JP5575486B2 - 筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子 - Google Patents

筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子 Download PDF

Info

Publication number
JP5575486B2
JP5575486B2 JP2009546436A JP2009546436A JP5575486B2 JP 5575486 B2 JP5575486 B2 JP 5575486B2 JP 2009546436 A JP2009546436 A JP 2009546436A JP 2009546436 A JP2009546436 A JP 2009546436A JP 5575486 B2 JP5575486 B2 JP 5575486B2
Authority
JP
Japan
Prior art keywords
dystrophin
aav
nnos
muscle
vector
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.)
Active
Application number
JP2009546436A
Other languages
English (en)
Japanese (ja)
Other versions
JP2010516252A5 (enExample
JP2010516252A (ja
Inventor
ドワン ドンシェン
イー ライ
ヨーンピーン ユエ
Original Assignee
ユニヴァーシティ オブ ミズーリー−コロンビア
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 ユニヴァーシティ オブ ミズーリー−コロンビア filed Critical ユニヴァーシティ オブ ミズーリー−コロンビア
Publication of JP2010516252A publication Critical patent/JP2010516252A/ja
Publication of JP2010516252A5 publication Critical patent/JP2010516252A5/ja
Application granted granted Critical
Publication of JP5575486B2 publication Critical patent/JP5575486B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Cardiology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Heart & Thoracic Surgery (AREA)
JP2009546436A 2007-01-18 2008-01-18 筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子 Active JP5575486B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US88112907P 2007-01-18 2007-01-18
US60/881,129 2007-01-18
US99932107P 2007-10-16 2007-10-16
US60/999,321 2007-10-16
PCT/US2008/000717 WO2008088895A2 (en) 2007-01-18 2008-01-18 Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma

Publications (3)

Publication Number Publication Date
JP2010516252A JP2010516252A (ja) 2010-05-20
JP2010516252A5 JP2010516252A5 (enExample) 2011-03-10
JP5575486B2 true JP5575486B2 (ja) 2014-08-20

Family

ID=39636602

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2009546436A Active JP5575486B2 (ja) 2007-01-18 2008-01-18 筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子

Country Status (5)

Country Link
US (1) US7892824B2 (enExample)
EP (1) EP2125006B1 (enExample)
JP (1) JP5575486B2 (enExample)
CN (1) CN101711164B (enExample)
WO (1) WO2008088895A2 (enExample)

