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AU2001296600A1 - Mini-dystrophin nucleic acid and peptide sequences - Google Patents

Mini-dystrophin nucleic acid and peptide sequences

Info

Publication number
AU2001296600A1
AU2001296600A1 AU2001296600A AU9660001A AU2001296600A1 AU 2001296600 A1 AU2001296600 A1 AU 2001296600A1 AU 2001296600 A AU2001296600 A AU 2001296600A AU 9660001 A AU9660001 A AU 9660001A AU 2001296600 A1 AU2001296600 A1 AU 2001296600A1
Authority
AU
Australia
Prior art keywords
mini
dystrophin
nucleic acid
peptide sequences
peptides
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.)
Abandoned
Application number
AU2001296600A
Other languages
English (en)
Inventor
Jeffrey S. Chamberlain
Scott Q. Harper
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 Michigan System
Original Assignee
University of Michigan System
University of Michigan Ann Arbor
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 Michigan System, University of Michigan Ann Arbor filed Critical University of Michigan System
Publication of AU2001296600A1 publication Critical patent/AU2001296600A1/en
Abandoned legal-status Critical Current

Links

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
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/022—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from an adenovirus

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
AU2001296600A 2000-10-06 2001-10-04 Mini-dystrophin nucleic acid and peptide sequences Abandoned AU2001296600A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US23884800P 2000-10-06 2000-10-06
US60/238,848 2000-10-06
PCT/US2001/031126 WO2002029056A2 (en) 2000-10-06 2001-10-04 Mini-dystrophin nucleic acid and peptide sequences

Publications (1)

Publication Number Publication Date
AU2001296600A1 true AU2001296600A1 (en) 2002-04-15

Family

ID=22899572

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2001296600A Abandoned AU2001296600A1 (en) 2000-10-06 2001-10-04 Mini-dystrophin nucleic acid and peptide sequences

Country Status (6)

Country Link
US (3) US6869777B2 (de)
EP (1) EP1366160B1 (de)
AT (1) ATE400656T1 (de)
AU (1) AU2001296600A1 (de)
DE (1) DE60134786D1 (de)
WO (1) WO2002029056A2 (de)

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AU2001261063A1 (en) 2000-04-28 2001-11-12 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
CA2406687A1 (en) * 2001-11-09 2003-05-09 Transgene S.A. Chimeric promoters for controlling expression in muscle cells
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
WO2005030292A2 (en) 2003-09-26 2005-04-07 The Trustees Of The University Of Pennsylvania Methods, compositions and apparatus for delivering heterologous molecules to cells
WO2005049850A2 (en) 2003-11-14 2005-06-02 University Of Washington Compositions and methods for systemic nucleic acid sequence delivery
WO2005118611A2 (en) * 2004-01-23 2005-12-15 The Trustees Of The University Of Pennsylvania Microutrophin and uses thereof
US20090054823A1 (en) 2004-09-30 2009-02-26 The Trustees Of The University Of Pennsylvania Perfusion circuit and use therein in targeted delivery of macromolecules
WO2007093412A2 (en) * 2006-02-16 2007-08-23 Fondazione Centro San Raffaele Del Monte Tabor Skeletal muscle periangioblasts and cardiac mesoangioblasts, method for isolation and uses thereof
WO2007123391A1 (en) 2006-04-20 2007-11-01 Academisch Ziekenhuis Leiden Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene.
EP1857548A1 (de) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Mittel und Verfahren zur Induktion von Exon-skipping
PT2049664E (pt) 2006-08-11 2012-01-03 Prosensa Technologies Bv Oligonucleotídeos de cadeia simples complementares dos elementos repetitivos para tratar perturbações genéticas associadas à instabilidade das repetições do adn
US7892824B2 (en) * 2007-01-18 2011-02-22 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
PL2203173T3 (pl) 2007-10-26 2016-06-30 Academisch Ziekenhuis Leiden Środki i sposoby przeciwdziałania zaburzeniom mięśni
NZ587178A (en) 2008-02-08 2011-11-25 Prosensa Holding Bv Methods and means for treating dna repeat instability associated genetic disorders
EP2119783A1 (de) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Verfahren für effizientes Exon (44)-Skipping bei Duchenne-Muskeldystrophie und entsprechende Mittel
WO2010123369A1 (en) 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
JP2015509922A (ja) 2012-01-27 2015-04-02 プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド
GB201212456D0 (en) 2012-07-12 2012-08-29 Univ Sheffield Treatment of muscular dystrophy
EP2960336A1 (de) * 2014-06-27 2015-12-30 Genethon Effiziente systemische Behandlung von Muskeldystrophie
CN107250364A (zh) 2015-01-16 2017-10-13 华盛顿大学 新的微小肌养蛋白及相关使用方法
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
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
EP3697915A4 (de) 2017-10-18 2021-12-08 Research Institute at Nationwide Children's Hospital Adeno-assoziierte virusvektorverabreichung von muskelspezifischem mikrodystrophin zur behandlung von muskeldystrophie
CN120393053A (zh) 2018-06-18 2025-08-01 全国儿童医院研究所 用于治疗肌营养不良的肌肉特异性微小肌营养不良蛋白的腺相关病毒载体递送
US20210403947A1 (en) 2018-10-24 2021-12-30 Bristol-Myers Squibb Company Miniaturized dystrophins and uses thereof
JP7635139B2 (ja) * 2019-03-25 2025-02-25 ジェネトン オーバーラップａａｖベクターを使用する大きいサイズのクアシジストロフィンの産生
JP2022534119A (ja) 2019-05-30 2022-07-27 ソリッド・バイオサイエンシーズ・インコーポレーテッド 組換えヘルペスウイルスベクター
EP4048286A4 (de) * 2019-10-25 2023-12-06 Audentes Therapeutics, Inc. Zusammensetzungen und verfahren zum behandeln von glykogenlagerungsstörungen
EP4054618A1 (de) 2019-11-06 2022-09-14 Association Institut de Myologie Kombinationstherapie bei muskelerkrankungen
BR112022021635A2 (pt) 2020-04-29 2022-12-06 Bristol Myers Squibb Co Distrofinas miniaturizadas tendo domínios de fusão de espectrina e usos das mesmas
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
RU2767335C1 (ru) * 2021-03-02 2022-03-17 Общество с ограниченной ответственностью «Марлин Биотех» Химерные белки на основе утрофина и дистрофина человека и их применение для лечения миодистрофии Дюшенна
WO2022216749A1 (en) 2021-04-05 2022-10-13 Solid Biosciences Inc. Recombinant herpesvirales vector
EP4413018A1 (de) 2021-10-07 2024-08-14 RegenxBio Inc. Rekombinante adeno-assoziierte viren zur gezielten abgabe
US20250312398A1 (en) * 2022-04-29 2025-10-09 University Of Washington Method for expressing a muscle-specific gene and cassettes for same
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
WO2024216118A1 (en) * 2023-04-14 2024-10-17 Precision Biosciences, Inc. Muscle-specific expression cassettes

