JP2019533439A5 - - Google Patents

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JP2019533439A5
JP2019533439A5 JP2019515997A JP2019515997A JP2019533439A5 JP 2019533439 A5 JP2019533439 A5 JP 2019533439A5 JP 2019515997 A JP2019515997 A JP 2019515997A JP 2019515997 A JP2019515997 A JP 2019515997A JP 2019533439 A5 JP2019533439 A5 JP 2019533439A5
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nucleic acid
sequence
transgene
isolated nucleic
itr
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JP2019515997A
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JP2019533439A (ja
JP6884430B2 (ja
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Priority claimed from PCT/US2017/052902 external-priority patent/WO2018057855A1/en
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Priority to JP2021077175A priority Critical patent/JP7205933B2/ja
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Publication of JP6884430B2 publication Critical patent/JP6884430B2/ja
Priority to JP2022205188A priority patent/JP7613759B2/ja
Priority to JP2024224502A priority patent/JP2025063053A/ja
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JP2019515997A 2016-09-22 2017-09-22 ハンチントン病のaav処置 Active JP6884430B2 (ja)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2021077175A JP7205933B2 (ja) 2016-09-22 2021-04-30 ハンチントン病のaav処置
JP2022205188A JP7613759B2 (ja) 2016-09-22 2022-12-22 ハンチントン病のaav処置
JP2024224502A JP2025063053A (ja) 2016-09-22 2024-12-19 ハンチントン病のaav処置

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662398487P 2016-09-22 2016-09-22
US62/398,487 2016-09-22
PCT/US2017/052902 WO2018057855A1 (en) 2016-09-22 2017-09-22 Aav treatment of huntington's disease

Related Child Applications (1)

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JP2021077175A Division JP7205933B2 (ja) 2016-09-22 2021-04-30 ハンチントン病のaav処置

Publications (3)

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JP2019533439A JP2019533439A (ja) 2019-11-21
JP2019533439A5 true JP2019533439A5 (enExample) 2020-12-03
JP6884430B2 JP6884430B2 (ja) 2021-06-09

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JP2019515997A Active JP6884430B2 (ja) 2016-09-22 2017-09-22 ハンチントン病のaav処置
JP2021077175A Active JP7205933B2 (ja) 2016-09-22 2021-04-30 ハンチントン病のaav処置
JP2022205188A Active JP7613759B2 (ja) 2016-09-22 2022-12-22 ハンチントン病のaav処置
JP2024224502A Pending JP2025063053A (ja) 2016-09-22 2024-12-19 ハンチントン病のaav処置

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JP2021077175A Active JP7205933B2 (ja) 2016-09-22 2021-04-30 ハンチントン病のaav処置
JP2022205188A Active JP7613759B2 (ja) 2016-09-22 2022-12-22 ハンチントン病のaav処置
JP2024224502A Pending JP2025063053A (ja) 2016-09-22 2024-12-19 ハンチントン病のaav処置

Country Status (20)

