CL2014003464A1 - Método para mejorar la integridad deteriorada de la unión neuromuscular que comprende la administración de una composición que comprende un vector viral adeno-asociado recombinante (raav) que comprende una molécula de ácido nucleico que codifica la alfa-glucosidasa ácida (gaa). - Google Patents

Método para mejorar la integridad deteriorada de la unión neuromuscular que comprende la administración de una composición que comprende un vector viral adeno-asociado recombinante (raav) que comprende una molécula de ácido nucleico que codifica la alfa-glucosidasa ácida (gaa).

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Publication number
CL2014003464A1
CL2014003464A1 CL2014003464A CL2014003464A CL2014003464A1 CL 2014003464 A1 CL2014003464 A1 CL 2014003464A1 CL 2014003464 A CL2014003464 A CL 2014003464A CL 2014003464 A CL2014003464 A CL 2014003464A CL 2014003464 A1 CL2014003464 A1 CL 2014003464A1
Authority
CL
Chile
Prior art keywords
raav
gaa
glucosidase
administration
improving
Prior art date
Application number
CL2014003464A
Other languages
English (en)
Inventor
Barry John Byrne
Darin J Falk
Christina Pacak
Lara Robert Deruisseau
Cathryn Mah
David D Fuller
Original Assignee
Univ Florida
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
Priority claimed from US13/527,350 external-priority patent/US20120322861A1/en
Application filed by Univ Florida filed Critical Univ Florida
Publication of CL2014003464A1 publication Critical patent/CL2014003464A1/es

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CL2014003464A 2012-06-19 2014-12-19 Método para mejorar la integridad deteriorada de la unión neuromuscular que comprende la administración de una composición que comprende un vector viral adeno-asociado recombinante (raav) que comprende una molécula de ácido nucleico que codifica la alfa-glucosidasa ácida (gaa). CL2014003464A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US13/527,350 US20120322861A1 (en) 2007-02-23 2012-06-19 Compositions and Methods for Treating Diseases

Publications (1)

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CL2014003464A1 true CL2014003464A1 (es) 2015-06-12

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CL2014003464A CL2014003464A1 (es) 2012-06-19 2014-12-19 Método para mejorar la integridad deteriorada de la unión neuromuscular que comprende la administración de una composición que comprende un vector viral adeno-asociado recombinante (raav) que comprende una molécula de ácido nucleico que codifica la alfa-glucosidasa ácida (gaa).

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US (2) US10912804B2 (es)
EP (4) EP4124346A3 (es)
JP (1) JP2015523998A (es)
KR (1) KR20150020250A (es)
CN (1) CN104619354A (es)
AU (1) AU2013277215A1 (es)
BR (1) BR112014031788A2 (es)
CA (1) CA2875523A1 (es)
CL (1) CL2014003464A1 (es)
CY (1) CY1123485T1 (es)
DK (1) DK3292875T3 (es)
ES (1) ES2813558T3 (es)
HK (1) HK1209633A1 (es)
HR (1) HRP20201224T1 (es)
HU (1) HUE051632T2 (es)
IL (1) IL236298A0 (es)
LT (1) LT3292875T (es)
MX (1) MX2014015985A (es)
PL (1) PL3292875T3 (es)
PT (1) PT3292875T (es)
RS (1) RS61055B1 (es)
RU (1) RU2015101276A (es)
SI (1) SI3292875T1 (es)
SM (1) SMT202000445T1 (es)
WO (1) WO2013192317A2 (es)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
EP4124346A3 (en) 2012-06-19 2023-04-19 University of Florida Research Foundation, Inc. Compositions and methods for treating diseases
JP2016523835A (ja) * 2013-05-15 2016-08-12 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入
US11866727B2 (en) * 2015-11-06 2024-01-09 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1A
ES2871527T3 (es) 2016-01-15 2021-10-29 Univ Jichi Medical Virión de virus adenoasociado para usar en el tratamiento de la epilepsia
EP3293259A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
KR102617502B1 (ko) * 2016-09-12 2023-12-26 제네똥 산-알파 글루코시다제 변이체 및 이의 용도
AU2018281280B2 (en) 2017-06-07 2024-12-19 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
EP3684938A1 (en) 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
JP6567012B2 (ja) * 2017-09-28 2019-08-28 キヤノン株式会社 情報処理装置、情報処理方法、およびプログラム
WO2019146745A1 (ja) * 2018-01-26 2019-08-01 国立大学法人徳島大学 テイ-サックス病及びザンドホッフ病治療用の新規アデノ随伴ウイルスビリオン
CN111936621B (zh) * 2018-02-05 2025-05-13 奥登茨治疗公司 转录调控元件和它的用途
CA3086942A1 (en) * 2018-02-07 2019-08-15 Genethon Hybrid regulatory elements
CN112040985A (zh) 2018-02-07 2020-12-04 瑞泽恩制药公司 用于递送治疗性蛋白质的方法和组合物
EP3793591A1 (en) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
BR112021001878A2 (pt) * 2018-07-31 2021-04-27 Cornell University métodos de terapia genética para o controle da função do órgão
EP3996696A1 (en) * 2019-07-09 2022-05-18 Genethon Treatment of glycogen storage disease (gsd)
AU2020372429A1 (en) * 2019-10-25 2022-04-28 Audentes Therapeutics, Inc. Compositions and methods for treating glycogen storage disorders

