid Chemical compound OC1O[C@@H](C(O)=O)[C@@H](O)[C@H](O)[C@H]1O AEMOLEFTQBMNLQ-HNFCZKTMSA-N 0.000 description 1
- 108010001831 LDL receptors Proteins 0.000 description 1
- 108010092277 Leptin Proteins 0.000 description 1
- 102000016267 Leptin Human genes 0.000 description 1
- 102000004882 Lipase Human genes 0.000 description 1
- 108090001060 Lipase Proteins 0.000 description 1
- 239000004367 Lipase Substances 0.000 description 1
- 241000406668 Loxodonta cyclotis Species 0.000 description 1
- 208000015439 Lysosomal storage disease Diseases 0.000 description 1
- 102100024295 Maltase-glucoamylase Human genes 0.000 description 1
- 108700005084 Multigene Family Proteins 0.000 description 1
- 241000699660 Mus musculus Species 0.000 description 1
- 206010028289 Muscle atrophy Diseases 0.000 description 1
- 108060008487 Myosin Proteins 0.000 description 1
- 102000003505 Myosin Human genes 0.000 description 1
- 101001055320 Myxine glutinosa Insulin-like growth factor Proteins 0.000 description 1
- 102000008763 Neurofilament Proteins Human genes 0.000 description 1
- 108010088373 Neurofilament Proteins Proteins 0.000 description 1
- 241000283973 Oryctolagus cuniculus Species 0.000 description 1
- 238000012408 PCR amplification Methods 0.000 description 1
- 241000282577 Pan troglodytes Species 0.000 description 1
- 108090000445 Parathyroid hormone Proteins 0.000 description 1
- 102000035195 Peptidases Human genes 0.000 description 1
- 108091005804 Peptidases Proteins 0.000 description 1
- 208000006735 Periostitis Diseases 0.000 description 1
- 241000009328 Perro Species 0.000 description 1
- 108010039918 Polylysine Proteins 0.000 description 1
- 241000282405 Pongo abelii Species 0.000 description 1
- 101710098940 Pro-epidermal growth factor Proteins 0.000 description 1
- 239000004365 Protease Substances 0.000 description 1
- 102000009572 RNA Polymerase II Human genes 0.000 description 1
- 108010009460 RNA Polymerase II Proteins 0.000 description 1
- 230000006819 RNA synthesis Effects 0.000 description 1
- 101000883446 Rattus norvegicus Desmin Proteins 0.000 description 1
- 101000702488 Rattus norvegicus High affinity cationic amino acid transporter 1 Proteins 0.000 description 1
- 108020004511 Recombinant DNA Proteins 0.000 description 1
- 241000283984 Rodentia Species 0.000 description 1
- 241000714474 Rous sarcoma virus Species 0.000 description 1
- 102000011971 Sphingomyelin Phosphodiesterase Human genes 0.000 description 1
- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 description 1
- 241000282898 Sus scrofa Species 0.000 description 1
- 108091005735 TGF-beta receptors Proteins 0.000 description 1
- 102000004887 Transforming Growth Factor beta Human genes 0.000 description 1
- 108090001012 Transforming Growth Factor beta Proteins 0.000 description 1
- 102000016715 Transforming Growth Factor beta Receptors Human genes 0.000 description 1
- 229920004890 Triton X-100 Polymers 0.000 description 1
- 239000013504 Triton X-100 Substances 0.000 description 1
- 102000013127 Vimentin Human genes 0.000 description 1
- 108010065472 Vimentin Proteins 0.000 description 1
- 101000756604 Xenopus laevis Actin, cytoplasmic 1 Proteins 0.000 description 1
- 210000001789 adipocyte Anatomy 0.000 description 1
- 230000002411 adverse Effects 0.000 description 1
- 102000005840 alpha-Galactosidase Human genes 0.000 description 1
- 108010030291 alpha-Galactosidase Proteins 0.000 description 1
- 108010028144 alpha-Glucosidases Proteins 0.000 description 1
- 102000002014 alpha-N-Acetylgalactosaminidase Human genes 0.000 description 1
- 108010015684 alpha-N-Acetylgalactosaminidase Proteins 0.000 description 1
- 238000000137 annealing Methods 0.000 description 1
- 230000033228 biological regulation Effects 0.000 description 1
- 230000015572 biosynthetic process Effects 0.000 description 1