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US (3) US10457940B2 (enExample)
EP (1) EP3515505A4 (enExample)
JP (4) JP6884430B2 (enExample)
KR (1) KR102271675B1 (enExample)
CN (2) CN117025677A (enExample)
AU (2) AU2017330411B2 (enExample)
BR (2) BR122020019033B1 (enExample)
CA (1) CA3037929A1 (enExample)
CL (1) CL2019000732A1 (enExample)
CO (1) CO2019003846A2 (enExample)
IL (3) IL308514B1 (enExample)
MX (2) MX2019003376A (enExample)
MY (1) MY195386A (enExample)
NZ (1) NZ751950A (enExample)
PE (2) PE20241233A1 (enExample)
PH (1) PH12019500626A1 (enExample)
RU (1) RU2749971C2 (enExample)
SA (1) SA519401379B1 (enExample)
WO (1) WO2018057855A1 (enExample)
ZA (1) ZA201902226B (enExample)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10975391B2 (en) 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
RU2764587C2 (ru) 2016-05-18 2022-01-18 Вояджер Терапьютикс, Инк. Способы и композиции для лечения хореи гентингтона
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
SG11201909868YA (en) * 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
CN111742060A (zh) * 2018-02-13 2020-10-02 东丽株式会社 用于痴呆的检测的试剂盒或器件和方法
CA3133899A1 (en) * 2018-03-30 2019-10-03 University Of Geneva Micro rna expression constructs and uses thereof
WO2019213128A1 (en) 2018-04-30 2019-11-07 The Brigham And Women's Hospital, Inc. Compositions and therapeutics methods of microrna gene delivery
WO2019222413A1 (en) 2018-05-15 2019-11-21 University Of Massachusetts Modified aav constructs and uses thereof
EP3887522A1 (en) 2018-11-29 2021-10-06 University of Massachusetts Modulation of sptlc1 via recombinant adeno-associated vectors
AU2020206617A1 (en) * 2019-01-09 2021-08-26 Centro De Neurociencias E Biologia Celular Double stranded RNA and uses thereof
AR120341A1 (es) * 2019-11-01 2022-02-09 Alnylam Pharmaceuticals Inc COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
WO2021127455A1 (en) * 2019-12-20 2021-06-24 Asklepios Biopharmaceutical, Inc. Methods for treating huntington's disease
CA3193406A1 (en) * 2020-09-21 2022-03-24 Asklepios Biopharmaceutical, Inc. Methods for treating neurological disease
US20240084323A1 (en) * 2021-01-13 2024-03-14 Dignity Health Modulation of chitinase protein expression
WO2022206819A1 (zh) * 2021-03-30 2022-10-06 南京大学 一种用于治疗亨廷顿病的rna递送系统
CN115487315B (zh) * 2022-04-20 2023-08-04 暨南大学 治疗亨廷顿病的药物
US20240360443A1 (en) 2023-04-09 2024-10-31 Dystrogen Gene Therapies Inc. Nucleic acid molecule and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide cag repeats
WO2025027116A1 (en) 2023-08-01 2025-02-06 Institut Curie Nanoparticles comprising nucleic acid sequences encoding cyclic gmp-amp synthase
WO2025078598A1 (en) * 2023-10-11 2025-04-17 Uniqure Biopharma B.V. Novel nucleic acid regulation of huntingtin gene
WO2025160434A1 (en) * 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting huntington's disease

