ATE400656T1 - Mini-dystrophin nukleinsäure- und peptidsequenzen - Google Patents

Mini-dystrophin nukleinsäure- und peptidsequenzen

Info

Publication number
ATE400656T1
ATE400656T1 AT01977484T AT01977484T ATE400656T1 AT E400656 T1 ATE400656 T1 AT E400656T1 AT 01977484 T AT01977484 T AT 01977484T AT 01977484 T AT01977484 T AT 01977484T AT E400656 T1 ATE400656 T1 AT E400656T1
Authority
AT
Austria
Prior art keywords
mini
dystrophin
nucleic acid
peptide sequences
peptides
Prior art date
Application number
AT01977484T
Other languages
English (en)
Inventor
Jeffrey Chamberlain
Scott Harper
Original Assignee
Univ Michigan
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 Univ Michigan filed Critical Univ Michigan
Application granted granted Critical
Publication of ATE400656T1 publication Critical patent/ATE400656T1/de

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)
AT01977484T 2000-10-06 2001-10-04 Mini-dystrophin nukleinsäure- und peptidsequenzen ATE400656T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US23884800P 2000-10-06 2000-10-06

Publications (1)

Publication Number Publication Date
ATE400656T1 true ATE400656T1 (de) 2008-07-15

Family

ID=22899572

Family Applications (1)

Application Number Title Priority Date Filing Date
AT01977484T ATE400656T1 (de) 2000-10-06 2001-10-04 Mini-dystrophin nukleinsäure- und peptidsequenzen

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)

Families Citing this family (42)

* Cited by examiner, † Cited by third party
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WO2001083695A2 (en) 2000-04-28 2001-11-08 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
US20070073264A1 (en) * 2003-09-26 2007-03-29 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
US7771993B2 (en) * 2004-01-23 2010-08-10 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
JP2009531021A (ja) * 2006-02-16 2009-09-03 フォンダッチォーネ・セントロ・サン・ラファエル・デル・モンテ・タボール 骨格筋周囲血管芽細胞および心筋中胚葉性血管芽細胞、それらの単離および使用方法
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
JP6047270B2 (ja) 2006-08-11 2016-12-21 バイオマリン テクノロジーズ ベー．フェー． Ｄｎａリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段
WO2008088895A2 (en) 2007-01-18 2008-07-24 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma
NZ584793A (en) 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
JP2011510678A (ja) 2008-02-08 2011-04-07 プロセンサ ホールディング ビーブイ Ｄｎａ反復不安定性関連遺伝性障害を治療するための方法及び手段
EP2119783A1 (de) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Verfahren für effizientes Exon (44)-Skipping bei Duchenne-Muskeldystrophie und entsprechende Mittel
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
CA2862628C (en) 2012-01-27 2021-08-24 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
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
WO2016115543A2 (en) 2015-01-16 2016-07-21 University Of Washington Novel micro-dystrophins and related methods of use
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
CA2971303C (en) 2016-06-21 2026-03-03 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
WO2019078916A1 (en) 2017-10-18 2019-04-25 Research Institute At Nationwide Children's Hospital ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION OF MUSCLE-SPECIFIC MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY
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
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
EP3947427A1 (de) * 2019-03-25 2022-02-09 Genethon Herstellung von grossen quasidystrophinen mit überlappenden aav-vektoren
EP3976806A1 (de) 2019-05-30 2022-04-06 Solidus Biosciences, Inc. Rekombinanter herpesvirusvektor
IL291944A (en) 2019-10-10 2022-06-01 Solid Biosciences Inc Modified aav capsids and uses thereof
AU2020372429A1 (en) * 2019-10-25 2022-04-28 Audentes Therapeutics, Inc. Compositions and methods for treating glycogen storage disorders
WO2021089736A1 (en) 2019-11-06 2021-05-14 Association Institut De Myologie Combined therapy for muscular diseases
AU2020392243A1 (en) 2019-11-28 2022-06-16 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
US11535868B2 (en) 2020-04-29 2022-12-27 Bristol-Myers Squibb Company Miniaturized dystrophins having spectrin fusion domains and uses thereof
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
CA3218333A1 (en) 2021-04-05 2022-10-13 Solid Biosciences Inc. Recombinant herpesvirales vector
WO2023015304A1 (en) * 2021-08-05 2023-02-09 Insmed Incorporated Adeno-associated virus particles and methods of use thereof
EP4413018A1 (de) 2021-10-07 2024-08-14 RegenxBio Inc. Rekombinante adeno-assoziierte viren zur gezielten abgabe
WO2023212643A1 (en) * 2022-04-29 2023-11-02 University Of Washington Method for expressing a muscle-specific gene and cassettes for same
EP4694904A2 (de) 2023-04-13 2026-02-18 REGENXBIO Inc. Targeting von aav-kapsiden, verfahren zur herstellung und verwendung davon
CN121311497A (zh) 2023-04-14 2026-01-09 精密生物科学公司 肌肉特异性表达盒

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US5239060A (en) * 1986-07-25 1993-08-24 The Children's Medical Center Corporation Muscular dystrophy protein, dystrophin
US5541074A (en) * 1986-07-25 1996-07-30 The Children's Medical Center Corporation Antibodies to dystrophin and uses therefor
WO1988006630A1 (en) 1987-03-02 1988-09-07 Genex Corporation Method for the preparation of binding molecules
US5223409A (en) * 1988-09-02 1993-06-29 Protein Engineering Corp. Directed evolution of novel binding proteins
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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
US5449616A (en) * 1990-05-23 1995-09-12 University Of Iowa Research Foundation Nucleic acid encoding dystrophin-associated protein
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
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
AU2555697A (en) * 1996-03-27 1997-10-17 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
WO2001083695A2 (en) 2000-04-28 2001-11-08 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof

Also Published As

Publication number Publication date
EP1366160B1 (de) 2008-07-09
EP1366160A2 (de) 2003-12-03
AU2001296600A1 (en) 2002-04-15
DE60134786D1 (de) 2008-08-21
US8501920B2 (en) 2013-08-06
US20050049219A1 (en) 2005-03-03
US20080167260A1 (en) 2008-07-10
WO2002029056A2 (en) 2002-04-11
US20030216332A1 (en) 2003-11-20
US6869777B2 (en) 2005-03-22
WO2002029056A3 (en) 2003-09-18

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