Families Citing this family (81)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2596506C (en) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
US8236557B2 (en) * 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
US8889641B2 (en) 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
EP2524037B1 (en) 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
JP6141021B2 (ja) * 2010-02-05 2017-06-07 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル パルボウイルスの形質導入を増強するための組成物および方法
JP6042825B2 (ja) 2011-02-10 2016-12-14 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 改変された形質導入プロファイルを有するウイルスベクターならびにその製造および使用の方法
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
CN103405774A (zh) * 2012-10-24 2013-11-27 尹海芳 一种新型药物靶向运输辅助剂
WO2014081855A1 (en) * 2012-11-20 2014-05-30 Universite De Montreal Methods and compositions for muscular dystrophies
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
WO2014143932A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
IL298049B2 (en) 2013-03-15 2023-10-01 Univ North Carolina Chapel Hill Methods and compounds for ligation of paired AAV glycan vectors
US10907176B2 (en) 2015-01-14 2021-02-02 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
KR102684387B1 (ko) * 2015-01-16 2024-07-11 유니버시티 오브 워싱톤 신규한 마이크로-디스트로핀 및 관련된 사용 방법
EP3258961A4 (en) 2015-02-20 2018-08-22 Board of Regents, The University of Texas System Methods and compositions for attenuated chlamydia as vaccine and vector
GB201507842D0 (en) * 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
EP3831842A1 (en) 2015-09-28 2021-06-09 The University of North Carolina at Chapel Hill Methods and compositions for antibody-evading virus vectors
BR112018007066A2 (pt) 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
SG11201804166YA (en) 2015-11-16 2018-06-28 Res Inst Nationwide Childrens Hospital Materials and methods for treatment of titin-based myopathies and other titinopaties
CN116478254A (zh) 2015-12-14 2023-07-25 北卡罗来纳大学教堂山分校 增强细小病毒载体递送的修饰衣壳蛋白
SG11201809029RA (en) 2016-04-15 2018-11-29 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy
MA45477A (fr) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
CA2971303A1 (en) * 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
EP4011206B1 (en) 2016-06-21 2023-11-01 The Curators of the University of Missouri Modified dystrophin proteins
SG11201811603WA (en) 2016-07-26 2019-02-27 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
CA3073137A1 (en) 2016-08-19 2018-02-22 Jeffrey S. Bartlett Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus
KR20190088561A (ko) 2016-12-07 2019-07-26 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 인터류킨-1 수용체 길항제(IL-1RA) cDNA
EP3592848A1 (en) 2017-03-10 2020-01-15 Genethon Treatment of glycogen storage disease iii
EP3610023B1 (en) 2017-03-15 2023-10-04 The University of North Carolina at Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
EP3596112A2 (en) 2017-03-17 2020-01-22 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
CN110892062A (zh) 2017-05-05 2020-03-17 佛罗里达大学研究基金会 表达耳畸蛋白的组合物和方法
MA50836A (fr) 2017-10-18 2020-08-26 Res Inst Nationwide Childrens Hospital Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique de muscles pour traiter la dystrophie musculaire
SG11202006722RA (en) 2018-01-31 2020-08-28 Res Inst Nationwide Childrens Hospital Gene therapy for limb-girdle muscular dystrophy type 2c
EP3759218A4 (en) 2018-02-28 2021-12-08 The University of North Carolina at Chapel Hill PROCEDURES AND COMPOSITIONS FOR ANTIBODY ELIMINATING VIRUS VECTORS
CN112272672A (zh) 2018-04-03 2021-01-26 斯特里迪比奥公司 抗体逃避性病毒载体
KR20210006358A (ko) 2018-04-03 2021-01-18 스트라이드바이오 인코포레이티드 안 조직을 표적으로 하기 위한 바이러스 벡터
JP2021519581A (ja) 2018-04-03 2021-08-12 ストライドバイオ，インコーポレイテッド 抗体を回避するウイルスベクター
JP7575273B2 (ja) 2018-04-16 2024-10-29 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 デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
CN119410636A (zh) 2018-04-27 2025-02-11 分贝治疗公司 肌球蛋白15启动子及其用途
SG11202010830WA (en) 2018-05-09 2020-11-27 Biomarin Pharm Inc Methods of treating phenylketonuria
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
BR112020025995A2 (pt) * 2018-06-18 2021-03-23 Research Institute At Nationwide Children's Hospital administração de microdistrofina músculo-específica por vetor de vírus adeno-associado para tratar a distrofia muscular
JP2021528959A (ja) 2018-06-22 2021-10-28 アスクレピオス バイオファーマシューティカル， インコーポレイテッド 細胞内で存続する遺伝子送達のためのベクター
EP3814514A1 (en) 2018-06-29 2021-05-05 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
WO2020093018A1 (en) 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
CN114072179B (zh) 2019-02-08 2024-11-01 分贝治疗公司 肌球蛋白15启动子及其用途
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
US12377170B2 (en) 2019-02-26 2025-08-05 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of β-sarcoglycan and the treatment of muscular dystrophy
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
WO2020193636A1 (en) * 2019-03-25 2020-10-01 Genethon Production of large-sized quasidystrophins using overlapping aav vectors
AU2020263392A1 (en) 2019-04-26 2021-10-28 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV2.5 vector
SG11202113052YA (en) 2019-05-30 2021-12-30 Solid Biosciences Inc Recombinant herpesvirales vector
KR20220032595A (ko) * 2019-07-10 2022-03-15 유니버시티 오브 워싱톤 바-바이러스 면역-표적화
DK4017871T5 (da) 2019-08-21 2024-08-05 Res Inst Nationwide Childrens Hospital Adenoassocieret virusvektoradministration af alfa-sarcoglycan og behandling af muskeldystrofi
US11905523B2 (en) 2019-10-17 2024-02-20 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick Disease type-C
CA3155381A1 (en) 2019-11-06 2021-05-14 Association Institut De Myologie Combined therapy for muscular diseases
BR112022009895A2 (pt) * 2019-11-28 2022-08-09 Regenxbio Inc Construtos de terapia de gene de microdistrofina e usos dos mesmos
IL295741A (en) 2020-04-01 2022-10-01 Univ Florida Dual aav-myo7a vectors with improved safety for the treatment of ush1b
CA3174312A1 (en) 2020-04-29 2021-11-04 Glen BANKS Miniaturized dystrophins having spectrin fusion domains and uses thereof
IL300728A (en) 2020-08-19 2023-04-01 Sarepta Therapeutics Inc Adeno-associated virus vectors for the treatment of Rett syndrome
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
AU2021369480B2 (en) 2020-10-28 2025-05-22 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV2.5 vector
WO2022093769A1 (en) 2020-10-28 2022-05-05 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
TW202242124A (zh) 2021-01-14 2022-11-01 美商史崔德生物公司 靶向ｔ細胞之ａａｖ載體
WO2022216749A1 (en) 2021-04-05 2022-10-13 Solid Biosciences Inc. Recombinant herpesvirales vector
EP4323015A4 (en) 2021-04-16 2025-10-22 Askbio Inc RATIONALLY POLYPLOID AAV VIRIONS CROSS THE BLOOD-BRAIN BARRIER AND TRIGGER A REDUCED HUMORAL RESPONSE
WO2022232141A1 (en) * 2021-04-26 2022-11-03 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
US20240390522A1 (en) * 2021-09-28 2024-11-28 University Of Florida Research Foundation, Incorporated Gene therapy for duchenne muscular dystrophy
US20250001006A1 (en) 2021-10-07 2025-01-02 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
CN115960946A (zh) * 2021-10-09 2023-04-14 中国科学院分子植物科学卓越创新中心 调节植物镁离子吸收和转运的方法
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
US20250099620A1 (en) * 2022-01-17 2025-03-27 Dinaqor Ag Gene therapy composition and treatment for dystrophin-related cardiomyopathy
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
TW202342759A (zh) 2022-02-04 2023-11-01 美商史崔德生物公司 重組腺相關病毒載體及其使用方法
WO2023212193A1 (en) * 2022-04-27 2023-11-02 The Curators Of The University Of Missouri Micro-dystrophin for heart protection
WO2024124019A2 (en) 2022-12-07 2024-06-13 Ginkgo Bioworks, Inc. Aav vectors targeting hematopoietic stem cells
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
WO2025017168A1 (en) * 2023-07-19 2025-01-23 Genethon Novel optimized utrophin micro-genes