Family Cites Families (142)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
US5252334A (en) 1989-09-08 1993-10-12 Cygnus Therapeutic Systems Solid matrix system for transdermal drug delivery
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5697899A (en) 1995-02-07 1997-12-16 Gensia Feedback controlled drug delivery system
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5656016A (en) 1996-03-18 1997-08-12 Abbott Laboratories Sonophoretic drug delivery system
US5797898A (en) 1996-07-02 1998-08-25 Massachusetts Institute Of Technology Microchip drug delivery devices
US5783208A (en) 1996-07-19 1998-07-21 Theratech, Inc. Transdermal drug delivery matrix for coadministering estradiol and another steroid
US5779708A (en) 1996-08-15 1998-07-14 Cyberdent, Inc. Intraosseous drug delivery device and method
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6177403B1 (en) 1996-10-21 2001-01-23 The Trustees Of The University Of Pennsylvania Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal
US6544523B1 (en) 1996-11-13 2003-04-08 Chiron Corporation Mutant forms of Fas ligand and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
US20030110526A1 (en) 1998-08-25 2003-06-12 Robert H. Brown Dysferlin mutations
JP4693244B2 (ja) 1999-03-18 2011-06-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
WO2001029243A1 (en) 1999-10-15 2001-04-26 Dalhousie University Method and vector for producing and transferring trans-spliced peptides
US7638120B2 (en) 2000-03-14 2009-12-29 Thomas Jefferson University High transgene expression of a pseudotyped adeno-associated virus type
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
CA2452980A1 (en) 2001-07-03 2003-01-16 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods of administering vectors to synaptically connected neurons
CA2921821A1 (en) * 2001-07-12 2003-01-23 University Of Massachusetts In vivo production of small interfering rnas that mediate gene silencing
WO2003006616A2 (en) 2001-07-13 2003-01-23 University Of Iowa Research Foundation Pseudotyped adeno-associated viruses and uses thereof
AU2002326589B2 (en) * 2001-08-07 2008-06-05 University Of Delaware Compositions and methods for the prevention and treatment of Huntington's disease
BR122016004546B8 (pt) 2001-11-13 2021-07-27 Univ Pennsylvania vírus adenoassociado recombinante, método de geração do referido vírus e composição compreendendo o referido vírus
EP3339430A1 (en) 2001-12-17 2018-06-27 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses thereof
US20050137153A1 (en) 2002-02-20 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) gene expression using short interfering nucleic acid (siNA)
AU2003221733A1 (en) 2002-04-17 2003-11-03 University Of Florida Research Foundation, Inc. Improved raav vectors
AU2003221745A1 (en) 2002-04-19 2003-11-03 University Of Florida rAAV VECTOR-BASED COMPOSITIONS AND METHODS FOR THE PREVENTION AND TREATMENT OF MAMMALIAN DISEASES
CA2426283C (en) 2002-04-29 2006-06-27 The Trustees Of The University Of Pennsylvania Method for direct rescue and amplification of integrated viruses from cellular dna of tissues
US20060093589A1 (en) 2004-02-19 2006-05-04 Warrington Kenneth H Vp2-modified raav vector compositions and uses therefor
US20050255086A1 (en) 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US20050106731A1 (en) * 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
JP4966006B2 (ja) 2003-01-28 2012-07-04 セレクティス カスタムメイドメガヌクレアーゼおよびその使用
US20040219528A1 (en) 2003-04-15 2004-11-04 Morris David W. Novel therapeutic targets in cancer
US7387896B2 (en) 2003-03-26 2008-06-17 The Regents Of The University Of Michigan MicroRNA vectors
US20060228800A1 (en) 2003-05-15 2006-10-12 Shi-Lung Lin Novel Transgenic Methods Using intronic RNA
US7459547B2 (en) 2003-06-02 2008-12-02 University Of Massachusetts Methods and compositions for controlling efficacy of RNA silencing
US8680063B2 (en) * 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
CA3072423A1 (en) * 2003-09-30 2005-04-14 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
CN103060324B (zh) 2004-03-05 2015-04-01 贝尼泰克生物制药有限公司 用于RNAi试剂同时递送的多启动子表达盒
WO2005096781A2 (en) 2004-04-06 2005-10-20 University Of Massachusetts Methods and compositions for treating gain-of-function disorders using rna interference
WO2005116224A2 (en) 2004-05-18 2005-12-08 Children's Memorial Hospital Tetracycline-regulated adeno-associated viral (aav) vectors for gene delivery to the nervous system
US7427396B2 (en) 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
NZ555830A (en) 2004-12-15 2009-01-31 Univ North Carolina Chimeric vectors
EP1877556B1 (en) 2005-03-25 2011-09-14 Medtronic, Inc. Use of anti-tnf or anti-il1 rnai to suppress pro- inflammatory cytokine actions locally to treat pain