- 239000003114 blood coagulation factor Substances 0.000 description 1
- 229940019700 blood coagulation factors Drugs 0.000 description 1
- 210000000988 bone and bone Anatomy 0.000 description 1
- 210000001185 bone marrow Anatomy 0.000 description 1
- 229910000389 calcium phosphate Inorganic materials 0.000 description 1
- 239000001506 calcium phosphate Substances 0.000 description 1
- 235000011010 calcium phosphates Nutrition 0.000 description 1
- 101150055766 cat gene Proteins 0.000 description 1
- 125000002091 cationic group Chemical group 0.000 description 1
- 230000011712 cell development Effects 0.000 description 1
- 210000000170 cell membrane Anatomy 0.000 description 1
- 230000004663 cell proliferation Effects 0.000 description 1
- 239000003153 chemical reaction reagent Substances 0.000 description 1
- 238000010367 cloning Methods 0.000 description 1
- 238000012411 cloning technique Methods 0.000 description 1
- 239000002131 composite material Substances 0.000 description 1
- 239000000470 constituent Substances 0.000 description 1
- 238000010276 construction Methods 0.000 description 1
- 230000008602 contraction Effects 0.000 description 1
- 210000004748 cultured cell Anatomy 0.000 description 1
- 230000001419 dependent effect Effects 0.000 description 1
- 238000013461 design Methods 0.000 description 1
- 239000003599 detergent Substances 0.000 description 1
- 238000011161 development Methods 0.000 description 1
- 230000018109 developmental process Effects 0.000 description 1
- 238000010586 diagram Methods 0.000 description 1
- 230000004069 differentiation Effects 0.000 description 1
- 230000005014 ectopic expression Effects 0.000 description 1
- 239000002158 endotoxin Substances 0.000 description 1
- 210000002919 epithelial cell Anatomy 0.000 description 1
- 210000003527 eukaryotic cell Anatomy 0.000 description 1
- 210000005046 glial fibrillary acidic protein Anatomy 0.000 description 1
- 230000034659 glycolysis Effects 0.000 description 1
- 239000000122 growth hormone Substances 0.000 description 1
- 230000005745 host immune response Effects 0.000 description 1
- 102000057393 human VIM Human genes 0.000 description 1
- 239000000819 hypertonic solution Substances 0.000 description 1
- 229940021223 hypertonic solution Drugs 0.000 description 1
- 210000003111 iliac vein Anatomy 0.000 description 1
- 238000002513 implantation Methods 0.000 description 1
- 238000000338 in vitro Methods 0.000 description 1
- 238000011534 incubation Methods 0.000 description 1
- 239000000411 inducer Substances 0.000 description 1
- 230000002401 inhibitory effect Effects 0.000 description 1
- 238000003780 insertion Methods 0.000 description 1
- 230000037431 insertion Effects 0.000 description 1
- 229940125396 insulin Drugs 0.000 description 1
- 230000010189 intracellular transport Effects 0.000 description 1
- 230000037427 ion transport Effects 0.000 description 1
- 229940039781 leptin Drugs 0.000 description 1
- NRYBAZVQPHGZNS-ZSOCWYAHSA-N leptin Chemical compound O=C([C@H](CO)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CCC(N)=O)NC(=O)[C@H](CC=1C2=CC=CC=C2NC=1)NC(=O)[C@H](CC(C)C)NC(=O)[C@@H](NC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CCC(N)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CO)NC(=O)CNC(=O)[C@H](CCC(N)=O)NC(=O)[C@@H](N)CC(C)C)CCSC)N1CCC[C@H]1C(=O)NCC(=O)N[C@@H](CS)C(O)=O NRYBAZVQPHGZNS-ZSOCWYAHSA-N 0.000 description 1
- 235000019421 lipase Nutrition 0.000 description 1
- 239000002502 liposome Substances 0.000 description 1
- 238000012423 maintenance Methods 0.000 description 1
- 210000001161 mammalian embryo Anatomy 0.000 description 1
- 238000004519 manufacturing process Methods 0.000 description 1
- 230000001404 mediated effect Effects 0.000 description 1
- 210000002901 mesenchymal stem cell Anatomy 0.000 description 1
