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)
  • Saccharide Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (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 (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE437944T1 (de) * 2000-04-28 2009-08-15 Asklepios Biopharmaceutical In Für das dystrophin-minigen kodierende dna- sequenzen und verfahren zu deren verwendung
CA2406687A1 (en) * 2001-11-09 2003-05-09 Transgene S.A. Chimeric promoters for controlling expression in muscle cells
CA2524255C (en) 2003-03-21 2014-02-11 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
WO2006039218A2 (en) 2004-09-30 2006-04-13 The Trustees Of The University Of Pennsylvania Perfusion circuit and use therein in targeted delivery of macromolecules
EP1991664B1 (de) * 2006-02-16 2018-03-28 Ospedale San Raffaele S.r.l. Skelettmuskel-periangioblasten und herz-mesangioblasten, verfahren zur isolierung und verwendungen davon
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
AU2007282224B2 (en) 2006-08-11 2013-08-29 Vico Therapeutics B.V. Methods and means for treating DNA repeat instability associated genetic disorders
JP5575486B2 (ja) * 2007-01-18 2014-08-20 ユニヴァーシティ オブ ミズーリー−コロンビア 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
AU2008317566B2 (en) 2007-10-26 2014-05-01 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
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
CA2759899A1 (en) 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
CN112251436A (zh) 2012-01-27 2021-01-22 比奥马林技术公司 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
IL286316B2 (en) 2015-01-16 2023-03-01 Univ Washington New micro-dystrophins and related method of use
AU2017250791B2 (en) 2016-04-15 2022-11-24 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of micro-dystrophin 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
WO2019012336A2 (en) 2017-03-17 2019-01-17 Newcastle University ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY
JP7213238B2 (ja) 2017-10-18 2023-01-26 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達
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
PE20211110A1 (es) * 2018-10-24 2021-06-17 Bristol Myers Squibb Co Distrofinas miniaturizadas y usos de las mismas
US12466864B2 (en) * 2019-03-25 2025-11-11 Genethon Production of large-sized quasidystrophins using overlapping AAV vectors
AU2020284255A1 (en) 2019-05-30 2022-01-06 Solid Biosciences Inc. Recombinant herpesvirales vector
WO2021081338A1 (en) * 2019-10-25 2021-04-29 Audentes Therapeutics, Inc. Compositions and methods for treating glycogen storage disorders
AU2020378619B2 (en) 2019-11-06 2026-03-05 Association Institut De Myologie Combined therapy for muscular diseases
AU2021265103A1 (en) 2020-04-29 2023-01-19 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
US20240191251A1 (en) 2021-04-05 2024-06-13 Solid Biosciences Inc. Recombinant herpesvirales vector
WO2023060269A1 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
US20250312398A1 (en) * 2022-04-29 2025-10-09 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
KR20260010765A (ko) * 2023-04-14 2026-01-21 프리시젼 바이오사이언시스 인코포레이티드 근육-특이적 발현 카세트

Family Cites Families (17)

* Cited by examiner, † Cited by third party
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
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
BR8907069A (pt) 1988-09-06 1991-01-02 Asarco Inc Dispositivo queimador e metodo para fundir cobre
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
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
US5260209A (en) * 1990-05-23 1993-11-09 University Of Iowa Research Foundation Nucleic acids encoding dystrophin-associated proteins
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
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
EP0897425A1 (de) * 1996-03-27 1999-02-24 Dana-Farber Cancer Institute Trio molekuele und deren verwendungen
EP1059354A2 (de) 1999-06-10 2000-12-13 Ceres Incorporated DNS-fragmente mit bestimmter Sequenz und die dadurch kodierte Polypeptide
ATE437944T1 (de) 2000-04-28 2009-08-15 Asklepios Biopharmaceutical In Für das dystrophin-minigen kodierende dna- sequenzen und verfahren zu deren verwendung

Also Published As

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

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