[go: up one dir, main page]

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)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (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 (45)

* 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
US6869777B2 (en) 2000-10-06 2005-03-22 Regents Of The University Of Michigan Mini-dystrophin nucleic acid sequences
EP2017338A1 (de) * 2001-05-24 2009-01-21 Genzyme Corporation Muskelspezifische Expressionsvektoren
CA3001404C (en) 2002-11-25 2020-07-28 Masafumi Matsuo Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors
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
EP3808845A1 (de) 2004-06-28 2021-04-21 The University Of Western Australia Antisense-oligonukleotide zur induzierung von exon-skipping und 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
US20100184833A1 (en) 2006-08-11 2010-07-22 Prosenta 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
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
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
EP2505211B1 (de) 2007-07-12 2020-04-08 BioMarin Technologies B.V. Moleküle zur Targeting von Verbindungen auf verschiedene ausgewählte Organe oder Gewebe
EP2614827B1 (de) 2007-10-26 2017-06-28 Academisch Ziekenhuis Leiden Mittel und Verfahren zur Bekämpfung von Muskelleiden
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
CN101980726A (zh) 2008-02-08 2011-02-23 普罗森那控股公司 治疗与dna重复不稳定性相关的遗传病症的方法和装置
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
CN102203253B (zh) 2008-10-24 2016-04-06 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
CA2780563A1 (en) 2009-11-12 2011-05-19 The University Of Western Australia Antisense molecules and methods for treating pathologies
BR112012020257A8 (pt) 2010-02-11 2018-02-14 Recombinetics Inc métodos e aparelhos para produzir artiodátilos transgênicos
US10920242B2 (en) 2011-02-25 2021-02-16 Recombinetics, Inc. Non-meiotic allele introgression
JP2015509922A (ja) 2012-01-27 2015-04-02 プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド
US20140140969A1 (en) * 2012-11-20 2014-05-22 Sangamo Biosciences, Inc. Methods and compositions for muscular dystrophies
KR102258326B1 (ko) 2013-03-14 2021-06-02 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
EA201591792A1 (ru) 2013-03-15 2016-02-29 Сарепта Терапьютикс, Инк. Улучшенные композиции для лечения мышечной дистрофии
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
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
BR112020021179A2 (pt) * 2018-04-16 2021-03-02 The Trustees Of The University Of Pennsylvania composições e métodos para tratar distrofia muscular de duchenne
BR112021009481A2 (pt) * 2018-11-16 2021-08-17 Astellas Pharma Inc. método para tratamento da distrofia muscular direcionando gene de utrofina.
EP3976806A1 (de) 2019-05-30 2022-04-06 Solidus Biosciences, Inc. Rekombinanter herpesvirusvektor
EP3990030A1 (de) 2019-06-27 2022-05-04 Pfizer Inc. Verfahren zur behandlung von duchenne-muskeldystrophie mittels aav-mini-dystrophin-gentherapie
JP2023500884A (ja) 2019-11-06 2023-01-11 アソシエーション・アンスティトゥート・ドゥ・マイオロジー 筋疾患のための併用療法
CN115485291A (zh) 2020-04-29 2022-12-16 百时美施贵宝公司 具血影蛋白融合结构域的微型化抗肌萎缩蛋白及其用途
CA3200401A1 (en) 2020-11-03 2022-05-12 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
AU2021404944A1 (en) 2020-12-23 2023-07-06 Pfizer Inc. 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
JP2703893B2 (ja) * 1985-07-05 1998-01-26 ホワイトヘッド・インスティテュ−ト・フォ−・バイオメディカル・リサ−チ 外来遺伝子物質を発現する上皮細胞
US5239060A (en) * 1986-07-25 1993-08-24 The Children's Medical Center Corporation Muscular dystrophy protein, dystrophin
JP3082204B2 (ja) * 1988-09-01 2000-08-28 ホワイトヘッド・インスティチュート・フォー・バイオメディカル・リサーチ 両栄養性および環境栄養性宿主域を持つ組換え体レトロウイルス
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
EP0932418B1 (de) * 1996-09-06 2007-12-05 The Trustees Of The University Of Pennsylvania Verfaheren zur durch rekombinante adeno-assoziierte virus-gerichtete gentherapie
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
US6544786B1 (en) * 1999-10-15 2003-04-08 University Of Pittsburgh Of The Commonwealth Of Higher Education 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
ATE437944T1 (de) * 2000-04-28 2009-08-15 Asklepios Biopharmaceutical In Für das dystrophin-minigen kodierende dna- sequenzen und verfahren zu deren verwendung
US6869777B2 (en) * 2000-10-06 2005-03-22 Regents Of The University Of Michigan Mini-dystrophin nucleic acid sequences
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
US20060073586A1 (en) 2006-04-06
US20030171312A1 (en) 2003-09-11
EP1287125B1 (de) 2009-07-29
US7510867B2 (en) 2009-03-31
WO2001083695A3 (en) 2002-03-07
WO2001083695A9 (en) 2002-12-12
AU2001261063A1 (en) 2001-11-12
CA2407309C (en) 2011-08-02
CN100422320C (zh) 2008-10-01
CA2407309A1 (en) 2001-11-08
CN1439051A (zh) 2003-08-27
US7001761B2 (en) 2006-02-21
EP1287125A2 (de) 2003-03-05
ES2330615T3 (es) 2009-12-14
EP1287125A4 (de) 2005-04-27
DE60139394D1 (de) 2009-09-10
WO2001083695A2 (en) 2001-11-08

Similar Documents

Publication Publication Date Title
ATE437944T1 (de) Für das dystrophin-minigen kodierende dna- sequenzen und verfahren zu deren verwendung
Thorner et al. Age dependence of adenovirus-specific neutralizing antibody titers in individuals from sub-Saharan Africa
CY1109746T1 (el) Χιμαιρικα μορια snrna που φερουν αντιπληροφοριακες αλληλουχιες εναντι των συνδεσμων συρραφης του γονιδιου δυστροφινης και θεραπευτικες εφαρμογες αυτων
NO990025L (no) Hamster-EF-1<alfa> transkripsjonsregulatorisk DNA
IL234063A (en) Adenovirus containing capsid aavrh.34 and a heterologous gene, its preparation and preparations containing it
DE69936104D1 (de) Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
DE60117550D1 (de) Doppelsträngige parvovirus-vektoren
GB0124317D0 (en) Biological products
Dufour et al. Structure and functional relevance of a transcription-regulating sequence involved in coronavirus discontinuous RNA synthesis
DK1569661T3 (da) Antisense design
FI964792A0 (fi) raf-geenin ilmentymisen muuttaminen antisense-oligonukleotideillä
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
ATE404695T1 (de) Einfache katalytische dna-biosensoren für ionen auf grundlage von farbveränderungen
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
DE60028544D1 (de) Prostata-spezifische gene zur diagnose, prognose und behandlung von prostata-krebs
DE60331758D1 (de) Neue vollständige genomische rna des japanenzephalitis-virus, infektiöse jev cdna daraus und deren verwendung
DE69739346D1 (de) Gereinigtes sr-p70 protein
DK0643719T3 (da) Nukleinsyresekvenser, som koder for eller er komplementære til nukleinsyresekvenser, som koder for interleukin 9-receptor
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
DE60130465D1 (de) Mutierte muskelspezifische enhancer
WO2005033280A3 (en) Hiv-dependent expression constructs and uses therefor
MD2149G2 (ro) Primer oligonucleotidic pentru depistarea ARN-ului virusului hepatitei C
ATE430806T1 (de) (-)-strang rns virusvektor für nervenzellen

Legal Events

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