- 108020004999 messenger RNA Proteins 0.000 description 1
- 229910021645 metal ion Inorganic materials 0.000 description 1
- 210000003632 microfilament Anatomy 0.000 description 1
- 239000000203 mixture Substances 0.000 description 1
- 230000004899 motility Effects 0.000 description 1
- 230000004118 muscle contraction Effects 0.000 description 1
- 201000000585 muscular atrophy Diseases 0.000 description 1
- 210000003365 myofibril Anatomy 0.000 description 1
- 210000003061 neural cell Anatomy 0.000 description 1
- 230000001537 neural effect Effects 0.000 description 1
- 210000005044 neurofilament Anatomy 0.000 description 1
- 210000004498 neuroglial cell Anatomy 0.000 description 1
- 230000003472 neutralizing effect Effects 0.000 description 1
- 238000007899 nucleic acid hybridization Methods 0.000 description 1
- 230000003204 osmotic effect Effects 0.000 description 1
- 210000003460 periosteum Anatomy 0.000 description 1
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 description 1
- 229950007002 phosphocreatine Drugs 0.000 description 1
- 210000002826 placenta Anatomy 0.000 description 1
- 108010031345 placental alkaline phosphatase Proteins 0.000 description 1
- 239000013600 plasmid vector Substances 0.000 description 1
- 229920000656 polylysine Polymers 0.000 description 1
- 229920005862 polyol Polymers 0.000 description 1
- 150000003077 polyols Chemical class 0.000 description 1
- 229920001184 polypeptide Polymers 0.000 description 1
- 230000007542 postnatal development Effects 0.000 description 1
- 239000002243 precursor Substances 0.000 description 1
- 108010066381 preproinsulin Proteins 0.000 description 1
- 108090000765 processed proteins & peptides Proteins 0.000 description 1
- 102000004196 processed proteins & peptides Human genes 0.000 description 1
- 238000002708 random mutagenesis Methods 0.000 description 1
- 108020003175 receptors Proteins 0.000 description 1
- 102000005962 receptors Human genes 0.000 description 1
- 230000008929 regeneration Effects 0.000 description 1
- 238000011069 regeneration method Methods 0.000 description 1
- 230000008439 repair process Effects 0.000 description 1
- 238000011160 research Methods 0.000 description 1
- 230000029058 respiratory gaseous exchange Effects 0.000 description 1
- 108091008146 restriction endonucleases Proteins 0.000 description 1
- 238000012552 review Methods 0.000 description 1
- 239000000523 sample Substances 0.000 description 1
- 230000012232 skeletal muscle contraction Effects 0.000 description 1
- 230000022379 skeletal muscle tissue development Effects 0.000 description 1
- 210000003491 skin Anatomy 0.000 description 1
- 210000000329 smooth muscle myocyte Anatomy 0.000 description 1
- 238000012453 sprague-dawley rat model Methods 0.000 description 1
- 238000010561 standard procedure Methods 0.000 description 1
- 239000000126 substance Substances 0.000 description 1
- 238000006467 substitution reaction Methods 0.000 description 1
- 235000000346 sugar Nutrition 0.000 description 1
- 150000008163 sugars Chemical class 0.000 description 1
- 238000012385 systemic delivery Methods 0.000 description 1
- 230000009885 systemic effect Effects 0.000 description 1
- ZRKFYGHZFMAOKI-QMGMOQQFSA-N tgfbeta Chemical compound C([C@H](NC(=O)[C@H](C(C)C)NC(=O)CNC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](CCCNC(N)=N)NC(=O)[C@H](CC(N)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H]([C@@H](C)O)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@H]([C@@H](C)O)NC(=O)[C@H](CC(C)C)NC(=O)CNC(=O)[C@H](C)NC(=O)[C@H](CO)NC(=O)[C@H](CCC(N)=O)NC(=O)[C@@H](NC(=O)[C@H](C)NC(=O)[C@H](C)NC(=O)[C@@H](NC(=O)[C@H](CC(C)C)NC(=O)[C@@H](N)CCSC)C(C)C)[C@@H](C)CC)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H](C(C)C)C(=O)N[C@@H](CC=1C=CC=CC=1)C(=O)N[C@@H](C)C(=O)N1[C@@H](CCC1)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](C)C(=O)N[C@@H](CC=1C=CC=CC=1)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](C)C(=O)N[C@@H](CC(C)C)C(=O)N1[C@@H](CCC1)C(=O)N1[C@@H](CCC1)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CO)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CC(C)C)C(O)=O)C1=CC=C(O)C=C1 ZRKFYGHZFMAOKI-QMGMOQQFSA-N 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
