SG11201811115TA - Optimized mini-dystrophin genes and expression cassettes and their use - Google Patents
Optimized mini-dystrophin genes and expression cassettes and their useInfo
- Publication number
- SG11201811115TA SG11201811115TA SG11201811115TA SG11201811115TA SG11201811115TA SG 11201811115T A SG11201811115T A SG 11201811115TA SG 11201811115T A SG11201811115T A SG 11201811115TA SG 11201811115T A SG11201811115T A SG 11201811115TA SG 11201811115T A SG11201811115T A SG 11201811115TA
- Authority
- SG
- Singapore
- Prior art keywords
- chapel hill
- international
- mini
- dystrophin
- june
- Prior art date
Links
- 108090000623 proteins and genes Proteins 0.000 title abstract 2
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 235000017166 Bambusa arundinacea Nutrition 0.000 abstract 1
- 235000017491 Bambusa tulda Nutrition 0.000 abstract 1
- 241001330002 Bambuseae Species 0.000 abstract 1
- 235000015334 Phyllostachys viridis Nutrition 0.000 abstract 1
- 108010067390 Viral Proteins Proteins 0.000 abstract 1
- 239000011425 bamboo Substances 0.000 abstract 1
- 239000003814 drug Substances 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 1
- 102000040430 polynucleotide Human genes 0.000 abstract 1
- 108091033319 polynucleotide Proteins 0.000 abstract 1
- 239000002157 polynucleotide Substances 0.000 abstract 1
- 239000013603 viral vector Substances 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
- A01K67/027—New breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
- A01K67/0278—Humanized animals, e.g. knockin
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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- 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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
- C07K14/4708—Duchenne dystrophy
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin, cold insoluble globulin [CIG]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
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- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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- 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
Abstract
INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 28 December 2017 (28.12.2017) WIP0 I PCT omit VIII mum o III om OH OH 010 mil Km lII ois (10) International Publication Number WO 2017/221145 Al (51) International Patent Classification: C07K 14/47 (2006.01) (21) International Application Number: PCT/IB2017/053656 (22) International Filing Date: 20 June 2017 (20.06.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/352,675 21 June 2016 (21.06.2016) US 62/516,449 07 June 2017 (07.06.2017) US (71) Applicants: BAMBOO THERAPEUTICS, INC. [US/US]; 6101 Quadrangle Drive, Suite 200, Chapel Hill, NC 27517 (US). THE UNIVERSITY OF NORTH CAR- OLINA AT CHAPEL HILL [US/US]; 100 Europa Drive, Suite 430, Chapel Hill, NC 27517 (US). (72) Inventors: MCPHEE, Scott W.J.; 1313 Arboretum Dri- ve, Chapel Hill, NC 27517 (US). QIAO, Chunping; 119 Woodshire Lane, Chapel Hill, NC 27514 (US). LI, Juan; 4205 Taylor Hall Place, Chapel Hill, NC 27517 (US). XIAO, Xiao; 4205 Taylor Hall Place, Chapel Hill, NC 27517 (US). MCINTYRE, Maritza; 13512 Oriental Street, Rockville, MD 20853 (US). SAMULSKI, Richard J.; 201 Southwind Lane, Hillsborough, NC 27278 (US). Published: — with international search report (Art. 21(3)) with sequence listing part of description (Rule 5.2(a)) (74) Agent: WALDRON, Roy F. et al.; Pfizer INC., 235 East 42nd Street, MS 235/9/S20, New York, NY 10017 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, 1-1 EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, 11 71' TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, 1-1 KM, ML, MR, NE, SN, TD, TG). 1-1 N 1-1 (54) Title: OPTIMIZED MINI-DYSTROPHIN GENES AND EXPRESSION CASSETTES AND THEIR USE \" (57) : This invention relates to polynucleotides encoding mini-dystrophin proteins, viral vectors comprising the same, and 0 methods of using the same for delivery of mini-dystrophin to a cell or a subject.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662352675P | 2016-06-21 | 2016-06-21 | |
US201762516449P | 2017-06-07 | 2017-06-07 | |
PCT/IB2017/053656 WO2017221145A1 (en) | 2016-06-21 | 2017-06-20 | Optimized mini-dystrophin genes and expression cassettes and their use |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11201811115TA true SG11201811115TA (en) | 2019-01-30 |
Family
ID=59350997
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11201811115TA SG11201811115TA (en) | 2016-06-21 | 2017-06-20 | Optimized mini-dystrophin genes and expression cassettes and their use |
Country Status (18)
Country | Link |
---|---|
US (3) | US20170368198A1 (en) |
EP (1) | EP3472194A1 (en) |
JP (2) | JP6793758B2 (en) |
KR (1) | KR102568278B1 (en) |
CN (2) | CN109641944B (en) |
AU (1) | AU2017281983B2 (en) |
BR (1) | BR112018076394A2 (en) |
CA (1) | CA2971303A1 (en) |
CO (1) | CO2019000395A2 (en) |
IL (2) | IL263199B2 (en) |
MX (1) | MX2018015921A (en) |
MY (1) | MY197846A (en) |
PE (1) | PE20190563A1 (en) |
PH (1) | PH12018502645A1 (en) |
RU (1) | RU2753194C2 (en) |
SG (1) | SG11201811115TA (en) |
TW (1) | TWI719223B (en) |
WO (1) | WO2017221145A1 (en) |
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CA2971303A1 (en) * | 2016-06-21 | 2017-12-21 | Bamboo Therapeutics, Inc. | Optimized mini-dystrophin genes and expression cassettes and their use |
US11534501B2 (en) | 2017-10-18 | 2022-12-27 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
CN108660159B (en) * | 2018-04-12 | 2020-10-30 | 四川大学 | Recombinant bat gland related viral vector and its use |
JP2021521782A (en) * | 2018-04-16 | 2021-08-30 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Compositions and Methods for Treating Duchenne Muscular Dystrophy |
JP2022505816A (en) * | 2018-10-24 | 2022-01-14 | ブリストル-マイヤーズ スクイブ カンパニー | Miniaturized dystrophins and their use |
CN113677697A (en) * | 2019-03-25 | 2021-11-19 | 吉尼松公司 | Production of large dystrophin mimetics using overlapping AAV vectors |
CN111088284B (en) * | 2019-05-31 | 2022-02-01 | 北京京谱迪基因科技有限公司 | AAV vector carrying truncated dystrophin gene expression frame, preparation method and application |
EP3990030A1 (en) * | 2019-06-27 | 2022-05-04 | Pfizer Inc. | Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy |
CN114302968A (en) * | 2019-08-29 | 2022-04-08 | 美国西门子医学诊断股份有限公司 | Reagents and methods for detecting AAV shedding |
CN111057157A (en) * | 2020-01-02 | 2020-04-24 | 北京辉润合众生物科技有限公司 | Fusion protein mDp116 for resisting amyotrophy |
WO2021222332A1 (en) | 2020-04-29 | 2021-11-04 | Bristol-Myers Squibb Company | Miniaturized dystrophins having spectrin fusion domains and uses thereof |
CN116348149A (en) | 2020-06-15 | 2023-06-27 | 全国儿童医院研究所 | Adeno-associated viral vector delivery for muscular dystrophy |
WO2022029543A1 (en) * | 2020-08-06 | 2022-02-10 | Intas Pharmaceuticals Ltd. | Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy |
WO2022076750A2 (en) | 2020-10-07 | 2022-04-14 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns or muscle delivery |
US20230374542A1 (en) * | 2020-10-07 | 2023-11-23 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21 (lgmd21) |
US20240003898A1 (en) * | 2020-10-30 | 2024-01-04 | Pfizer Inc. | Methods for measuring dystrophin in tissue samples |
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WO2022122733A1 (en) * | 2020-12-08 | 2022-06-16 | Genethon | New gene therapy for the treatment of duchenne muscular dystrophy |
EP4263840A1 (en) | 2020-12-21 | 2023-10-25 | Pfizer Inc. | Methods and systems for improved cell transfection |
US20240011012A1 (en) | 2020-12-21 | 2024-01-11 | Pfizer Inc. | Methods and compositions for inhibiting excess nucleic acid precipitation |
WO2022137076A1 (en) | 2020-12-23 | 2022-06-30 | Pfizer Inc. | Methods for purification of aav vectors by affinity chromatography |
CN112860362B (en) * | 2021-02-05 | 2022-10-04 | 达而观数据(成都)有限公司 | Visual debugging method and system for robot automation process |
RU2767335C1 (en) * | 2021-03-02 | 2022-03-17 | Общество с ограниченной ответственностью «Марлин Биотех» | Chimeric proteins based on human utrophin and dystrophin and use thereof for treating duchenne muscular dystrophy |
WO2022229351A1 (en) | 2021-04-28 | 2022-11-03 | Graviton Bioscience Bv | Selective inhibitors of rock2 for the treatment of muscular dystrophy |
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CN114316070B (en) * | 2021-12-29 | 2022-11-15 | 上海勉亦生物科技有限公司 | Transgenic expression cassettes for the treatment of muscular dystrophy |
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