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 (39)

* Cited by examiner, † Cited by third party
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CA2407309C (en) 2000-04-28 2011-08-02 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
US20030157064A1 (en) * 2001-11-09 2003-08-21 Pascal Neuville Chimeric promoters for controlling expression in muscle cells
WO2004083432A1 (en) * 2003-03-21 2004-09-30 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
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
WO2008018795A1 (en) 2006-08-11 2008-02-14 Prosensa Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders
WO2008088895A2 (en) 2007-01-18 2008-07-24 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
ES2639852T3 (es) 2007-10-26 2017-10-30 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar los trastornos musculares
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
JP2012524540A (ja) 2009-04-24 2012-10-18 プロセンサ テクノロジーズ ビー．ブイ． Ｄｍｄを処置するためのイノシンを含むオリゴヌクレオチド
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
IL315273A (en) 2015-01-16 2024-10-01 Univ Washington Novel micro-dystrophins and related methods of use
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
EP3596112A2 (de) 2017-03-17 2020-01-22 Newcastle University Freisetzung eines adeno-assoziierten virusvektors eines fragments von mikrodystrophin zur behandlung von muskeldystrophie
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
JP7646362B2 (ja) 2018-06-18 2025-03-17 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肉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デリバリー
WO2020086844A1 (en) * 2018-10-24 2020-04-30 Bristol-Myers Squibb Company Miniaturized dystrophins and uses thereof
CN113677697B (zh) * 2019-03-25 2024-06-04 吉尼松公司 使用重叠的aav载体生产大型拟抗肌萎缩蛋白
EP3976806A1 (de) 2019-05-30 2022-04-06 Solidus Biosciences, Inc. Rekombinanter herpesvirusvektor
CA3158281A1 (en) * 2019-10-25 2021-04-29 Audentes Therapeutics, Inc. Compositions and methods for treating glycogen storage disorders
CA3155381A1 (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
EP4320139A1 (de) 2021-04-05 2024-02-14 Solid Biosciences Inc. Rekombinanter herpesvirusvektor
WO2023060269A1 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
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
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
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
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
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
US5308752A (en) 1990-05-23 1994-05-03 Univ. Of Iowa Research Foundation Diagnosis of autosomal muscular dystrophy
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
CA2407309C (en) 2000-04-28 2011-08-02 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof

Also Published As

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

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