TW202525835A - 腺相關病毒載體遞送肌肉特異性微小肌縮蛋白以治療肌肉萎縮症 - Google Patents

腺相關病毒載體遞送肌肉特異性微小肌縮蛋白以治療肌肉萎縮症 Download PDF

Info

Publication number
TW202525835A
TW202525835A TW114108403A TW114108403A TW202525835A TW 202525835 A TW202525835 A TW 202525835A TW 114108403 A TW114108403 A TW 114108403A TW 114108403 A TW114108403 A TW 114108403A TW 202525835 A TW202525835 A TW 202525835A
Authority
TW
Taiwan
Prior art keywords
muscle
aav
mhck7
raav
plasmid
Prior art date
Application number
TW114108403A
Other languages
English (en)
Chinese (zh)
Inventor
克拉帕克 路易斯 羅帝諾
傑瑞 R 麥德爾
Original Assignee
美國全美兒童醫院之研究學會
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 美國全美兒童醫院之研究學會 filed Critical 美國全美兒童醫院之研究學會
Publication of TW202525835A publication Critical patent/TW202525835A/zh

Links

Classifications

    • 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
    • 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
    • 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
    • A61K9/0029—Parenteral nutrition; Parenteral nutrition compositions as drug carriers
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; 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
    • 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
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Nutrition Science (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
TW114108403A 2017-03-17 2018-03-19 腺相關病毒載體遞送肌肉特異性微小肌縮蛋白以治療肌肉萎縮症 TW202525835A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762473148P 2017-03-17 2017-03-17
US62/473,148 2017-03-17

Publications (1)

Publication Number Publication Date
TW202525835A true TW202525835A (zh) 2025-07-01

Family

ID=63523321

Family Applications (2)

Application Number Title Priority Date Filing Date
TW114108403A TW202525835A (zh) 2017-03-17 2018-03-19 腺相關病毒載體遞送肌肉特異性微小肌縮蛋白以治療肌肉萎縮症
TW107109334A TWI878204B (zh) 2017-03-17 2018-03-19 腺相關病毒載體遞送肌肉特異性微小肌縮蛋白以治療肌肉萎縮症

Family Applications After (1)

Application Number Title Priority Date Filing Date
TW107109334A TWI878204B (zh) 2017-03-17 2018-03-19 腺相關病毒載體遞送肌肉特異性微小肌縮蛋白以治療肌肉萎縮症

Country Status (33)

Country Link
US (2) US20200199621A1 (enExample)
EP (2) EP4245852A3 (enExample)
JP (3) JP7676111B2 (enExample)
KR (3) KR102683682B1 (enExample)
CN (2) CN110997923B (enExample)
AR (1) AR111292A1 (enExample)
AU (3) AU2018233732B2 (enExample)
BR (1) BR112019019248A2 (enExample)
CA (1) CA3056638A1 (enExample)
CO (1) CO2019011250A2 (enExample)
CY (1) CY1126080T1 (enExample)
DK (1) DK3596222T5 (enExample)
EA (1) EA201992201A1 (enExample)
ES (1) ES2948233T3 (enExample)
FI (1) FI3596222T3 (enExample)
HR (1) HRP20230522T1 (enExample)
HU (1) HUE062476T2 (enExample)
IL (3) IL320791A (enExample)
LT (1) LT3596222T (enExample)
MA (1) MA52112B1 (enExample)
MD (1) MD3596222T2 (enExample)
MX (3) MX2019011046A (enExample)
MY (1) MY203547A (enExample)
PL (1) PL3596222T3 (enExample)
PT (1) PT3596222T (enExample)
RS (1) RS64299B1 (enExample)
SA (1) SA519410146B1 (enExample)
SG (1) SG11201908575SA (enExample)
SI (1) SI3596222T1 (enExample)
SM (1) SMT202300201T1 (enExample)
TW (2) TW202525835A (enExample)
WO (1) WO2018170408A1 (enExample)
ZA (1) ZA201906251B (enExample)