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Publication number Priority date Publication date Assignee Title
US5541074A (en) * 1986-07-25 1996-07-30 The Children's Medical Center Corporation Antibodies to dystrophin and uses therefor
US5239060A (en) * 1986-07-25 1993-08-24 The Children's Medical Center Corporation Muscular dystrophy protein, dystrophin
ATE114723T1 (de) 1987-03-02 1994-12-15 Enzon Lab Inc Organismus als träger für ''single chain antibody domain (scad)''.
US5223409A (en) * 1988-09-02 1993-06-29 Protein Engineering Corp. Directed evolution of novel binding proteins
FI901749A0 (fi) 1988-09-06 1990-04-06 Asarco Inc Foerfarande och braennare foer smaeltning av koppar.
US5096815A (en) * 1989-01-06 1992-03-17 Protein Engineering Corporation Generation and selection of novel dna-binding proteins and polypeptides
US5198346A (en) * 1989-01-06 1993-03-30 Protein Engineering Corp. Generation and selection of novel DNA-binding proteins and polypeptides
US5308752A (en) * 1990-05-23 1994-05-03 Univ. Of Iowa Research Foundation Diagnosis of autosomal muscular dystrophy
US5686073A (en) * 1990-05-23 1997-11-11 The University Of Iowa Research Foundation Polyclonal and monoclonal antibodies against a 43 KDA dystrophin associated protein
US5260209A (en) * 1990-05-23 1993-11-09 University Of Iowa Research Foundation Nucleic acids encoding dystrophin-associated proteins
US5449616A (en) * 1990-05-23 1995-09-12 University Of Iowa Research Foundation Nucleic acid encoding dystrophin-associated protein
US5430129A (en) * 1991-01-16 1995-07-04 University Of Iowa Research Foundation Purified, native dystrophin
US5863743A (en) * 1994-08-12 1999-01-26 University Of Iowa Research Foundation Merosin deficiency-type congenital muscular dystrophy
GB9622174D0 (en) 1995-12-19 1996-12-18 Medical Res Council Gene expression
WO1997035979A1 (en) * 1996-03-27 1997-10-02 Dana-Farber Cancer Institute Novel trio molecules and uses related thereto
EP1059354A2 (de) 1999-06-10 2000-12-13 Ceres Incorporated DNS-fragmente mit bestimmter Sequenz und die dadurch kodierte Polypeptide
AU2001261063A1 (en) 2000-04-28 2001-11-12 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof

Also Published As

Publication number Publication date
US8501920B2 (en) 2013-08-06
US20050049219A1 (en) 2005-03-03
ATE400656T1 (de) 2008-07-15
EP1366160A2 (de) 2003-12-03
US6869777B2 (en) 2005-03-22
WO2002029056A3 (en) 2003-09-18
EP1366160B1 (de) 2008-07-09
US20080167260A1 (en) 2008-07-10
DE60134786D1 (de) 2008-08-21
US20030216332A1 (en) 2003-11-20
WO2002029056A2 (en) 2002-04-11

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