ATE437944T1 - 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
ATE437944T1
ATE437944T1 AT01934920T AT01934920T ATE437944T1 AT E437944 T1 ATE437944 T1 AT E437944T1 AT 01934920 T AT01934920 T AT 01934920T AT 01934920 T AT01934920 T AT 01934920T AT E437944 T1 ATE437944 T1 AT E437944T1
Authority
AT
Austria
Prior art keywords
dystrophin
present
provides
minigen
dna sequences
Prior art date
Application number
AT01934920T
Other languages
English (en)
Inventor
Xiao Xiao
Original Assignee
Asklepios Biopharmaceutical In
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 In filed Critical Asklepios Biopharmaceutical In
Application granted granted Critical
Publication of ATE437944T1 publication Critical patent/ATE437944T1/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
    • 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

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)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
AT01934920T 2000-04-28 2001-04-27 Für das dystrophin-minigen kodierende dna- sequenzen und verfahren zu deren verwendung ATE437944T1 (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
ATE437944T1 true ATE437944T1 (de) 2009-08-15

Family

ID=22743139

Family Applications (1)

Application Number Title Priority Date Filing Date
AT01934920T ATE437944T1 (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)

Families Citing this family (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2001261063A1 (en) * 2000-04-28 2001-11-12 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
EP1366160B1 (de) 2000-10-06 2008-07-09 The Regents Of The University Of Michigan Mini-dystrophin nukleinsäure- und peptidsequenzen
EP2017338A1 (de) * 2001-05-24 2009-01-21 Genzyme Corporation Muskelspezifische Expressionsvektoren
JP4777777B2 (ja) 2002-11-25 2011-09-21 雅文 松尾 ｍＲＮＡ前駆体のスプライシングを修飾するＥＮＡ核酸医薬
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
WO2005118611A2 (en) * 2004-01-23 2005-12-15 The Trustees Of The University Of Pennsylvania Microutrophin and uses thereof
EP3228711A1 (de) 2004-06-28 2017-10-11 The University Of Western Australia Antisense-oligonukleotide zur induktion von exon-skipping sowie verfahren zur verwendung 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
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
WO2009008727A2 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs or tissues
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
PT2203173E (pt) 2007-10-26 2016-03-15 Academisch Ziekenhuis Leiden Resumo
CA2714120A1 (en) 2008-02-08 2009-08-13 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
CN105779453A (zh) 2008-10-24 2016-07-20 萨雷普塔治疗公司 用于dmd的多外显子跳跃组合物
CN102459595A (zh) 2009-04-24 2012-05-16 普罗森那技术公司 用于治疗dmd的包含肌苷的寡核苷酸
JP5963678B2 (ja) 2009-11-12 2016-08-03 ジ ユニバーシティ オブ ウェスタン オーストラリア 病状を治療するためのアンチセンス分子及び方法
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
CA3120918A1 (en) 2012-01-27 2013-08-01 Biomarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
US20140140969A1 (en) * 2012-11-20 2014-05-22 Sangamo Biosciences, Inc. Methods and compositions for muscular dystrophies
JP6449231B2 (ja) 2013-03-14 2019-01-09 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィを処置するためのエキソンスキッピング組成物
MX2015013117A (es) 2013-03-15 2016-07-14 Sarepta Therapeutics Inc Composiciones mejoradas para tratar distrofia muscular.
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
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
US20210155956A1 (en) * 2018-04-16 2021-05-27 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
JP2022534119A (ja) 2019-05-30 2022-07-27 ソリッド・バイオサイエンシーズ・インコーポレーテッド 組換えヘルペスウイルスベクター
US20230241248A1 (en) 2019-06-27 2023-08-03 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
WO2021089736A1 (en) 2019-11-06 2021-05-14 Association Institut De Myologie Combined therapy for muscular diseases
WO2021222332A1 (en) 2020-04-29 2021-11-04 Bristol-Myers Squibb Company Miniaturized dystrophins having spectrin fusion domains and uses thereof
AU2021375404A1 (en) 2020-11-03 2023-06-08 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
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
WO2022216749A1 (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