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108463211B (zh) 2015-11-16 2022-09-27 全国儿童医院研究所 用于治疗肌联蛋白类肌病和其它肌联蛋白病变的材料和方法
CN110997923B (zh) * 2017-03-17 2024-01-02 全国儿童医院研究所 腺相关病毒载体递送肌肉特异性微肌营养不良蛋白以治疗肌营养不良症
US11338045B2 (en) 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
MX2020004035A (es) 2017-10-20 2020-11-09 Res Inst Nationwide Childrens Hospital Métodos y materiales para terapia génica con nt-3.
AU2019216257B2 (en) 2018-01-31 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for limb-girdle muscular dystrophy type 2C
IL324506A (en) 2018-06-18 2026-01-01 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector for delivery of muscle-specific microdystrophin for the treatment of muscular dystrophy
IL279789B2 (en) 2018-06-29 2026-04-01 Res Inst Nationwide Childrens Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A
CN113613668A (zh) 2018-10-24 2021-11-05 百时美施贵宝公司 小型化肌营养不良蛋白及其用途
AU2019406214A1 (en) * 2018-12-21 2021-08-05 Northwestern University Use of annexins in preventing and treating muscle membrane injury
US12630840B2 (en) * 2019-02-02 2026-05-19 University of Pittsburgh—of the Commonwealth System of Higher Education One-step gene therapy for Duchenne muscular dystrophy via gene replacement and anti-inflammation
KR20210133242A (ko) 2019-02-26 2021-11-05 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 B-사르코글리칸의 아데노 관련 바이러스 벡터 전달 및 근이영양증의 치료
EP3976806A1 (en) * 2019-05-30 2022-04-06 Solidus Biosciences, Inc. Recombinant herpesvirales vector
CA3150964A1 (en) 2019-08-21 2021-02-25 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
CN119061198A (zh) * 2019-08-29 2024-12-03 美国西门子医学诊断股份有限公司 用于检测aav脱落的试剂和方法
AU2020392243A1 (en) * 2019-11-28 2022-06-16 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
US20230040544A1 (en) * 2019-12-16 2023-02-09 Research Institute At Nationwide Children's Hospital Compositions and methods for restoring and maintaining the dystrophin-associated protein complex (dapc)
US11535868B2 (en) 2020-04-29 2022-12-27 Bristol-Myers Squibb Company Miniaturized dystrophins having spectrin fusion domains and uses thereof
IL300187A (en) * 2020-08-05 2023-03-01 Spacecraft Seven Llc Gene therapy CSRP3 (protein 3 rich in cysteine and glycine)
WO2022029543A1 (en) * 2020-08-06 2022-02-10 Intas Pharmaceuticals Ltd. Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
KR20250099412A (ko) * 2020-11-12 2025-07-01 프리시젼 바이오사이언시스 인코포레이티드 디스트로핀 유전자 내의 인식 서열에 대해 특이성을 갖는 조작된 메가뉴클레아제
EP4086276A1 (en) 2021-05-03 2022-11-09 Université d'Aix-Marseille Composition for treating dysferlinopathy
BR112023024078A2 (pt) * 2021-05-17 2024-01-30 Sarepta Therapeutics Inc Produção de vetores de aav recombinantes para o tratamento de distrofia muscular
EP4108263A3 (en) * 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
WO2023015304A1 (en) * 2021-08-05 2023-02-09 Insmed Incorporated Adeno-associated virus particles and methods of use thereof
JP2024531138A (ja) * 2021-08-11 2024-08-29 ウルトラジェニックス ファーマシューティカル 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組成物および方法
US12285497B2 (en) 2021-10-15 2025-04-29 Research Institute At Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
CN114316070B (zh) * 2021-12-29 2022-11-15 上海勉亦生物科技有限公司 用于治疗肌营养不良症的转基因表达盒
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
CN118460618A (zh) * 2022-04-19 2024-08-09 康霖生物科技（杭州）有限公司 一种用于遗传性凝血因子缺乏病治疗的核酸构建体
WO2023248251A1 (en) * 2022-06-24 2023-12-28 Indian Institute Of Technology Kanpur An optimized aav vector for gene therapy of muscular dystrophy
CN115819546B (zh) * 2022-08-19 2025-12-05 成都金唯科生物科技有限公司 表达微缩抗肌营养不良蛋白基因的腺相关病毒载体及其应用
WO2026076414A1 (en) * 2024-10-03 2026-04-09 General Medicines Llc Compositions and methods for modulating expression of therapeutic transgenes
WO2026080823A1 (en) 2024-10-10 2026-04-16 Research Institute At Nationwide Children's Hospital Methods for diagnosing and treating muscular dystrophy diseases

Family Cites Families (33)