EP3409296A1 (en) 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
US10000757B2 (en) 2005-05-27 2018-06-19 Ospedale San Raffaele S.R.L. Gene vector
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
ATE494372T1 (de) 2005-08-29 2011-01-15 Regulus Therapeutics Inc Verfahren für mir-122a-modulation
EP2487252B1 (en) 2006-01-05 2014-10-15 The Ohio State University Research Foundation MicroRNA-based methods for the diagnosis of colon cancer
EP1979485A2 (en) 2006-01-31 2008-10-15 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
CA2642848C (en) 2006-03-03 2016-07-12 Amorfix Life Sciences Ltd. Methods and compositions to treat and detect misfolded-sod1 mediated diseases
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
EP2046993A4 (en) * 2006-07-07 2010-11-17 Univ Massachusetts RNA SILENCING COMPOSITIONS, AND METHODS OF TREATING HUNTINGTON CHOREA
US20080199961A1 (en) * 2006-08-25 2008-08-21 Avi Biopharma, Inc. ANTISENSE COMPOSITION AND METHOD FOR INHIBITION OF miRNA BIOGENESIS
EP2799547B1 (en) 2006-11-08 2016-12-21 Veritas Bio, LLC In Vivo Delivery of RNA to a Target Cell
HRP20161150T1 (hr) 2007-04-09 2016-11-18 University Of Florida Research Foundation, Inc. PRIPRAVCI rAAV VEKTORA KOJI IMAJU TIROZIN-MODIFICIRANE CAPSID PROTEINE I POSTUPCI ZA UPORABU
CA2683469C (en) 2007-04-12 2019-11-26 The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Genetic suppression and replacement
US8399248B2 (en) 2007-05-03 2013-03-19 Merck Sharp & Dohme Corp. Methods of using MIR34 as a biomarker for TP53 functional status
WO2008147837A1 (en) 2007-05-23 2008-12-04 Dharmacon, Inc. Micro-rna scaffolds, non-naturally occurring micro-rnas, and methods for optimizing non-naturally occurring micro-rnas
AU2008260103B2 (en) 2007-05-31 2014-04-03 University Of Iowa Research Foundation Reduction of off-target RNA interference toxicity
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
WO2009079592A2 (en) 2007-12-17 2009-06-25 California Institute Of Technology Modulating immune system development and function through microrna mir-146
US20090215879A1 (en) 2008-02-26 2009-08-27 University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
EP2268811A1 (en) 2008-03-07 2011-01-05 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
WO2009146178A1 (en) 2008-04-15 2009-12-03 President And Fellows Of Harvard College Angiogenin and amyotrophic lateral sclerosis
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
EP2297322A1 (en) 2008-06-04 2011-03-23 The Board of Regents of The University of Texas System Modulation of gene expression through endogenous small rna targeting of gene promoters
WO2009154452A1 (en) 2008-06-17 2009-12-23 Amsterdam Molecular Therapeutics B.V. Parvoviral capsid with incorporated Gly-Ala repeat region
EP2310507A4 (en) 2008-07-08 2013-03-20 David Gladstone Inst METHOD AND COMPOSITIONS FOR ANGIOGENIC MODULATION
WO2010027446A2 (en) 2008-09-04 2010-03-11 Galenea Corp., Et Al. Synaptic vesicle cycling assays and systems
WO2010034314A1 (en) 2008-09-24 2010-04-01 Aarhus Universitet Retroviral delivery of synthectic gene cassettes
WO2010071454A1 (en) 2008-12-17 2010-06-24 Auckland Uniservices Limited Adeno-associated viral vectors and uses thereof
WO2010099383A2 (en) 2009-02-26 2010-09-02 The Johns Hopkins University Compositions and methods for ex vivo hepatic nucleic acid delivery
EP3733214A1 (en) * 2009-04-30 2020-11-04 Ospedale San Raffaele S.r.l. Gene vector
LT3421603T (lt) 2009-05-02 2022-01-10 Genzyme Corporation Genų terapija, skirta neurodegeneraciniams sutrikimams
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
EP2261242A1 (en) 2009-06-10 2010-12-15 Universite Catholique De Louvain Aspartate-N-acetyltransferase enzyme, diagnostic method and therapeutic method
EP2475675B1 (en) * 2009-09-11 2016-11-16 Ionis Pharmaceuticals, Inc. Modulation of huntingtin expression
US20120270930A1 (en) 2009-10-29 2012-10-25 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Methods and compositions for dysferlin exon-skipping
RU2606012C2 (ru) 2009-11-09 2017-01-10 Дженепод Терапевтикс Аб Новая вирусная векторная конструкция для нейронспецифического оптимизированного непрерывного синтеза dopa in vivo
CA2781332C (en) 2009-11-19 2018-09-04 National University Corporation Okayama University System for increasing gene expression and vector comprising the system
EP2524037B1 (en) 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
PT2529020T (pt) 2010-01-28 2018-07-30 Childrens Hospital Philadelphia Plataforma de fabrico escalável para purificação de vetor viral e vetores virais assim purificados para utilização na terapia génica
