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SG11201808941PA - Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy - Google Patents

Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy

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Publication number
SG11201808941PA
SG11201808941PA SG11201808941PA SG11201808941PA SG11201808941PA SG 11201808941P A SG11201808941P A SG 11201808941PA SG 11201808941P A SG11201808941P A SG 11201808941PA SG 11201808941P A SG11201808941P A SG 11201808941PA SG 11201808941P A SG11201808941P A SG 11201808941PA
Authority
SG
Singapore
Prior art keywords
mir
international
drive
micro
dystrophin
Prior art date
Application number
SG11201808941PA
Inventor
Louise Rodino-Klapac
Jerry Mendell
Original Assignee
Res Inst Nationwide Childrens Hospital
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Res Inst Nationwide Childrens Hospital filed Critical Res Inst Nationwide Childrens Hospital
Publication of SG11201808941PA publication Critical patent/SG11201808941PA/en

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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K35/761—Adenovirus
    • 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
    • A61K38/1719—Muscle proteins, e.g. myosin or actin
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61P19/04—Drugs for skeletal disorders for non-specific disorders of the connective tissue
    • 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
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • C—CHEMISTRY; METALLURGY
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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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    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • 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

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Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (10) International Publication Number (43) International Publication Date WO 2017/181015 Al 19 October 2017 (19.10.2017) WIPO I PCT 111111111111110111011111111111010111110 0111H 0101111101111111111111111110111111 International Patent Classification: Nationwide, Children's Hospital, 700 Children's Drive, A61K 48/00 (2006.01) C07K 14/47 (2006.01) Columbus, OH 43205 (US). A61P 21/00 (2006.01) C12N 15/86 (2006.01) (74) Agents: SINTICH, Sharon, M. et al.; Marshall, Gerstein (51) (21) International Application Number: PCT/US2017/027636 (22) International Filing Date: 14 April 2017 (14.04.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/323,163 15 April 2016 (15.04.2016) US 62/473,253 17 March 2017 (17.03.2017) US (71) Applicant: RESEARCH INSTITUTE AT NATION- WIDE CHILDREN'S HOSPITAL [US/US]; 700 Chil- dren's Drive, Room W172, Columbus, OH 43205 (US). (72) Inventors: RODINO-KLAPAC, Louise; 4912 Bixby Ridge Drive E, Groveport, OH 43125 (US). MENDELL, Jerry, R.; 8176 Copperfield Drive, Columbus, OH 43235 (US). HELLER, Kristin, N.; The Research Institute At & Boron LLP, 233 S. Wacker Drive, 6300 Willis Tower, Chicago, IL 60606-6357 (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, 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, [Continued on next page] (54) Title: ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT MUSCULAR DYS- TROPHY Figure 1 (57) : The invention provides for recombinant AAV vectors comprising a miniaturized human micro-dystrophin gene and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dys- trophy. scAANit.74.CMV.WR29c SEQ ID NO: 2: miR-29C IN A miR-30 BACKBONE (1(11'101(0(y(gaggrItst(AtatocafflatC(FITI)CCTGCACATCTTGGANACACTFGCT(;(;(ATTACT II \"I 1 CAGGrTAAI 11'NACIGAAGGCTCGAGAAGGTATATTGCTGITGACAGIGAGCGCAACCGA ITT( NT.QCFC.( rtc. , , T .TCTACCACCATTTC. ARTCCGTTAMMFAM iKASAUAIRAAUWAX1ACIIIAUSAGUAT1AMISAIIACAAAAICWAA1ACCOLUCIA IEICELMALVArAGGCCGG€C cut site (restriction site) miR - .39.hachbone de - ei stein loop (EWA 41'1'('ACACATG: SF() V) NO. 1.1 ni11-29c target (sense) strand (ACCGATTTEMATGETECTAGA; SEQ ID NO:3)) 1ri12-29e guidelantisenseJ strand LICTACCACCATTTGA(IATCGCTI4LSEQ ID NO: 4j Predicted hairpin structure (SEQ ID N0,6) G UTICA A C - --U CUG CAGUG GCG AACCGAUUUCAAAUGGUGCUAGA GUG A I III 11111 III 1 11111111111 III - - -A GGC GUCAU CGU UUGGCUAAAGUUUACCACGAUCU CAC G A UCC C A GUAGA C W O 20 17 / 18 10 15 Al miR-29C miR-30a shRNA-miR(luc) ggc acc Ca agatanalatac Captor. cagag 11111111111 III11111111 (Milli 11111 u aaCggegasag accacga paaga mu mu 12 a liC gag cuacaaacaucc gaccagaagau pug a 111 111111111111 11111111111 111 cc, gacculluguagg cugacaumagg can g Mika c Moshe Dicer WO 2017/181015 Al 1#11101MOIMEIMMEMOMIIIMMIIMOIMOVOIS DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, Published: LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, with international search report (Art. 21(3)) GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). with sequence listing part of description (Rule 5.2(a))
SG11201808941PA 2016-04-15 2017-04-14 Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy SG11201808941PA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662323163P 2016-04-15 2016-04-15
US201762473253P 2017-03-17 2017-03-17
PCT/US2017/027636 WO2017181015A1 (en) 2016-04-15 2017-04-14 Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy

