CN101711164B - 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因 - Google Patents

可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因 Download PDF

Info

Publication number
CN101711164B
CN101711164B CN200880002602.3A CN200880002602A CN101711164B CN 101711164 B CN101711164 B CN 101711164B CN 200880002602 A CN200880002602 A CN 200880002602A CN 101711164 B CN101711164 B CN 101711164B
Authority
CN
China
Prior art keywords
dystrophin
nnos
gene
aav
seq
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
CN200880002602.3A
Other languages
English (en)
Chinese (zh)
Other versions
CN101711164A (zh
Inventor
段东升
赖毅
岳永平
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Missouri Columbia
Original Assignee
University of Missouri Columbia
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 University of Missouri Columbia filed Critical University of Missouri Columbia
Publication of CN101711164A publication Critical patent/CN101711164A/zh
Application granted granted Critical
Publication of CN101711164B publication Critical patent/CN101711164B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707Muscular dystrophy
    • C07K14/4708Duchenne dystrophy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/40Systems 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)
  • Orthopedic Medicine & Surgery (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Cardiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hospice & Palliative Care (AREA)
  • Heart & Thoracic Surgery (AREA)
CN200880002602.3A 2007-01-18 2008-01-18 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因 Active CN101711164B (zh)

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 (2)

Publication Number Publication Date
CN101711164A CN101711164A (zh) 2010-05-19
CN101711164B true CN101711164B (zh) 2014-06-04

Family

ID=39636602

Family Applications (1)

Application Number Title Priority Date Filing Date
CN200880002602.3A Active CN101711164B (zh) 2007-01-18 2008-01-18 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因

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
AU2006213686A1 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, 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
ES2634118T3 (es) 2009-02-11 2017-09-26 The University Of North Carolina At Chapel Hill Vectores de virus modificados y métodos para fabricar y utilizar los mismos
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
CA2788682C (en) 2010-02-05 2019-03-05 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
EP4234571A3 (en) 2011-02-10 2023-09-27 The University of North Carolina at Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
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
CA2904396A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav vectors
EP3517612A1 (en) 2013-03-15 2019-07-31 The University of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
WO2016115382A1 (en) 2015-01-14 2016-07-21 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
AU2016206518B2 (en) 2015-01-16 2020-03-05 University Of Washington Novel micro-dystrophins and related methods of use
CA2977071A1 (en) 2015-02-20 2016-08-25 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
MA45819A (fr) 2015-10-09 2018-08-15 Sarepta Therapeutics Inc Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés
KR102877920B1 (ko) 2015-11-16 2025-10-30 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법
JP7406783B2 (ja) 2015-12-14 2023-12-28 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質
HUE066442T2 (hu) 2016-04-15 2024-08-28 Res Institute At Nationwide ß-szarkoglikán és mikroRNS-29 adenoasszociáltvírus-vektorral történõ bejuttatása és izomdisztrófia kezelése
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
IL263801B2 (en) 2016-07-26 2024-01-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
KR102759317B1 (ko) 2016-08-19 2025-01-31 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 재조합 자기-상보적인 아데노-부속 바이러스를 이용한 병태의 치료 방법 및 조성물
MX2019006614A (es) 2016-12-07 2019-10-15 Univ Florida Adnc del antagonista del receptor de interleucina-1.
EP3592848A1 (en) 2017-03-10 2020-01-15 Genethon Treatment of glycogen storage disease iii
EP4303225B1 (en) 2017-03-15 2025-11-26 The University of North Carolina at Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
JP7162021B2 (ja) 2017-03-17 2022-10-27 ニューカッスル ユニバーシティ 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達
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
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
SG11202006722RA (en) 2018-01-31 2020-08-28 Res Inst Nationwide Childrens Hospital Gene therapy for limb-girdle muscular dystrophy type 2c
CN112041442A (zh) 2018-02-28 2020-12-04 北卡罗来纳大学查佩尔希尔分校 用于逃避抗体的病毒载体的方法和组合物
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
WO2019195449A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
EP3780952A4 (en) 2018-04-16 2022-03-09 The Trustees of the University of Pennsylvania COMPOSITIONS AND METHODS OF TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
JP7331011B2 (ja) 2018-04-27 2023-08-22 デシベル セラピューティクス インコーポレイテッド ミオシン15プロモーター及びその使用
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
WO2019217513A2 (en) 2018-05-09 2019-11-14 Biomarin Pharmaceutical 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
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
MY208145A (en) 2018-06-18 2025-04-18 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
WO2019246544A2 (en) 2018-06-22 2019-12-26 Asklepios Biopharmaceutical, Inc. Vectors for gene delivery that persist within cells
KR20210028162A (ko) 2018-06-29 2021-03-11 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법
WO2020093018A1 (en) 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
CA3129422A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
WO2020163743A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
CA3131390A1 (en) 2019-02-26 2020-09-03 Research Institute Of Nationwide Children's Hospital Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
CA3130196A1 (en) * 2019-03-25 2020-10-01 Isabelle Richard Production of large-sized quasidystrophins using overlapping aav vectors
WO2020219656A1 (en) 2019-04-26 2020-10-29 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
CN114207120A (zh) 2019-05-30 2022-03-18 坚固生物科技公司 重组疱疹病毒载体
AU2020311399A1 (en) * 2019-07-10 2022-02-17 University Of Washington Non-viral immuno-targeting
HRP20240505T1 (hr) 2019-08-21 2024-07-05 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana vektorom povezanim s adenovirusom i liječenje mišićne distrofije
TW202128736A (zh) 2019-10-17 2021-08-01 美商史崔德生物公司 用於治療c型尼曼—匹克病之腺相關病毒載體
AU2020378619A1 (en) 2019-11-06 2022-06-09 Association Institut De Myologie Combined therapy for muscular diseases
KR20220107222A (ko) * 2019-11-28 2022-08-02 리젠엑스바이오 인크. 마이크로디스트로핀 유전자 치료 작제물 및 이의 용도
CN115666658A (zh) 2020-04-01 2023-01-31 佛罗里达州大学研究基金会 具有提高的治疗ush1b的安全性的双aav-myo7a载体
MX2022013477A (es) * 2020-04-29 2022-11-30 Bristol Myers Squibb Co Distrofinas miniaturizadas con dominios de fusion de espectrina y usos de las mismas.
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
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
JP2023547992A (ja) 2020-10-28 2023-11-15 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 二重グリカン結合aav2.5ベクターのための方法および組成物
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
WO2022221529A1 (en) 2021-04-16 2022-10-20 Asklepios Biopharmaceutical, Inc. Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response
CA3216744A1 (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
WO2023060269A1 (en) 2021-10-07 2023-04-13 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
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
CA3248878A1 (en) * 2022-01-17 2023-07-20 Dinaqor Ag GENE THERAPY COMPOSITION AND TREATMENT OF DYSTROPHIN-RELATED CARDIOMYOPATHY
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
WO2023150687A1 (en) 2022-02-04 2023-08-10 Ginkgo Bioworks, Inc. Recombinant adeno-associated virus vectors, and methods of use thereof
US20250288641A1 (en) * 2022-04-27 2025-09-18 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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1439051A (zh) * 2000-04-28 2003-08-27 肖啸 编码抗肌萎缩蛋白小基因的dna序列及其使用方法