WO2011109380A1 (en) 2010-03-01 2011-09-09 The Children's Hospital Of Philadelphia Nucleic acids for targeting multiple regions of the hcv genome
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
US9546369B2 (en) 2010-04-23 2017-01-17 University Of Massachusetts Multicistronic expression constructs
WO2011133901A2 (en) 2010-04-23 2011-10-27 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
US8785413B2 (en) 2010-09-27 2014-07-22 Florida Atlantic University Research Corporation Materials and methods for the treatment of pathological neovascularization in the eye
CA2827380C (en) 2011-02-12 2020-08-25 University Of Iowa Research Foundation Therapeutic nucleic acids
BR112013020734A2 (pt) 2011-02-17 2017-06-13 Univ Pennsylvania composições e métodos para alterar a especificidade do tecido e aprimorar a transferência gênica mediada por aav9
EP2500434A1 (en) 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
US20140147418A1 (en) 2011-04-15 2014-05-29 The Us Of America, As Represented By The Secretary Department Of Health And Human Services Aav mediated ctla-4 gene transfer to treat sjogren's syndrome
ES2661680T3 (es) 2011-04-21 2018-04-03 University Of Massachusetts Composiciones basadas en VAAr y métodos para tratar deficiencias de alfa-1 anti-tripsina
US8609088B2 (en) 2011-05-10 2013-12-17 Regents Of The University Of Minnesota Intranasal delivery of therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases
CN104011210B (zh) 2011-10-11 2018-05-01 布里格姆及妇女医院股份有限公司 神经退行性病症中的microRNA
US20130142861A1 (en) 2011-12-05 2013-06-06 National Yang Ming University Compositions And Method For Detecting And Treating Abnormal Liver Homeostasis And Hepatocarcinogenesis
MX367100B (es) 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
JP6385920B2 (ja) 2012-05-09 2018-09-05 オレゴン ヘルス アンド サイエンス ユニバーシティー アデノ随伴ウイルスプラスミド及びベクター
JP6417322B2 (ja) 2012-06-21 2018-11-07 アソシアシオン・アンスティテュ・ドゥ・ミオロジーAssociation Institut De Myologie 遺伝子治療ベクターの広範な遺伝子送達
US20140066595A1 (en) * 2012-09-04 2014-03-06 Thermo Fisher Scientific Biosciences Inc. Modulators of Protein Production in a Human Cell Line and Cell-free Extracts Produced Therefrom
SG11201504523UA (en) 2012-12-12 2015-07-30 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
AU2014205134B2 (en) 2013-01-14 2020-01-16 Recombinetics, Inc. Hornless livestock
EP2970946A4 (en) 2013-03-13 2016-09-07 Philadelphia Children Hospital ADENOASSOZED VIRUS VECTORS AND METHOD FOR USE THEREOF
IL298049B2 (en) 2013-03-15 2023-10-01 Univ North Carolina Chapel Hill Methods and compounds for ligation of paired AAV glycan vectors
CN105408352B (zh) 2013-04-18 2020-01-14 泰莱托恩基金会 通过双重aav载体有效递送大基因
WO2014186746A1 (en) 2013-05-16 2014-11-20 University Of Florida Research Foundation, Inc. HAIRPIN mRNA ELEMENTS AND METHODS FOR THE REGULATION OF PROTEIN TRANSLATION
ES2987399T3 (es) 2013-06-05 2024-11-14 Univ Duke Edición génica guiada por ARN y regulación génica
EP3011031B1 (en) 2013-06-17 2020-09-30 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
US10155794B2 (en) 2013-07-16 2018-12-18 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of disorders related to CEP290
JP6827320B2 (ja) 2013-08-05 2021-02-10 アカデミシュ・ジークンホイス・ライデン・ハー・オー・デー・エン・ライドス・ユニヴェルシタイル・メディシュ・セントルム LCA−8及び進行性RPを治療するための組換えAAV−Crumbsホモログ組成物及び方法
HUE050546T2 (hu) 2013-08-27 2020-12-28 Res Inst Nationwide Childrens Hospital Termékek és eljárások amiotróf laterálszklerózis kezelésére
RU2693891C1 (ru) * 2013-11-11 2019-07-05 Сангамо Байосайенсиз, Инк. Способы и композиции для лечения болезни хантингтона
GB201401707D0 (en) 2014-01-31 2014-03-19 Sec Dep For Health The Adeno-associated viral vectors
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
US10400227B2 (en) 2014-03-17 2019-09-03 The University Of Manitoba β-hexosaminidase protein variants and associated methods for treating GM2 gangliosidoses
EP3750907A3 (en) 2014-03-18 2021-04-28 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
US10975391B2 (en) 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
SI3137497T2 (sl) 2014-05-02 2025-07-31 Genzyme Corporation Vektorji AAV za gensko terapijo mrežnice in centralnega živčnega sistema
RU2711147C2 (ru) 2014-05-20 2020-01-15 Юниверсити Оф Айова Рисерч Фаундейшн Терапевтические соединения для лечения болезни хантингтона
JP7023108B2 (ja) 2014-10-21 2022-02-21 ユニバーシティ オブ マサチューセッツ 組み換えaavバリアントおよびその使用
US10174321B2 (en) 2014-12-24 2019-01-08 Uniqure Ip B.V. RNAI induced huntingtin gene suppression
EP3766973A1 (en) * 2015-04-03 2021-01-20 University Of Massachusetts Oligonucleotide compounds for targeting huntingtin mrna
US20190000940A1 (en) 2015-07-31 2019-01-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease

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