* 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
EP0733103B1 (en) 1993-11-09 2004-03-03 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
DE69433922T2 (de) 1993-11-09 2005-07-28 Medical College Of Ohio, Toledo Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
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
WO1996017947A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
JPH11511326A (ja) 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびａａｖの精製
JPH11514853A (ja) 1995-09-08 1999-12-21 ジエンザイム コーポレイション 遺伝子治療のための改良されたａａｖベクター
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
EP1696036B1 (en) 1996-09-06 2010-04-21 The Trustees of The University of Pennsylvania Use of recombinant adeno-associated virus in the manufacture of a medicament for gene therapy via muscle cells
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
CA2995542A1 (en) 1997-09-05 1999-03-11 Genzyme Corporation Methods for generating high titer helper-free preparations of recombinant aav vectors
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 (ja) 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 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
US20080044393A1 (en) * 2004-07-16 2008-02-21 White Robert L Retinal dystrophin transgene and methods of use thereof
NZ584793A (en) * 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
CN102459595A (zh) * 2009-04-24 2012-05-16 普罗森那技术公司 用于治疗dmd的包含肌苷的寡核苷酸
CA2842798C (en) 2011-07-25 2021-02-23 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
WO2013102904A1 (en) * 2012-01-05 2013-07-11 Hadasit Medical Research Services & Development Ltd. Methods and compositions for gene delivery
BR112014020325A2 (pt) * 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
US9862945B2 (en) * 2013-04-20 2018-01-09 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
AU2015208247B2 (en) * 2014-01-21 2021-05-27 Universiteit Gent Muscle-specific nucleic acid regulatory elements and methods and use thereof
WO2015141521A1 (ja) 2014-03-21 2015-09-24 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及び記録媒体
EP2960336A1 (en) * 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
GB201507842D0 (en) * 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
WO2017181011A1 (en) * 2016-04-15 2017-10-19 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of microrna-29 to treat muscular dystrophy
CN110997923B (zh) * 2017-03-17 2024-01-02 全国儿童医院研究所 腺相关病毒载体递送肌肉特异性微肌营养不良蛋白以治疗肌营养不良症

Also Published As

Publication number Publication date
AU2018233732A1 (en) 2019-10-03
KR20240112964A (ko) 2024-07-19
SA519410146B1 (ar) 2024-11-10
SMT202300201T1 (it) 2023-09-06
CO2019011250A2 (es) 2020-02-28
SG11201908575SA (en) 2019-10-30
EA201992201A1 (ru) 2020-03-19
MX2025007401A (es) 2025-08-01
HUE062476T2 (hu) 2023-11-28
JP7676111B2 (ja) 2025-05-14
BR112019019248A2 (pt) 2020-04-28
FI3596222T3 (fi) 2023-06-08
MD3596222T2 (ro) 2023-08-31
TW201840850A (zh) 2018-11-16
CY1126080T1 (el) 2023-11-15
ZA201906251B (en) 2024-01-31
PT3596222T (pt) 2023-06-20
IL311713B2 (en) 2025-10-01
JP2023053254A (ja) 2023-04-12
AU2025200437A1 (en) 2025-02-13
CN110997923B (zh) 2024-01-02
SI3596222T1 (sl) 2024-05-31
JP2025061676A (ja) 2025-04-11
IL320791A (en) 2025-07-01
DK3596222T5 (da) 2024-09-02
IL269391A (en) 2019-11-28
IL269391B2 (en) 2024-09-01
EP3596222A4 (en) 2021-01-06
MA52112A (fr) 2020-01-22
WO2018170408A1 (en) 2018-09-20
EP4245852A3 (en) 2023-11-22
US20220364117A1 (en) 2022-11-17
IL311713B1 (en) 2025-06-01
AU2018233732B2 (en) 2024-10-24
LT3596222T (lt) 2023-07-25
AR111292A1 (es) 2019-06-26
EP3596222A1 (en) 2020-01-22
CN118497275A (zh) 2024-08-16
EP4245852A2 (en) 2023-09-20
KR102887103B1 (ko) 2025-11-18
IL311713A (en) 2024-05-01
HRP20230522T1 (hr) 2023-08-04
MA52112B1 (fr) 2023-08-31
MX2025007402A (es) 2025-07-01
CA3056638A1 (en) 2018-09-20
MY203547A (en) 2024-07-02
RS64299B1 (sr) 2023-07-31
MX2019011046A (es) 2019-10-17
TWI878204B (zh) 2025-04-01
IL269391B1 (en) 2024-05-01
JP2020513811A (ja) 2020-05-21
AU2024266707B2 (en) 2025-11-27
KR20190130591A (ko) 2019-11-22
EP3596222B1 (en) 2023-04-12
KR102683682B1 (ko) 2024-07-09
ES2948233T3 (es) 2023-09-06
DK3596222T3 (da) 2023-06-19
AU2024266707A1 (en) 2025-01-30
KR20250163423A (ko) 2025-11-20
US20200199621A1 (en) 2020-06-25
PL3596222T3 (pl) 2023-10-09
CN110997923A (zh) 2020-04-10

Similar Documents

Publication Publication Date Title
AU2024266707B2 (en) Adeno-Associated Virus Vector Delivery Of Muscle Specific Micro-Dystrophin To Treat Muscular Dystrophy
AU2023201093B2 (en) Adeno-Associated Virus Vector Delivery Of Micro-Dystrophin To Treat Muscular Dystrophy
AU2019290544B2 (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
US20210393801A1 (en) Adeno-Associated Virus Vector Delivery for Muscular Dystrophies
US20240366789A1 (en) Aav-ids vectors for treatment of mucopolysaccharidosis ii
EA051747B1 (ru) Доставка специфического для мышц микродистрофина аденоассоциированным вирусным вектором для лечения мышечной дистрофии
WO2026041758A1 (en) Modified aav capsid with enhanced transduction efficiency