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2330615T3 (es) * 2000-04-28 2009-12-14 Asklepios Biopharmaceutical, Inc. Secuencias de adn que codifican para minigenes de distrofina y metodos de uso de las mismas.
US6869777B2 (en) 2000-10-06 2005-03-22 Regents Of The University Of Michigan Mini-dystrophin nucleic acid sequences

Also Published As

Publication number Publication date
WO2008088895A3 (en) 2008-10-30
HK1143326A1 (en) 2010-12-31
CN101711164A (zh) 2010-05-19
US7892824B2 (en) 2011-02-22
EP2125006A2 (en) 2009-12-02
EP2125006B1 (en) 2013-10-16
CN101711164B (zh) 2014-06-04
WO2008088895A2 (en) 2008-07-24
US20080249052A1 (en) 2008-10-09
JP2010516252A (ja) 2010-05-20
EP2125006A4 (en) 2010-11-24
WO2008088895A9 (en) 2008-09-18

Similar Documents

Publication Publication Date Title
JP5575486B2 (ja) 筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子
JP7761686B2 (ja) 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
CN100422320C (zh) 编码抗肌萎缩蛋白小基因的dna序列及其使用方法
US20240318149A1 (en) AAV Vectors
JP6832280B2 (ja) 新規のマイクロジストロフィンおよび使用の関連する方法
CN107295802B (zh) 用于高效基因组编辑的腺相关病毒载体变异体和其方法
CN107429252B (zh) 优化的rpe65启动子和编码序列
ES2946512T3 (es) Terapia génica para el tratamiento de una enfermedad degenerativa de la retina
JP7285022B2 (ja) 組換えヒトＩＩ型ミトコンドリアダイニン様ＧＴＰａｓｅの遺伝子配列及びその使用
JP2022505816A (ja) 小型化ジストロフィンおよびそれらの使用
EP3471544A1 (en) Modified dystrophin proteins
KR20240004564A (ko) 디스트로핀병증의 치료를 위한 마이크로디스트로핀 유전자 치료법 투여
CA3088079A1 (en) Compositions and methods for treating retinal disorders
EP4337779A1 (en) Vector system
KR20230003557A (ko) 스펙트린 융합 도메인을 갖는 소형화 디스트로핀 및 그의 용도
HK1143326B (en) Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma
KR20250096825A (ko) 목적 유전자용 발현 카세트 및 이의 용도
TW202134260A (zh) 微小肌縮蛋白基因療法之構築體及其用途
CA3219795A1 (en) Recombinant tert-encoding viral genomes and vectors
HK40104471A (zh) 用於目的基因的表达盒及其应用
EA046419B1 (ru) Миниатюризированные дистрофины и их применения

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20100119

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20110118

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20110118

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20130304

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20130603

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20130610

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20130904

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20140602

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20140702

R150 Certificate of patent or registration of utility model

Ref document number: 5575486

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250