- 230000008467 tissue growth Effects 0.000 description 1
- 231100000331 toxic Toxicity 0.000 description 1
- 230000002588 toxic effect Effects 0.000 description 1
- 231100000419 toxicity Toxicity 0.000 description 1
- 230000001988 toxicity Effects 0.000 description 1
- 238000010361 transduction Methods 0.000 description 1
- 230000026683 transduction Effects 0.000 description 1
- 230000009466 transformation Effects 0.000 description 1
- 238000011830 transgenic mouse model Methods 0.000 description 1
- 230000001052 transient effect Effects 0.000 description 1
- 230000010474 transient expression Effects 0.000 description 1
- 238000002054 transplantation Methods 0.000 description 1
- QORWJWZARLRLPR-UHFFFAOYSA-H tricalcium bis(phosphate) Chemical compound [Ca+2].[Ca+2].[Ca+2].[O-]P([O-])([O-])=O.[O-]P([O-])([O-])=O QORWJWZARLRLPR-UHFFFAOYSA-H 0.000 description 1
- 230000007306 turnover Effects 0.000 description 1
- 208000001072 type 2 diabetes mellitus Diseases 0.000 description 1
- 210000003954 umbilical cord Anatomy 0.000 description 1
- 230000002792 vascular Effects 0.000 description 1
- 210000005048 vimentin Anatomy 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
-
- 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
- A61P3/06—Antihyperlipidemics
-
- 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
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
- C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Diabetes (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Hematology (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Obesity (AREA)
- Emergency Medicine (AREA)
- Endocrinology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US29330401P | 2001-05-24 | 2001-05-24 | |
| US60/293,304 | 2001-05-24 | ||
| PCT/US2002/016748 WO2002095006A2 (en) | 2001-05-24 | 2002-05-22 | Muscle-specific expression vectors |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2448120A1 true CA2448120A1 (en) | 2002-11-28 |
Family
ID=23128549
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA002448120A Abandoned CA2448120A1 (en) | 2001-05-24 | 2002-05-22 | Muscle-specific expression vectors |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US20030100526A1 (enExample) |
| EP (2) | EP1390490B1 (enExample) |
| JP (1) | JP2004535801A (enExample) |
| AT (1) | ATE428777T1 (enExample) |
| CA (1) | CA2448120A1 (enExample) |
| DE (1) | DE60231975D1 (enExample) |
| WO (1) | WO2002095006A2 (enExample) |
Families Citing this family (67)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2406687A1 (en) * | 2001-11-09 | 2003-05-09 | Transgene S.A. | Chimeric promoters for controlling expression in muscle cells |
| US20080044393A1 (en) * | 2004-07-16 | 2008-02-21 | White Robert L | Retinal dystrophin transgene and methods of use thereof |
| US8557969B2 (en) | 2004-12-01 | 2013-10-15 | The Regents Of The University Of Michigan | Compositions and methods for regulating cardiac performance |
| WO2008054713A2 (en) * | 2006-10-30 | 2008-05-08 | Thomas Jefferson University | Tissue specific gene therapy treatment |
| US20120322861A1 (en) * | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| WO2012136811A1 (en) | 2011-04-06 | 2012-10-11 | Universite Pierre Et Marie Curie (Paris 6) | A skeletal muscle-specific enhancer |
| EP4124346A3 (en) * | 2012-06-19 | 2023-04-19 | University of Florida Research Foundation, Inc. | Compositions and methods for treating diseases |
| EP2885008B1 (en) | 2012-08-15 | 2018-11-07 | University Of Virginia Patent Foundation | Compositions and methods for treating peripheral arterial disease |
| CN106414739A (zh) | 2012-12-20 | 2017-02-15 | 萨雷普塔医疗公司 | 用于治疗肌营养不良症的改良的外显子跳跃组合物 |
| EP3760720A1 (en) | 2013-03-14 | 2021-01-06 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
| US9217148B2 (en) | 2013-03-14 | 2015-12-22 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
| WO2015035364A1 (en) * | 2013-09-09 | 2015-03-12 | University Of Washington Through Its Center For Commercialization | Proteins for targeting neuronal nitric oxide synthase to muscle sarcolemma and related methods of use |
| AU2015208247B2 (en) | 2014-01-21 | 2021-05-27 | Universiteit Gent | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
| US10472408B2 (en) | 2015-03-05 | 2019-11-12 | Peter Und Traudl Engelhorn-Stiftung Zur Förderung Der Lebenswissenschaften | Fusion proteins comprising partial tetraspanin sequences and a system thereof for presenting peptides on the cell surface |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| CN104894651B (zh) * | 2015-06-29 | 2017-04-12 | 天津诺禾医学检验所有限公司 | 微量起始dna的高通量测序文库构建方法及其所构建的高通量测序文库 |
| KR102799163B1 (ko) * | 2015-09-17 | 2025-04-21 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | Galgt2 유전자 치료 방법 및 물질 |
| AU2017250017B2 (en) | 2016-04-14 | 2022-12-22 | Benitec IP Holdings Inc. | Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof |
| WO2017223128A1 (en) | 2016-06-21 | 2017-12-28 | The Curators Of The University Of Missouri | Modified dystrophin proteins |
| US11512327B2 (en) | 2017-08-03 | 2022-11-29 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of AAV |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| CN111936621B (zh) | 2018-02-05 | 2025-05-13 | 奥登茨治疗公司 | 转录调控元件和它的用途 |
| CA3086942A1 (en) | 2018-02-07 | 2019-08-15 | Genethon | Hybrid regulatory elements |
| CA3097192A1 (en) | 2018-04-27 | 2019-10-31 | Voyager Therapeutics, Inc. | Methods for measuring the potency of aadc viral vectors |
| WO2019222329A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| CN112424359A (zh) | 2018-05-15 | 2021-02-26 | 沃雅戈治疗公司 | 用于治疗帕金森氏病的组合物和方法 |
| WO2019241486A1 (en) | 2018-06-13 | 2019-12-19 | Voyager Therapeutics, Inc. | Engineered 5' untranslated regions (5' utr) for aav production |
| US20210254103A1 (en) | 2018-07-02 | 2021-08-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
| WO2020072849A1 (en) | 2018-10-04 | 2020-04-09 | Voyager Therapeutics, Inc. | Methods for measuring the titer and potency of viral vector particles |
| AU2019354793A1 (en) | 2018-10-05 | 2021-05-13 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding AAV production proteins |
| US20210371470A1 (en) | 2018-10-12 | 2021-12-02 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| WO2020081490A1 (en) | 2018-10-15 | 2020-04-23 | Voyager Therapeutics, Inc. | EXPRESSION VECTORS FOR LARGE-SCALE PRODUCTION OF rAAV IN THE BACULOVIRUS/Sf9 SYSTEM |
| MX2021005720A (es) | 2018-11-16 | 2021-07-21 | Astellas Pharma Inc | Metodo para tratar la distrofia muscular mediante el direccionamiento del gen de utrofina. |
| CA3125770A1 (en) | 2019-01-18 | 2020-07-23 | Voyager Therapeutics, Inc. | Methods and systems for producing aav particles |
| CA3130221A1 (en) | 2019-02-28 | 2020-09-03 | Benitec IP Holdings Inc. | Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd) |
| KR20210148273A (ko) * | 2019-04-08 | 2021-12-07 | 제네똥 | 근육 발현을 위한 하이브리드 프로모터 |
| EP3966227A1 (en) | 2019-05-07 | 2022-03-16 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
| JP7565620B2 (ja) | 2019-05-28 | 2024-10-11 | 株式会社モダリス | Ｄｍｐｋ遺伝子を標的とした筋ジストロフィーの治療方法 |
| BR112022002889A2 (pt) | 2019-08-16 | 2022-05-17 | Modalis Therapeutics Corp | Método para tratar distrofia muscular tendo como alvo o gene lama1 |
