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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JP6884430B2 (ja
JP2019533439A (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 (OSRAM) 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 (19)

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US (4) US10457940B2 (OSRAM)
EP (1) EP3515505A4 (OSRAM)
JP (4) JP6884430B2 (OSRAM)
KR (1) KR102271675B1 (OSRAM)
CN (2) CN110035776B (OSRAM)
AU (2) AU2017330411B2 (OSRAM)
BR (2) BR112019005548B1 (OSRAM)
CA (1) CA3037929A1 (OSRAM)
CL (1) CL2019000732A1 (OSRAM)
CO (1) CO2019003846A2 (OSRAM)
IL (3) IL265528B2 (OSRAM)
MX (2) MX2019003376A (OSRAM)
MY (1) MY195386A (OSRAM)
PE (2) PE20241233A1 (OSRAM)
PH (1) PH12019500626A1 (OSRAM)
RU (1) RU2749971C2 (OSRAM)
SA (1) SA519401379B1 (OSRAM)
WO (1) WO2018057855A1 (OSRAM)
ZA (1) ZA201902226B (OSRAM)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3134522B1 (en) 2014-04-25 2021-10-06 University of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics Inc composições e métodos de tratamento da doença de huntington
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
US12371710B2 (en) 2018-05-15 2025-07-29 University Of Massachusetts Modified AAV constructs and uses thereof
US12391947B2 (en) 2018-11-29 2025-08-19 University Of Massachusetts Modulation of SPTLC1 via recombinant adeno-associated vectors
BR112021013109A2 (pt) * 2019-01-09 2021-10-13 Universidade De Coimbra Rna de fita dupla e seus usos.
CA3155921A1 (en) * 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. HUNTINGTINE (HTT) TARGETING RNAI AGENT COMPOSITIONS AND METHODS OF USE THEREOF
AU2020407119A1 (en) * 2019-12-20 2022-07-14 AskBio Inc. Methods for treating Huntington's disease
US20230365963A1 (en) * 2020-09-21 2023-11-16 Asklepios Biopharmaceutical, Inc. Methods for treating neurological disease
US20240084323A1 (en) * 2021-01-13 2024-03-14 Dignity Health Modulation of chitinase protein expression
CN115141848A (zh) * 2021-03-30 2022-10-04 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
JP2001500015A (ja) 1996-09-06 2001-01-09 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 T7ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法
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
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
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
WO2003004660A1 (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
ES2606290T3 (es) * 2001-07-12 2017-03-23 University Of Massachusetts Producción in vivo de ARN de interferencia pequeños que median el silenciamiento génico
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
SG168422A1 (en) 2001-11-13 2011-02-28 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2526341T3 (es) 2001-12-17 2015-01-09 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
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)
WO2003089612A2 (en) 2002-04-17 2003-10-30 University Of Florida Research Foundation, Inc. IMPROVED rAAV VECTORS
WO2003089011A1 (en) 2002-04-19 2003-10-30 University Of Florida rAAV VECTOR-BASED COMPOSITIONS AND METHODS FOR THE PREVENTION AND TREATMENT OF MAMMALIAN DISEASES
EP1359217B1 (en) 2002-04-29 2006-12-13 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
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US20050255086A1 (en) 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
US20050106731A1 (en) * 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
WO2004067753A2 (en) 2003-01-28 2004-08-12 Cellectis Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof.
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
AU2004248136B2 (en) 2003-06-02 2011-09-15 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
EP3910063A1 (en) 2003-09-30 2021-11-17 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
KR101246862B1 (ko) 2004-03-05 2013-03-27 베니텍 리미티드 RNAi 제제의 동시 전달을 위한 다중 프로모터 발현카세트
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
CN101124328A (zh) 2004-12-15 2008-02-13 北卡罗来纳查佩尔山大学 嵌合载体
ATE524546T1 (de) 2005-03-25 2011-09-15 Medtronic Inc Verwendung von anti-tnf oder anti-il1-rnai zur unterdrückung der wirkung entzündungsfördernder cytokine zur lokalen schmerzbehandlung
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
SI2002003T1 (sl) 2005-05-27 2016-05-31 Ospedale San Raffaele S.R.L. Genski vektor, ki vsebuje mi-RNA
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
DE602006019455D1 (de) 2005-08-29 2011-02-17 Regulus Therapeutics Inc Verfahren für mir-122a-modulation
CN103642900B (zh) 2006-01-05 2016-04-13 俄亥俄州立大学研究基金会 用于诊断和治疗实体癌的基于微小rna的方法和组合物
WO2008016391A2 (en) 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
JP5823663B2 (ja) 2006-03-03 2015-11-25 プロミス ニューロサイエンシズ インコーポレイテッド ミスフォールドsod1媒介疾患を処置および検出するための方法および組成物
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
DK2094086T3 (da) 2006-11-08 2013-11-25 Veritas Bio LLC Indgivelse in vivo af dobbeltstrenget rna til en målcelle
EP3492596A1 (en) 2007-04-09 2019-06-05 University of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
ES2907452T3 (es) 2007-04-12 2022-04-25 The Provost Fellows Found Scholars & The Other Members Of Board Of The College Of The Holy & Undiv T Supresión y reemplazo genético
JP2010525826A (ja) 2007-05-03 2010-07-29 ロゼッタ インファーマティックス エルエルシー 癌を治療するためのmir34治療剤を含む組成物
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
ES2549122T3 (es) 2007-05-31 2015-10-23 University Of Iowa Research Foundation Reducción de toxicidad por interferencia de ARN desviada de su diana
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