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE3681787D1 (de) * 1985-07-05 1991-11-07 Whitehead Biomedical Inst Expression von fremdem genetischem material in epithelzellen.
US5239060A (en) * 1986-07-25 1993-08-24 The Children's Medical Center Corporation Muscular dystrophy protein, dystrophin
EP0432216A1 (de) * 1988-09-01 1991-06-19 Whitehead Institute For Biomedical Research Rekombinante retroviren mit amphotropen und ecotropen wirtsspektren
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
US5173414A (en) * 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
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
AU723497C (en) * 1996-09-06 2001-10-11 Trustees Of The University Of Pennsylvania, The 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
AU2001261063A1 (en) * 2000-04-28 2001-11-12 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
EP1366160B1 (de) * 2000-10-06 2008-07-09 The Regents Of The University Of Michigan Mini-dystrophin nukleinsäure- und peptidsequenzen
JP2004513586A (ja) 2000-11-07 2004-04-30 コーニンクレッカ　フィリップス　エレクトロニクス　エヌ　ヴィ 透かしを情報信号に埋め込む方法及び装置

Also Published As

Publication number Publication date
EP1287125A2 (de) 2003-03-05
EP1287125A4 (de) 2005-04-27
US20060073586A1 (en) 2006-04-06
WO2001083695A2 (en) 2001-11-08
US7001761B2 (en) 2006-02-21
CA2407309A1 (en) 2001-11-08
ES2330615T3 (es) 2009-12-14
AU2001261063A1 (en) 2001-11-12
WO2001083695A9 (en) 2002-12-12
DE60139394D1 (de) 2009-09-10
WO2001083695A3 (en) 2002-03-07
CN1439051A (zh) 2003-08-27
EP1287125B1 (de) 2009-07-29
CN100422320C (zh) 2008-10-01
US20030171312A1 (en) 2003-09-11
US7510867B2 (en) 2009-03-31
CA2407309C (en) 2011-08-02

Similar Documents

Publication Publication Date Title
ATE437944T1 (de) Für das dystrophin-minigen kodierende dna- sequenzen und verfahren zu deren verwendung
FR21C1037I1 (fr) Clades de virus adéno associés (aav), séquences, vecteurs les contenant et leurs utilisations
Yount et al. Reverse genetics with a full-length infectious cDNA of severe acute respiratory syndrome coronavirus
IL234063A (en) Adenovirus containing capsid aavrh.34 and a heterologous gene, its preparation and preparations containing it
IS7173A (is) Líffræðilegar afurðir
NO990025D0 (no) Hamster-EF-1<alfa> transkripsjonsregulatorisk DNA
ATE362542T1 (de) Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
ATE442152T1 (de) Antisense-entwurf
NO965080L (no) Antisens oligonukleotidmodulering av genekspresjon
ATE484596T1 (de) Mutantes herpes-simplex-virus und verwendung davon bei der behandlung von plattenepithelkarzinom
ATE238431T1 (de) Methode der veränderung der expression von genen bei der transkription und zum nachweis von chemischen substanzen, die wie modulatoren der genexpression wirken
CY1106369T1 (el) Μεθοδος ταχειας τυποποιησης μικροοργανισμων με ηλεκτροφορηση πηκτης παλλομενου πεδιου με την οποια τα δειγματα παρασκευαζονται σε ενα εγκλειομενο σε αυτοκλειστο και ευκαμπτο καλουπι
Doughty et al. Post-transplant quasispecies pattern remains stable over time in patients with recurrent cholestatic hepatitis due to hepatitis C virus
DE60028544D1 (de) Prostata-spezifische gene zur diagnose, prognose und behandlung von prostata-krebs
Chahal et al. miR-122–based therapies select for three distinct resistance mechanisms based on alterations in RNA structure
Lecomte et al. The SSV‐Seq 2.0 PCR‐Free method improves the sequencing of adeno‐associated viral vector genomes containing GC‐Rich regions and homopolymers
ATE427959T1 (de) Gereinigtes sr-p70 protein
ATE267402T1 (de) Verfahren zur testung des hormonellen effekts von substanzen
DK0643719T3 (da) Nukleinsyresekvenser, som koder for eller er komplementære til nukleinsyresekvenser, som koder for interleukin 9-receptor
DE60213578D1 (de) Replikationsprozess des hepatitis c virus
ATE332712T1 (de) Nukleinsaüre- und aminosaürensequenzen von infectious salmon anaemia virus, und deren verwendung als impfstoffe
Cho et al. Validation of trans-acting elements that promote exon 7 skipping of SMN2 in SMN2-GFP stable cell line
DK0868511T3 (da) Genekspression af utrophin
DE69809930T2 (de) Isolierung und verwendung von nukleinsäuremolekülen, welche für mitglieder der ssx-familie kodieren
WO2005033280A8 (en) Hiv-dependent expression constructs and uses therefor

Legal Events

Date Code Title Description
RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties