[go: up one dir, main page]

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 (fr) 2000-10-06 2001-10-04 Sequences peptidiques et d'acide nucleique de mini-dystrophine

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 (fr)
EP (1) EP1366160B1 (fr)
AT (1) ATE400656T1 (fr)
AU (1) AU2001296600A1 (fr)
DE (1) DE60134786D1 (fr)
WO (1) WO2002029056A2 (fr)

Families Citing this family (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN100422320C (zh) * 2000-04-28 2008-10-01 肖啸 编码抗肌萎缩蛋白小基因的dna序列及其使用方法
US20030157064A1 (en) * 2001-11-09 2003-08-21 Pascal Neuville Chimeric promoters for controlling expression in muscle cells
CA2524255C (fr) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation de la reconnaissance d'exons dans le pre-arnm par interference avec la structure d'arn secondaire
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
US7655467B2 (en) 2003-11-14 2010-02-02 The University Of Washington Compositions and methods for systemic nucleic acid sequence delivery
WO2005118611A2 (fr) * 2004-01-23 2005-12-15 The Trustees Of The University Of Pennsylvania Micro-utrophine et utilisations
WO2006039218A2 (fr) 2004-09-30 2006-04-13 The Trustees Of The University Of Pennsylvania Circuit de perfusion et son utilisation dans l'administration ciblee de macromolecules
WO2007093412A2 (fr) * 2006-02-16 2007-08-23 Fondazione Centro San Raffaele Del Monte Tabor Periangioblastes du muscle squelettique et mesangioblastes du muscle cardiaque, procede d'isolation et leurs utilisations
WO2007123391A1 (fr) 2006-04-20 2007-11-01 Academisch Ziekenhuis Leiden Intervention thérapeutique dans une maladie génétique chez un individu en modifiant l'expression d'un gène exprimé de manière aberrante.
EP1857548A1 (fr) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Procédé et moyen permettant d'induire un saut d'exon
WO2008018795A1 (fr) 2006-08-11 2008-02-14 Prosensa Technologies B.V. Procédés et moyens de traitement de troubles génétiques associés à l'instabilité des répétitions de l'ADN
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
PT2203173E (pt) 2007-10-26 2016-03-15 Academisch Ziekenhuis Leiden Resumo
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
EP2249874A1 (fr) 2008-02-08 2010-11-17 ProSensa Holding BV Méthodes et moyens pour traiter les affections génétiques associées à l instabilité de répétition d adn
EP2119783A1 (fr) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Procédé pour l'omission efficace de l'exon (44) dans la dystrophie musculaire de Duchenne et moyens connexes
NZ595955A (en) 2009-04-24 2012-10-26 Prosensa Technologies Bv Oligonucleotide comprising an inosine for treating dmd
NZ627896A (en) 2012-01-27 2016-11-25 Biomarin 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 (fr) 2014-06-27 2015-12-30 Genethon Traitement systémique efficace de pathologies dystrophiques musculaires
AU2016206518B2 (en) 2015-01-16 2020-03-05 University Of 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 (fr) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Genes de mini-dystrophine optimises et cassettes d'expression et leur utilisation
JP7162021B2 (ja) 2017-03-17 2022-10-27 ニューカッスル ユニバーシティ 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達
US11534501B2 (en) 2017-10-18 2022-12-27 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
MY208145A (en) 2018-06-18 2025-04-18 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
BR112021007480A2 (pt) 2018-10-24 2021-08-10 Bristol-Myers Squibb Company distrofinas miniaturizadas e seus usos
CA3130196A1 (fr) * 2019-03-25 2020-10-01 Isabelle Richard Production de quasi-dystrophines de grande taille a l'aide de vecteurs aav chevauchants
CN114207120A (zh) 2019-05-30 2022-03-18 坚固生物科技公司 重组疱疹病毒载体
KR20220105643A (ko) * 2019-10-25 2022-07-27 오덴테스 테라퓨틱스, 인크. 글리코겐 축적 장애를 치료하기 위한 조성물 및 방법
AU2020378619A1 (en) 2019-11-06 2022-06-09 Association Institut De Myologie Combined therapy for muscular diseases
MX2022013477A (es) 2020-04-29 2022-11-30 Bristol Myers Squibb Co Distrofinas miniaturizadas con dominios de fusion de espectrina y usos de las mismas.
WO2022076750A2 (fr) 2020-10-07 2022-04-14 Regenxbio Inc. Virus adéno-associés recombinants pour administration musculaire ou dans le snc
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 (fr) 2021-04-05 2022-10-13 Solid Biosciences Inc. Vecteur du virus de l'herpès simplex recombinant
WO2023060269A1 (fr) 2021-10-07 2023-04-13 Regenxbio Inc. Virus adéno-associés recombinants pour administration ciblée
WO2023212643A1 (fr) * 2022-04-29 2023-11-02 University Of Washington Procédé d'expression d'un gène spécifique d'un muscle et cassettes associées
WO2024216244A2 (fr) 2023-04-13 2024-10-17 Regenxbio Inc. Ciblage de capsides d'aav, méthodes de fabrication et d'utilisation de ceux-ci
AU2024255934A1 (en) * 2023-04-14 2025-11-20 Precision Biosciences, Inc. Muscle-specific expression cassettes

