DE60139394D1 - Für das dystrophin-minigen kodierende dna-sequenzen und verfahren zu deren verwendung - Google Patents

Für das dystrophin-minigen kodierende dna-sequenzen und verfahren zu deren verwendung

Info

Publication number
DE60139394D1
DE60139394D1 DE60139394T DE60139394T DE60139394D1 DE 60139394 D1 DE60139394 D1 DE 60139394D1 DE 60139394 T DE60139394 T DE 60139394T DE 60139394 T DE60139394 T DE 60139394T DE 60139394 D1 DE60139394 D1 DE 60139394D1
Authority
DE
Germany
Prior art keywords
dystrophin
present
provides
minigene
same
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.)
Expired - Lifetime
Application number
DE60139394T
Other languages
English (en)
Inventor
Xiao Xiao
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.)
Asklepios Biopharmaceutical Inc
Original Assignee
Asklepios Biopharmaceutical Inc
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 Asklepios Biopharmaceutical Inc filed Critical Asklepios Biopharmaceutical Inc
Application granted granted Critical
Publication of DE60139394D1 publication Critical patent/DE60139394D1/de
Anticipated expiration legal-status Critical
Expired - Lifetime 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
DE60139394T 2000-04-28 2001-04-27 Für das dystrophin-minigen kodierende dna-sequenzen und verfahren zu deren verwendung Expired - Lifetime DE60139394D1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US20077700P 2000-04-28 2000-04-28
PCT/US2001/013677 WO2001083695A2 (en) 2000-04-28 2001-04-27 Dna sequences encoding dystrophin minigenes and methods of use thereof

Publications (1)

Publication Number Publication Date
DE60139394D1 true DE60139394D1 (de) 2009-09-10

Family

ID=22743139

Family Applications (1)

Application Number Title Priority Date Filing Date
DE60139394T Expired - Lifetime DE60139394D1 (de) 2000-04-28 2001-04-27 Für das dystrophin-minigen kodierende dna-sequenzen und verfahren zu deren verwendung

Country Status (9)

Country Link
US (2) US7001761B2 (de)
EP (1) EP1287125B1 (de)
CN (1) CN100422320C (de)
AT (1) ATE437944T1 (de)
AU (1) AU2001261063A1 (de)
CA (1) CA2407309C (de)
DE (1) DE60139394D1 (de)
ES (1) ES2330615T3 (de)
WO (1) WO2001083695A2 (de)

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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
US7771993B2 (en) * 2004-01-23 2010-08-10 The Trustees Of The University Of Pennsylvania Microutrophin and uses thereof
PL1766010T3 (pl) 2004-06-28 2011-07-29 Univ Western Australia Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania
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
US7863017B2 (en) * 2006-12-01 2011-01-04 Wisconsin Alumni Research Foundation TAT-utrophin as a protein therapy for dystrophinopathies
EP2125006B1 (de) * 2007-01-18 2013-10-16 University of Missouri-Columbia Synthetische mini-/mikro-dystrophingene zur wiederherstellung von nnos beim sarkolemm
US20080221027A1 (en) * 2007-03-09 2008-09-11 The Regents Of The University Of California Composition and Methods for the Treatment of Duchene Muscular Dystrophy
CN101815535B (zh) 2007-07-12 2013-04-24 普罗森那技术公司 用于使化合物靶向多种选定器官或组织的分子
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
WO2009054725A2 (en) 2007-10-26 2009-04-30 Academisch Ziekenhuis Leiden 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
US20090280103A1 (en) * 2008-04-04 2009-11-12 Martin Flueck Regulation of muscle repair
EP2119783A1 (de) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Verfahren für effizientes Exon (44)-Skipping bei Duchenne-Muskeldystrophie und entsprechende Mittel
LT3133160T (lt) 2008-10-24 2019-04-10 Sarepta Therapeutics, Inc. Kompozicijos su egzono praleidimu dmd gydymui
AU2010239779A1 (en) 2009-04-24 2011-11-17 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating DMD
CA2780563A1 (en) 2009-11-12 2011-05-19 The University Of Western Australia Antisense molecules and methods for treating pathologies
US20110197290A1 (en) * 2010-02-11 2011-08-11 Fahrenkrug Scott C Methods and materials for producing transgenic artiodactyls
US10920242B2 (en) 2011-02-25 2021-02-16 Recombinetics, Inc. Non-meiotic allele introgression
EP2806900B1 (de) 2012-01-27 2021-12-15 BioMarin Technologies B.V. Rna-modulierende oligonukleotide mit verbesserten eigenschaften zur behandlung der duchenne- und becker-muskeldystrophie
US20140140969A1 (en) * 2012-11-20 2014-05-22 Sangamo Biosciences, Inc. Methods and compositions for muscular dystrophies
EA035882B1 (ru) 2013-03-14 2020-08-27 Сарепта Терапьютикс, Инк. Антисмысловые олигонуклеотиды, обеспечивающие пропуск экзонов, для лечения мышечной дистрофии
CN105307723A (zh) 2013-03-15 2016-02-03 萨勒普塔医疗公司 改进的用于治疗肌营养不良的组合物
GB201507842D0 (en) * 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
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
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
WO2019204303A2 (en) * 2018-04-16 2019-10-24 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
SG11202105030VA (en) * 2018-11-16 2021-06-29 Astellas Pharma Inc Method for treating muscular dystrophy by targeting utrophin gene
CN114207120A (zh) 2019-05-30 2022-03-18 坚固生物科技公司 重组疱疹病毒载体
US20230241248A1 (en) 2019-06-27 2023-08-03 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
CA3155381A1 (en) 2019-11-06 2021-05-14 Association Institut De Myologie Combined therapy for muscular diseases
BR112022021635A2 (pt) 2020-04-29 2022-12-06 Bristol Myers Squibb Co Distrofinas miniaturizadas tendo domínios de fusão de espectrina e usos das mesmas
JP2023548816A (ja) 2020-11-03 2023-11-21 ファイザー・インク 陰イオン交換クロマトグラフィーによるａａｖベクターの精製方法
IL303959A (en) 2020-12-23 2023-08-01 Pfizer Methods for purification of AAV vectors by affinity chromatography
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
CN114316070B (zh) * 2021-12-29 2022-11-15 上海勉亦生物科技有限公司 用于治疗肌营养不良症的转基因表达盒
EP4215614A1 (de) 2022-01-24 2023-07-26 Dynacure Kombinationstherapie für dystrophin-verwandte krankheiten