US9090894B2 (en) 2007-12-17 2015-07-28 California Institute Of Technology Modulating immune system development and function through microRNA MIR-146
WO2009108274A2 (en) 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
US8404659B2 (en) 2008-03-07 2013-03-26 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
US20110171262A1 (en) 2008-06-17 2011-07-14 Andrew Christian Bakker Parvoviral capsid with incorporated gly-ala repeat region
US20110172293A1 (en) 2008-07-08 2011-07-14 Fish Jason E Methods and Compositions for Modulating Angiogenesis
US20120053084A1 (en) 2008-09-04 2012-03-01 Galenea Corp. 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
US20110305772A1 (en) 2009-02-26 2011-12-15 The Johns Hopkins University Compositions and methods for ex vivo hepatic nucleic acid delivery
AU2010243276B2 (en) * 2009-04-30 2016-09-15 Fondazione Telethon Ets Gene vector
EP3988660B1 (en) 2009-05-02 2025-05-14 Genzyme Corporation Gene therapy for neurodegenerative disorders
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
EP3626823A1 (en) * 2009-09-11 2020-03-25 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
US20120309816A1 (en) 2009-11-09 2012-12-06 Genepod Therapeutics Ab Novel viral vector construct for neuron specific optimized continuous DOPA synthesis in vivo
CN102741405B (zh) 2009-11-19 2015-03-04 国立大学法人冈山大学 提高基因表达的系统和保持有该系统的载体
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
WO2011094198A1 (en) 2010-01-28 2011-08-04 The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
WO2011109380A1 (en) 2010-03-01 2011-09-09 The Children's Hospital Of Philadelphia Nucleic acids for targeting multiple regions of the hcv genome
EP3540055A1 (en) 2010-04-23 2019-09-18 University of Massachusetts Cns targeting aav vectors and methods of use thereof
CA2833905C (en) 2010-04-23 2019-09-10 University Of Massachusetts Multicistronic expression constructs
US9272053B2 (en) 2010-04-23 2016-03-01 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
EP2673286B1 (en) 2011-02-12 2019-07-03 University of Iowa Research Foundation Therapeutic compounds
SG10202007803QA (en) 2011-02-17 2020-09-29 Univ Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
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
WO2012142434A2 (en) 2011-04-15 2012-10-18 THE UNITED STATES OF AMERICA, as represented by the secretary, Depart.of Healtth and Human Services Aav mediated ctla-4 gene transfer to treat sjogren's syndrome
US9226976B2 (en) 2011-04-21 2016-01-05 University Of Massachusetts RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
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
CN104520428B (zh) 2012-02-17 2018-09-21 费城儿童医院 将基因转移到细胞、器官和组织的aav载体组合物和方法
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
US20150182637A1 (en) 2012-06-21 2015-07-02 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
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
BR112015013784A2 (pt) 2012-12-12 2017-07-11 Massachusetts Inst Technology aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas
NZ629569A (en) 2013-01-14 2018-07-27 Recombinetics Inc Hornless livestock
CA2905952A1 (en) 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
IL293294B2 (en) 2013-03-15 2023-04-01 Univ North Carolina Chapel Hill Methods and compounds for ligation of paired aav glycan vectors
EP2986635B1 (en) 2013-04-18 2018-10-03 Fondazione Telethon Effective delivery of large genes by dual aav vectors
US10006049B2 (en) 2013-05-16 2018-06-26 University Of Florida Research Foundation, Incorporated Hairpin mRNA elements and methods for the regulation of protein translation
KR20230136697A (ko) 2013-06-05 2023-09-26 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
DK3011031T3 (da) 2013-06-17 2020-12-21 Broad Inst Inc Fremføring og anvendelse af crispr-cas-systemerne, vektorer og sammensætninger til levermålretning og -terapi
WO2015009575A1 (en) 2013-07-16 2015-01-22 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of disorders related to cep290
AU2014305218B2 (en) 2013-08-05 2020-01-16 Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum Recombinant AAV-Crumbs homologue composition and methods for treating LCA-8 and progressive RP
HRP20200862T1 (hr) 2013-08-27 2020-08-21 Research Institute At Nationwide Children's Hospital Proizvodi i metode liječenja amiotrofične lateralne skleroze
EP3068905A4 (en) * 2013-11-11 2017-07-05 Sangamo BioSciences, Inc. Methods and compositions for treating huntington's disease
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
HUE054748T2 (hu) 2014-03-17 2021-09-28 Hospital For Sick Children Béta-hexózaminidáz fehérje változatai és kapcsolódó eljárások GM2 gangliozidózis kezelésére
JP6756700B2 (ja) 2014-03-18 2020-09-16 ユニバーシティ オブ マサチューセッツ 筋萎縮性側索硬化症を処置するためのrAAVベースの組成物および方法
EP3134522B1 (en) 2014-04-25 2021-10-06 University of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
RU2711147C2 (ru) 2014-05-20 2020-01-15 Юниверсити Оф Айова Рисерч Фаундейшн Терапевтические соединения для лечения болезни хантингтона
RU2738421C2 (ru) 2014-10-21 2020-12-14 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
EA037696B1 (ru) 2014-12-24 2021-05-12 ЮНИКЕР АйПи Б.В. Супрессия гена гентингтина, индуцированная рнк-интерференцией
PL3277814T3 (pl) * 2015-04-03 2020-11-30 University Of Massachusetts Związki oligonukleotydowe ukierunkowane na mrna huntingtyny
WO2017023724A1 (en) 2015-07-31 2017-02-09 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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