| WO2021046155A1 (en) | 2019-09-03 | 2021-03-11 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
| WO2021202651A1 (en) | 2020-04-01 | 2021-10-07 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
| CA3180222A1 (en) | 2020-04-15 | 2021-10-21 | Voyager Therapeutics, Inc. | Tau binding compounds |
| US12630842B2 (en) | 2020-05-13 | 2026-05-19 | Voyager Therapeutics, Inc. | Redirection of tropism of AAV capsids |
| WO2021230385A1 (en) | 2020-05-15 | 2021-11-18 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting utrophin gene |
| WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
| WO2022056291A1 (en) * | 2020-09-11 | 2022-03-17 | Nature Technology Corporation | Muscle-specific hybrid promoter |
| WO2022073041A2 (en) | 2020-09-30 | 2022-04-07 | NGGT, Inc. | Dual functional expression vectors and methods of use thereof |
| JP2023548746A (ja) | 2020-10-13 | 2023-11-21 | クリヤ セラピューティクス インコーポレイテッド | サイトカインをコードする核酸を送達するためのウイルスベクターコンストラクトおよびがんを処置するためのその使用 |
| WO2022114243A1 (en) | 2020-11-25 | 2022-06-02 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting dmpk gene |
| CA3206107A1 (en) | 2021-01-26 | 2022-08-04 | Brian Furmanski | Vector constructs for delivery of nucleic acids encoding therapeutic anti-tnf antibodies and methods of using the same |
| EP4036238A1 (en) * | 2021-01-27 | 2022-08-03 | The University Court of the University of Edinburgh | Functional nucleic acid molecule and method |
| IL304956A (en) | 2021-02-12 | 2023-10-01 | Merand Pharmaceuticals Inc | Agents, compositions, and methods for the treatment of hypoxia and ischemia-related disorders |
| EP4301860A1 (en) | 2021-03-04 | 2024-01-10 | Kriya Therapeutics, Inc. | Viral vector constructs incorporating dna for inhibiting toll like receptors and methods of using the same |
| WO2023012313A1 (en) * | 2021-08-04 | 2023-02-09 | Genethon | Hybrid promoters for gene expression in muscles and in the cns |
| WO2023023055A1 (en) | 2021-08-16 | 2023-02-23 | Renagade Therapeutics Management Inc. | Compositions and methods for optimizing tropism of delivery systems for rna |
| TW202328067A (zh) | 2021-09-14 | 2023-07-16 | 美商雷納嘉德醫療管理公司 | 環狀脂質及其使用方法 |
| WO2023044343A1 (en) | 2021-09-14 | 2023-03-23 | Renagade Therapeutics Management Inc. | Acyclic lipids and methods of use thereof |
| WO2023044483A2 (en) | 2021-09-20 | 2023-03-23 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
| JP2025503637A (ja) | 2022-01-09 | 2025-02-04 | クリヤ セラピューティクス， インコーポレイテッド | 治療用抗ｉｇｆ－１ｒ抗体をコードする核酸の送達のためのベクター構築物およびそれを使用する方法 |
| US20250243510A1 (en) * | 2022-03-16 | 2025-07-31 | Rutgers, The State University Of New Jersey | Controlled muscle-specific gene delivery |
| CA3255619A1 (en) | 2022-04-07 | 2023-10-12 | Renagade Therapeutics Management Inc. | CYCLIC LIPIDS AND LIPID NANOPARTICLES (LPNs) FOR THE SUPPLY OF NUCLEIC ACIDS OR PEPTIDES INTENDED FOR USE IN VACCINATION AGAINST INFECTIOUS AGENTS |
| WO2023220695A2 (en) | 2022-05-13 | 2023-11-16 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
| US20250243263A1 (en) | 2022-06-22 | 2025-07-31 | Voyager Therapeutics, Inc. | Tau binding compounds |
| JP2025526502A (ja) * | 2022-07-11 | 2025-08-13 | ヴァイタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための治療用サテライト細胞の製造 |
| JP2025535203A (ja) | 2022-09-15 | 2025-10-23 | ボイジャー セラピューティクス インコーポレイテッド | タウ結合化合物 |
| AU2024287813A1 (en) | 2023-07-07 | 2026-01-22 | Kriya Therapeutics, Inc. | Periocular delivery of aav vectors for treating ophthalmic pathologies |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5266488A (en) * | 1988-04-06 | 1993-11-30 | The Regents Of The University Of California | Troponin T gene promoter and derivatives thereof |
| US5795872A (en) * | 1995-09-19 | 1998-08-18 | Pharmadigm, Inc. | DNA construct for immunization |
| US5846528A (en) * | 1996-01-18 | 1998-12-08 | Avigen, Inc. | Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence |
| US5858351A (en) * | 1996-01-18 | 1999-01-12 | Avigen, Inc. | Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors |
| DE69918090T2 (de) * | 1998-04-24 | 2005-06-16 | University Of Florida, Gainesville | Rekombinanter adeno-assoziierter viraler vektor, der alpha-1 antitrypsin kodiert, zur gentherapie |
| WO2001083695A2 (en) * | 2000-04-28 | 2001-11-08 | Xiao Xiao | Dna sequences encoding dystrophin minigenes and methods of use thereof |
| US6593110B2 (en) * | 2000-05-04 | 2003-07-15 | California Institute Of Technology | Checkpoint-activating oligonucleotides |
-
2002
- 2002-05-22 EP EP02739449A patent/EP1390490B1/en not_active Expired - Lifetime
- 2002-05-22 AT AT02739449T patent/ATE428777T1/de not_active IP Right Cessation
- 2002-05-22 WO PCT/US2002/016748 patent/WO2002095006A2/en not_active Ceased
- 2002-05-22 DE DE60231975T patent/DE60231975D1/de not_active Expired - Lifetime
- 2002-05-22 JP JP2002592469A patent/JP2004535801A/ja active Pending
- 2002-05-22 EP EP08103273A patent/EP2017338A1/en not_active Withdrawn
- 2002-05-22 CA CA002448120A patent/CA2448120A1/en not_active Abandoned
- 2002-05-24 US US10/156,604 patent/US20030100526A1/en not_active Abandoned
-
2007
- 2007-03-12 US US11/716,899 patent/US20110212529A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| EP1390490B1 (en) | 2009-04-15 |
| WO2002095006A2 (en) | 2002-11-28 |
| US20030100526A1 (en) | 2003-05-29 |
| DE60231975D1 (de) | 2009-05-28 |
| ATE428777T1 (de) | 2009-05-15 |
| EP1390490A2 (en) | 2004-02-25 |
| WO2002095006A3 (en) | 2003-03-13 |
| EP1390490A4 (en) | 2005-05-11 |
| JP2004535801A (ja) | 2004-12-02 |
| EP2017338A1 (en) | 2009-01-21 |
| US20110212529A1 (en) | 2011-09-01 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| EP1390490B1 (en) | Muscle-specific expression vectors | |
| AU2023214366B2 (en) | Gene therapies for lysosomal disorders | |
| Tripathy et al. | Stable delivery of physiologic levels of recombinant erythropoietin to the systemic circulation by intramuscular injection of replication-defective adenovirus. | |
| WO2021163357A2 (en) | Gene therapy vectors for treating heart disease | |
| CA2684534C (en) | Constructs for enhancement of gene expression in muscle | |
| WO2020165603A1 (en) | Myc, cyclin t1 and/or cdk9 for use in the treatment of degenerative heart and cns disorders | |
| RS20050614A (sr) | Ekspresiona kaseta i vektor za prolaznu ili stabilnu ekspresiju egzogenih molekula | |
| KR20220035107A (ko) | Arsa 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 | |
| CN102127546B (zh) | 一种骨骼肌特异性actin启动子及其应用 | |
| Liu et al. | Synthetic promoter for efficient and muscle-specific expression of exogenous genes | |
| KR20140015999A (ko) | 신규 ＭＡＲｓ 및 이를 이용하여 목적 단백질을 생산하는 방법 | |
| AU2002312095A1 (en) | Muscle-specific expression vectors | |
| CN114144203A (zh) | 源于显性变异基因的疾患的治疗剂 | |
| CN106497930B (zh) | 骨骼肌细胞高效特异性启动子及其应用 | |
| RU2686102C1 (ru) | Рекомбинантный вектор для создания плазмидных генетических конструкций, обладающих повышенной длительностью экспрессии целевых генов | |
| JP2003503034A (ja) | 核内ｐｐａｒレセプターを使用する発現調節システム | |
| CA2834729A1 (en) | A skeletal muscle-specific enhancer | |
| US20230141434A1 (en) | Direct in vivo reprogramming using transcription factor etv2 gene for endothelial cell and vessel formation | |
| WO2025122739A1 (en) | Compositions and methods for treating a heart disease | |
| HK40047428A (en) | Gene therapies for lysosomal disorders | |
| CN117402875A (zh) | 利用rna剪接调节剂调控基因表达的核酸分子 | |
| EP3087995A1 (en) | Mutants of srsf1 (asf/sf2) for treating spinal muscular atrophy | |
| IL135430A (en) | Use of a polynucleotide capable of expressing a cytotoxic gene product in the production of a pharmaceutical composition for treating tumors |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request | ||
| FZDE | Discontinued |