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US5239060A (en) * 1986-07-25 1993-08-24 The Children's Medical Center Corporation Muscular dystrophy protein, dystrophin
WO1990002806A1 (en) * 1988-09-01 1990-03-22 Whitehead Institute For Biomedical Research Recombinant retroviruses with amphotropic and ecotropic host ranges
US5399346A (en) * 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
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US5661033A (en) * 1992-11-25 1997-08-26 The Board Of Trustees Of The Leland Stanford Junior University Gene transfer using herpes virus vectors as a tool for neuroprotection
US5985846A (en) * 1995-06-07 1999-11-16 Baylor College Of Medicine Gene therapy for muscular dystrophy
GB9622174D0 (en) * 1995-12-19 1996-12-18 Medical Res Council Gene expression
IL128779A0 (en) * 1996-09-06 2000-01-31 Univ Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
US5994132A (en) * 1996-10-23 1999-11-30 University Of Michigan Adenovirus vectors
US6207455B1 (en) * 1997-05-01 2001-03-27 Lung-Ji Chang Lentiviral vectors
US6410300B1 (en) * 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
JPH11318467A (ja) * 1998-05-08 1999-11-24 Japan Science & Technology Corp 短縮型ジストロフィン
WO2001029243A1 (en) * 1999-10-15 2001-04-26 Dalhousie University Method and vector for producing and transferring trans-spliced peptides
US20020076754A1 (en) * 2000-04-20 2002-06-20 Liangwu Sun Overcoming AAV vector size limitation through viral DNA hetero-dimerization
DE60139394D1 (de) * 2000-04-28 2009-09-10 Asklepios Biopharmaceutical In Für das dystrophin-minigen kodierende dna-sequenzen und verfahren zu deren verwendung
ATE400656T1 (de) * 2000-10-06 2008-07-15 Univ Michigan Mini-dystrophin nukleinsäure- und peptidsequenzen
KR20020071927A (ko) 2000-11-07 2002-09-13 코닌클리케 필립스 일렉트로닉스 엔.브이. 정보 신호에 워터마크를 삽입하기 위한 방법 및 장치

Also Published As

Publication number Publication date
WO2001083695A9 (en) 2002-12-12
WO2001083695A3 (en) 2002-03-07
WO2001083695A2 (en) 2001-11-08
ES2330615T3 (es) 2009-12-14
AU2001261063A1 (en) 2001-11-12
EP1287125A2 (de) 2003-03-05
CA2407309A1 (en) 2001-11-08
EP1287125B1 (de) 2009-07-29
CN100422320C (zh) 2008-10-01
US20060073586A1 (en) 2006-04-06
CN1439051A (zh) 2003-08-27
US7001761B2 (en) 2006-02-21
US20030171312A1 (en) 2003-09-11
EP1287125A4 (de) 2005-04-27
CA2407309C (en) 2011-08-02
ATE437944T1 (de) 2009-08-15
US7510867B2 (en) 2009-03-31

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Legal Events

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Representative=s name: KOCH & SCHIWECK PATENTANWAELTE, 80339 MUENCHEN