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE60134786D1 (de) 2000-10-06 2008-08-21 Univ Michigan Mini-dystrophin nukleinsäure- und peptidsequenzen

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1439051A (zh) * 2000-04-28 2003-08-27 肖啸 编码抗肌萎缩蛋白小基因的dna序列及其使用方法

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
LAI Y ET AL.Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors.《NATURE BIOTECHNOLOGY》.2005, *
LI SHENG ET AL.A highly functional mini-dystrophin/GFP fusion gene for cell and gene therapy studies of Duchenne muscular dystrophy.《HUMAN MOLECULAR GENETICS》.2006, *
RAFAEL J A ET AL.FORCED EXPRESSION OF DYSTROPHIN DELETION CONSTRCTS REVEALS STRUCTURE-FUNCTION CORRELATIONS.《THE JOURNAL OF CELL BIOLOGY》.1996, *
RAFAEL J A ET AL.PREVENTION OF DYSTROPHIC PATHOLOGY IN MDX MICE BY A TRUNCATED DYSTROPHIN ISOFORM.《HUMAN MOLECULAR GENETICS》.1994, *
SCOTT JEANNINE M ET AL.Viral vectors for gene transfer of micro-, mini-, or full-length dystrophin.《NEUROMUSCULAR DISORDERS》.2002, *
WELLS KIM E ET AL.Relocalization of neuronal nitric oxide synthase (nNOS) as a marker for complete restoration of the dystrophin associated protein complex in skeletal muscle.《NEUROMUSCULAR DISORDERS》.2003, *
Yi Lai et al.Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy.《The Journal of Clinical Investigation》.2009, *

Also Published As

Publication number Publication date
CN101711164A (zh) 2010-05-19
US7892824B2 (en) 2011-02-22
HK1143326A1 (en) 2010-12-31
WO2008088895A9 (en) 2008-09-18
WO2008088895A2 (en) 2008-07-24
EP2125006B1 (en) 2013-10-16
US20080249052A1 (en) 2008-10-09
JP5575486B2 (ja) 2014-08-20
JP2010516252A (ja) 2010-05-20
WO2008088895A3 (en) 2008-10-30
EP2125006A4 (en) 2010-11-24
EP2125006A2 (en) 2009-12-02

Similar Documents

Publication Publication Date Title
CN101711164B (zh) 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因
CN100422320C (zh) 编码抗肌萎缩蛋白小基因的dna序列及其使用方法
US8236557B2 (en) Hybrid-AAV vectors to deliver large gene expression cassette
CN107250364A (zh) 新的微小肌养蛋白及相关使用方法
CA3078277A1 (en) Aav vectors
US20220175964A1 (en) Dystrophin r16/r17 syntrophin pdz fusion proteins
JP2022505816A (ja) 小型化ジストロフィンおよびそれらの使用
JP2023510784A (ja) 脳腫瘍を処置するための血液脳関門を横断する免疫療法剤を送達するための方法および組成物
US20230407331A1 (en) Miniaturized dystrophins having spectrin fusion domains and uses thereof
HK1143326B (en) Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma
WO2023124741A1 (zh) 用于治疗肌营养不良症的转基因表达盒
US20250099618A1 (en) Recombinant tert-encoding viral genomes and vectors
EA046419B1 (ru) Миниатюризированные дистрофины и их применения
CN117980490A (zh) 通过多种载体的共同递送产生大型蛋白质
Class et al. Patent application title: Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma Inventors: Dongsheng Duan (Columbia, MO, US) Yi Lai (Columbia, MO, US) Yongping Yue (Columbia, MO, US)

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 1143326

Country of ref document: HK

C14 Grant of patent or utility model
GR01 Patent grant
REG Reference to a national code

Ref country code: HK

Ref legal event code: GR

Ref document number: 1143326

Country of ref document: HK