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
WO1990002909A1 (fr) 1988-09-06 1990-03-22 Asarco Incorporated Procede et bruleur permettant de fondre le cuivre
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
AU2555697A (en) * 1996-03-27 1997-10-17 Dana-Farber Cancer Institute Novel trio molecules and uses related thereto
EP1059354A2 (fr) 1999-06-10 2000-12-13 Ceres Incorporated Fragments d'ADN avec des séquences déterminées et polypeptides encodées par lesdits fragments
CN100422320C (zh) 2000-04-28 2008-10-01 肖啸 编码抗肌萎缩蛋白小基因的dna序列及其使用方法

Also Published As

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

Similar Documents

Publication Publication Date Title
AU2001296600A1 (en) Mini-dystrophin nucleic acid and peptide sequences
WO2001075087A8 (fr) Variantes de lasubtilisine
WO2005121331A8 (fr) Polypeptides galnact2 tronques et acides nucleiques
WO2007095350A3 (fr) Compositions et procedes utilisant des fragments de peptide du facteur derive d'epithelium pigmentaire (pedf)
EP1159441B8 (fr) Administration de substances a des cellules
WO2002006457A3 (fr) Genes de lipase
WO2006083276A3 (fr) Polypeptides et conjugues interferon-alpha
WO2002022821A3 (fr) Peptides antimicrobiens et methodes d'utilisation
WO2005052143A3 (fr) Adenovirus, acides nucleiques les codant et utilisations associees
MX2010004371A (es) Proteasa de streptomyces.
WO2005113592A3 (fr) Polypeptides et conjugues interferon-alpha
DE60323210D1 (en) Eukaryontische signalsequenzen für polypeptid-expr
AU2002217010A1 (en) Peptides, the production and use thereof for binding immunoglobulins
WO2004046365A3 (fr) Polypeptides et conjugues interferon-alpha
WO2004024750A3 (fr) Ligands liant cd44
IL175021A0 (en) Chromatin insulator, its preparation and use in protein expression
WO2007044083A3 (fr) Polypeptides d'interferon-alpha mis au point genetiquement
WO2002036627A3 (fr) Interferons, utilisations et compositions associees
ATE337336T1 (de) Nukleinsäuren, die für stichodactylidae chromoproteine kodieren
AU2003287115A1 (en) Fluorescent proteins from copepoda species and methods for using same
WO2001049721A3 (fr) Nouveaux genes bacteriens et proteines qui sont essentielles pour la viabilite cellulaire et leurs utilisations
HK1039793A1 (zh) 一種細胞凋亡蛋白酶天冬氨酸特異性半胱氨酸蛋白酶-14，編碼核酸及其使用方法
WO2002081623A3 (fr) Acides nucleiques a cycle cellulaire, polypeptides et utilisations correspondantes
WO2007029262A3 (fr) Compositions et methodes d'utilisation de ces dernieres en vue de detecter des virus
AU2001238064A1 (en) 18477, a human protein kinase and uses therefor