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 (40)

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EP1287125B1 (de) * 2000-04-28 2009-07-29 Asklepios Biopharmaceutical, Inc. Für das dystrophin-minigen kodierende dna-sequenzen und verfahren zu deren verwendung
US20030157064A1 (en) * 2001-11-09 2003-08-21 Pascal Neuville 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
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
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
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
EP2203173B1 (de) 2007-10-26 2015-12-23 Academisch Ziekenhuis Leiden Mittel und verfahren zur bekämpfung von muskelleiden
WO2009099326A1 (en) 2008-02-08 2009-08-13 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
EP2421971B1 (de) 2009-04-24 2016-07-06 BioMarin Technologies B.V. Oligonukleotid mit einem inosin zur behandlung von dmd
CN118581086A (zh) 2012-01-27 2024-09-03 比奥马林技术公司 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
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
JP6832280B2 (ja) * 2015-01-16 2021-02-24 ユニバーシティ オブ ワシントンＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｗａｓｈｉｎｇｔｏｎ 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
DK3442602T3 (da) 2016-04-15 2025-05-19 Res Inst Nationwide Childrens Hospital Adeno-associeret virusvektors frigørelse af mikrodystrofin til behandling af muskeldystrofi
CA2971303C (en) 2016-06-21 2026-03-03 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
MA47800A (fr) 2017-03-17 2020-01-22 Univ Newcastle Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire
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
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
BR112021007480A2 (pt) * 2018-10-24 2021-08-10 Bristol-Myers Squibb Company distrofinas miniaturizadas e seus usos
WO2020193636A1 (en) * 2019-03-25 2020-10-01 Genethon Production of large-sized quasidystrophins using overlapping aav vectors
AU2020284255A1 (en) 2019-05-30 2022-01-06 Solid Biosciences Inc. Recombinant herpesvirales vector
CN114828858A (zh) * 2019-10-25 2022-07-29 奥登茨治疗公司 用于治疗糖原贮积症的组合物和方法
WO2021089736A1 (en) 2019-11-06 2021-05-14 Association Institut De Myologie Combined therapy for muscular diseases
TW202206447A (zh) 2020-04-29 2022-02-16 美商必治妥美雅史谷比公司 具血影蛋白(spectrin)融合結構域之微型化肌肉萎縮蛋白及其用途
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
US20240191251A1 (en) 2021-04-05 2024-06-13 Solid Biosciences Inc. Recombinant herpesvirales vector
CA3224482A1 (en) * 2021-08-05 2023-02-09 Brian Kaspar 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
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
KR20260010765A (ko) * 2023-04-14 2026-01-21 프리시젼 바이오사이언시스 인코포레이티드 근육-특이적 발현 카세트

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US5541074A (en) 1986-07-25 1996-07-30 The Children's Medical Center Corporation Antibodies to dystrophin and uses therefor
DE3852304T3 (de) 1987-03-02 1999-07-01 Enzon Labs Inc., Piscataway, N.J. 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
WO1990002909A1 (en) 1988-09-06 1990-03-22 Asarco Incorporated Method and burner for melting copper
US5198346A (en) 1989-01-06 1993-03-30 Protein Engineering Corp. Generation and selection of novel DNA-binding proteins and polypeptides
US5096815A (en) 1989-01-06 1992-03-17 Protein Engineering Corporation 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
US5449616A (en) 1990-05-23 1995-09-12 University Of Iowa Research Foundation Nucleic acid encoding 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
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
EP1287125B1 (de) * 2000-04-28 2009-07-29 Asklepios Biopharmaceutical, Inc. Für das dystrophin-minigen kodierende dna-sequenzen und verfahren zu deren verwendung

Also Published As

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

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