Family Cites Families (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US6451600B1 (en) 1989-12-22 2002-09-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
WO1995011640A1 (en) 1993-10-26 1995-05-04 Howmedica Inc. Prosthesis with integral proximal spacer
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US6083725A (en) 1996-09-13 2000-07-04 Transkaryotic Therapies, Inc. Tranfected human cells expressing human α-galactosidase A protein
US6426208B1 (en) 1999-11-12 2002-07-30 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
EP1390490B1 (en) * 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
EP1463805B1 (en) 2001-12-17 2014-10-22 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
WO2003074711A1 (en) 2002-03-04 2003-09-12 Transgene S.A. Expression cassette for persistence of expression of a gene of interest in muscle cells
US20030223966A1 (en) 2002-04-30 2003-12-04 Fraites Thomas J. Treatment for pompe disease
US7858367B2 (en) 2002-04-30 2010-12-28 Duke University Viral vectors and methods for producing and using the same
AU2003302724A1 (en) * 2002-08-13 2004-07-09 Mcivor, Scott, R. Methods of using vectors to treat metabolic disorders
PT1587923E (pt) 2003-01-22 2011-12-07 Univ Duke Construções melhoradas para a expressão de polipéptidos lisossomais
JP2007514403A (ja) * 2003-08-21 2007-06-07 キュー セラピューティクス インコーポレイティッド リソソーム蓄積症において欠損しているリソソームプロ酵素の持続的分泌のための遺伝子改変体細胞
WO2005049850A2 (en) * 2003-11-14 2005-06-02 University Of Washington Compositions and methods for systemic nucleic acid sequence delivery
JP2007513968A (ja) 2003-12-12 2007-05-31 ウイスコンシン アラムニ リサーチ ファンデーション 哺乳動物神経細胞へのsiRNAのデリバリー
US7771993B2 (en) * 2004-01-23 2010-08-10 The Trustees Of The University Of Pennsylvania Microutrophin and uses thereof
WO2005077333A2 (en) 2004-02-10 2005-08-25 University Of Florida Research Foundation, Inc. Gel-based delivery of recombinant adeno-associated virus vectors
DE602005020745D1 (de) * 2004-02-10 2010-06-02 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
CA2583473A1 (en) * 2004-10-05 2007-04-04 University Of Georgia Research Foundation, Inc. Neuronal progenitors from feeder-free human embryonic stem cell culture
US8058291B2 (en) 2005-04-06 2011-11-15 Adamas Pharmaceuticals, Inc. Methods and compositions for the treatment of CNS-related conditions
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
EP1951751B1 (en) 2005-11-14 2017-11-01 Merial, Inc. Gene therapy for renal failure
US7867484B2 (en) * 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
CN101404927A (zh) 2006-02-09 2009-04-08 建新公司 慢速脑室内递送
US20100305068A1 (en) * 2006-11-09 2010-12-02 Foldrx Pharmaceuticals, Inc. Compounds and methods for modulating protein trafficking
CN101636200A (zh) * 2006-11-13 2010-01-27 齐斯特治疗公司 用于治疗庞贝氏症的方法
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
WO2008103993A2 (en) * 2007-02-23 2008-08-28 University Of Florida Research Foundation, Inc. Compositions and methods for treating glycogen storage diseases
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
SG176064A1 (en) * 2009-05-14 2011-12-29 Oatmeal Biotechnologies Group L L C Platform technologies for spontaneously occurring diseases
SG176628A1 (en) * 2009-06-05 2012-01-30 Link Medicine Corp Aminopyrrolidinone derivatives and uses thereof
US20130166313A1 (en) 2010-04-02 2013-06-27 Shire Human Genetic Therapies, Inc. Systems and methods for managing treatment of an orphan disease
HUE041571T2 (hu) 2010-04-23 2019-05-28 Univ Florida RAAV-guanilát-cikláz-készítmények és ejárások 1-es típusú Leber-féle veleszületett vakság (LCA1) kezelésére
US8679478B2 (en) * 2010-10-04 2014-03-25 Duke University Methods of lysosomal storage disease therapy
US20120141420A1 (en) * 2010-11-30 2012-06-07 Sygnis Bioscience Gmbh & Co. Kg Targeted delivery of g-csf for the treatment of amyotrophic lateral sclerosis
EP2852388A4 (en) 2012-05-23 2016-01-13 Univ Johns Hopkins COMPOUNDS AND METHOD FOR USE THEREOF FOR THE TREATMENT OF NEURODEEGENERATIVE DISEASES
EP4124346A3 (en) 2012-06-19 2023-04-19 University of Florida Research Foundation, Inc. Compositions and methods for treating diseases

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