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SG11201808941PA true SG11201808941PA (en) 2018-11-29

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US (5) US11723986B2 (en)
EP (4) EP3442601B1 (en)
JP (5) JP7153562B2 (en)
KR (2) KR20240042136A (en)
CN (1) CN109069672B (en)
AU (3) AU2017250791B2 (en)
BR (1) BR112018071182A2 (en)
CA (2) CA3020999A1 (en)
CO (1) CO2018012084A2 (en)
ES (1) ES2977730T3 (en)
IL (2) IL262194B2 (en)
MA (2) MA44683A (en)
MX (2) MX2018012604A (en)
MY (1) MY195438A (en)
SG (1) SG11201808941PA (en)
WO (3) WO2017181014A1 (en)
ZA (1) ZA201806746B (en)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA45477A (en) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital ADENOASSOCIATED VIRUS VECTOR VECTOR MICROARN-29 AND MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY
JP7162021B2 (en) 2017-03-17 2022-10-27 ニューカッスル ユニバーシティ Adeno-associated viral vector delivery of microdystrophin fragments to treat muscular dystrophy
SG11201908575SA (en) * 2017-03-17 2019-10-30 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
EP3697915A4 (en) * 2017-10-18 2021-12-08 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
SG11202003285PA (en) * 2017-10-20 2020-05-28 Res Inst Nationwide Childrens Hospital Methods and materials for nt-3 gene therapy
CN118373887A (en) * 2018-03-16 2024-07-23 国家儿童医院研究所 Enhancement of tissue-specific gene delivery by capsid modification
WO2019220435A1 (en) * 2018-05-15 2019-11-21 Ramot At Tel-Aviv University Ltd. Mir126-5p for treating motor neuron diseases
AU2019290544A1 (en) * 2018-06-18 2021-01-28 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
AU2019288291B2 (en) * 2018-06-18 2024-09-26 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
EP3598978B1 (en) 2018-07-26 2024-05-29 EXOFIX S.r.l. Fibroadipogenic progenitor-derived exosomes for regeneration of dystrophic muscles
US20220031865A1 (en) * 2018-12-12 2022-02-03 Solid Biosciences Inc. Combination therapy for treating muscular dystrophy
US20220204574A1 (en) * 2019-03-25 2022-06-30 Genethon Production of large-sized quasidystrophins using overlapping aav vectors
WO2021142435A1 (en) * 2020-01-10 2021-07-15 Solid Biosciences Inc. Combination therapy for treating muscular dystrophy
WO2021142447A1 (en) * 2020-01-10 2021-07-15 Solid Biosciences Inc. Viral vector for combination therapy
BR112022021635A2 (en) 2020-04-29 2022-12-06 Bristol Myers Squibb Co MINIATURIZED DYSTROPHINS HAVING SPECTRIN FUSION DOMAINS AND USES THEREOF
US20210393801A1 (en) * 2020-06-15 2021-12-23 Research Institute At Nationwide Children's Hospital Adeno-Associated Virus Vector Delivery for Muscular Dystrophies
CN111733185A (en) * 2020-06-30 2020-10-02 华南农业大学 Method for realizing improvement of animal meat quality through Mdfi gene editing and application
CA3228365A1 (en) 2021-08-11 2023-02-16 Solid Biosciences Inc. Treatment of muscular dystrophy
EP4401794A1 (en) * 2021-09-16 2024-07-24 University Of Florida Research Foundation, Incorporated Aav particles with modified inverted terminal repeats for enhanced gene expression in muscle
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
WO2024044725A2 (en) 2022-08-24 2024-02-29 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof

Family Cites Families (52)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
DE69433592T2 (en) 1993-11-09 2005-02-10 Targeted Genetics Corp., Seattle THE ACHIEVEMENT OF HIGH TITERS OF THE RECOMBINANT AAV VECTOR
JP3952312B2 (en) 1993-11-09 2007-08-01 メディカル カレッジ オブ オハイオ A stable cell line capable of expressing adeno-associated virus replication genes
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
EP0796339A1 (en) 1994-12-06 1997-09-24 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (en) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc PROCESS FOR PURIFYING VIRUSES BY CHROMATOGRAPHY
AU722196B2 (en) 1995-08-30 2000-07-27 Genzyme Corporation Chromatographic purification of adenovirus and AAV
JPH11514853A (en) 1995-09-08 1999-12-21 ジエンザイム コーポレイション Improved AAV vector for gene therapy
US5795872A (en) * 1995-09-19 1998-08-18 Pharmadigm, Inc. DNA construct for immunization
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
KR20000068501A (en) 1996-09-06 2000-11-25 트러스티스 오브 더 유니버시티 오브 펜실바니아 Method for recombinant adeno-associated virus-directed gene therapy
JP2001514845A (en) 1997-09-05 2001-09-18 ターゲティッド ジェネティクス コーポレイション Method for generating a high titer helper-free preparation of a recombinant AAV vector
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
JPH11318467A (en) 1998-05-08 1999-11-24 Japan Science & Technology Corp Shortened type dystrophin
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP2004514407A (en) 2000-04-28 2004-05-20 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 AAV5 capsid pseudotyped in heterologous capsid and recombinant AAV vector comprising AAV5 vector
AU2001261063A1 (en) 2000-04-28 2001-11-12 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
AU2294102A (en) 2000-07-14 2002-01-30 Univ Michigan Mutant muscle-specific enhancers
EP1366160B1 (en) 2000-10-06 2008-07-09 The Regents Of The University Of Michigan Mini-dystrophin nucleic acid and peptide sequences
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
WO2003052051A2 (en) 2001-12-17 2003-06-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
EP2295604B1 (en) * 2004-02-09 2015-04-08 Thomas Jefferson University Diagnosis and treatment of cancers with microRNA located in or near cancer-associated chromosomal features
US20080044393A1 (en) 2004-07-16 2008-02-21 White Robert L Retinal dystrophin transgene and methods of use thereof
US20100184209A1 (en) 2006-02-17 2010-07-22 Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
WO2008088895A2 (en) 2007-01-18 2008-07-24 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma
CA2694930C (en) 2007-07-31 2019-11-12 Board Of Regents, The University Of Texas System A micro-rna family that modulates fibrosis and uses thereof
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
WO2010071454A1 (en) 2008-12-17 2010-06-24 Auckland Uniservices Limited Adeno-associated viral vectors and uses thereof
AU2012286994B2 (en) 2011-07-25 2017-08-10 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of DUX4
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
JP2015503924A (en) 2012-01-05 2015-02-05 ハダシット メディカル リサーチ サービシーズ アンド ディベロップメント リミテッド Methods and compositions for gene transfer
DE102012007232B4 (en) 2012-04-07 2014-03-13 Susanne Weller Method for producing rotating electrical machines
US9624282B2 (en) 2012-11-26 2017-04-18 The Curators Of The University Of Missouri Microdystrophin peptides and methods for treating muscular dystrophy using the same
JP2015092462A (en) 2013-09-30 2015-05-14 Tdk株式会社 Positive electrode and lithium ion secondary battery using the same
GB201400598D0 (en) 2014-01-14 2014-03-05 Univ Glasgow Materials and methods for modulation of tendon healing
CA2936726C (en) 2014-01-21 2024-04-30 Vrije Universiteit Brussel Muscle-specific nucleic acid regulatory elements and methods and use thereof
JP6202701B2 (en) 2014-03-21 2017-09-27 株式会社日立国際電気 Substrate processing apparatus, semiconductor device manufacturing method, and program
US9439924B2 (en) 2014-04-09 2016-09-13 University Of Houston Therapeutic miRNAs for treating heart and skeletal muscle diseases
US10119140B2 (en) 2014-04-18 2018-11-06 Board Of Regents Of The Nevada System Of Higher Education On Behalf Of The University Of Nevada, Reno Methods for enhancing or decreasing the levels of MIR124 and MIR29 in subjects with muscular dystrophy
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
JP6197169B2 (en) 2014-09-29 2017-09-20 東芝メモリ株式会社 Manufacturing method of semiconductor device
WO2016057975A2 (en) * 2014-10-10 2016-04-14 Research Institute At Nationwide Children's Hospital Guided injections for aav gene transfer to muscle
IL297239B1 (en) 2015-01-16 2024-10-01 Univ Washington Novel micro-dystrophins and related methods of use
GB201507842D0 (en) 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
CA2998636A1 (en) 2015-09-17 2017-03-23 Research Institute At Nationwide Children's Hospital Methods and materials for galgt2 gene therapy
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
MA45477A (en) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital ADENOASSOCIATED VIRUS VECTOR VECTOR MICROARN-29 AND MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY
FI3442600T3 (en) 2016-04-15 2024-05-14 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
AU2019290544A1 (en) 2018-06-18 2021-01-28 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy

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