JP2019533439A5 - - Google Patents

Download PDF

Info

Publication number
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
Authority
JP
Japan
Prior art keywords
nucleic acid
sequence
transgene
isolated nucleic
itr
Prior art date
Legal status (The legal status 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 status listed.)
Granted
Application number
JP2019515997A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019533439A (ja
JP6884430B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2017/052902 external-priority patent/WO2018057855A1/en
Publication of JP2019533439A publication Critical patent/JP2019533439A/ja
Publication of JP2019533439A5 publication Critical patent/JP2019533439A5/ja
Priority to JP2021077175A priority Critical patent/JP7205933B2/ja
Application granted granted Critical
Publication of JP6884430B2 publication Critical patent/JP6884430B2/ja
Priority to JP2022205188A priority patent/JP7613759B2/ja
Priority to JP2024224502A priority patent/JP2025063053A/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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)

Application Number Title Priority Date Filing Date
JP2021077175A Division JP7205933B2 (ja) 2016-09-22 2021-04-30 ハンチントン病のaav処置

Publications (3)

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

Family

ID=61691085

Family Applications (4)

Application Number Title Priority Date Filing Date
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

Family Applications After (3)

Application Number Title Priority Date Filing Date
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)

Country Link
US (4) US10457940B2 (enExample)
EP (1) EP3515505A4 (enExample)
JP (4) JP6884430B2 (enExample)
KR (1) KR102271675B1 (enExample)
CN (2) CN110035776B (enExample)
AU (2) AU2017330411B2 (enExample)
BR (2) BR112019005548B1 (enExample)
CA (1) CA3037929A1 (enExample)
CL (1) CL2019000732A1 (enExample)
CO (1) CO2019003846A2 (enExample)
IL (3) IL265528B2 (enExample)
MX (2) MX2019003376A (enExample)
MY (1) MY195386A (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
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
MX2019013172A (es) * 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
KR20200120627A (ko) * 2018-02-13 2020-10-21 도레이 카부시키가이샤 인지증 검출을 위한 키트 또는 디바이스 및 방법
CN112105731B (zh) * 2018-03-30 2024-08-30 日内瓦大学 微小rna表达构建体及其用途
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
EP3887522A1 (en) 2018-11-29 2021-10-06 University of Massachusetts Modulation of sptlc1 via recombinant adeno-associated vectors
PE20211890A1 (es) * 2019-01-09 2021-09-22 Univ De Coimbra Arn bicatenario y usos del mismo
CA3155921A1 (en) * 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. HUNTINGTINE (HTT) TARGETING RNAI AGENT COMPOSITIONS AND METHODS OF USE THEREOF
CN115443339A (zh) * 2019-12-20 2022-12-06 阿斯克肋匹奥生物制药公司 用于治疗亨廷顿病的方法
JP2023542211A (ja) * 2020-09-21 2023-10-05 アスクレピオス バイオファーマシューティカル, インコーポレイテッド 神経疾患を処置するための方法
WO2022155364A1 (en) * 2021-01-13 2022-07-21 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
CA2264482A1 (en) 1996-09-06 1998-03-12 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
AU762220B2 (en) 1998-05-28 2003-06-19 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The 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
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
CA2921821A1 (en) * 2001-07-12 2003-01-23 University Of Massachusetts In vivo production of small interfering rnas that mediate gene silencing
EP1419245A4 (en) 2001-07-13 2006-04-05 Univ Iowa Res Found ADENO-ASSOCIATED PSEUDOTYPE VIRUSES AND USES THEREOF
CA2455424A1 (en) * 2001-08-07 2003-02-20 University Of Delaware Compositions and methods for the prevention and treatment of huntington's disease
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
PT2573170T (pt) 2001-12-17 2018-03-26 Univ Pennsylvania Sequências de um vírus adenoassociado (aav) de serotipo 9, vetor contendo as mesmas, e suas utilizações
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)
US20060292117A1 (en) 2002-04-17 2006-12-28 Loiler Scott A 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
ES2280694T3 (es) 2002-04-29 2007-09-16 The Trustees Of The University Of Pennsylvania Metodo para la extraccion directa y la amplificacion de virus integrados del adn celular de tejidos.
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
JP2006518372A (ja) 2003-01-28 2006-08-10 セレクティス 脊椎動物の体組織においてエクスビボかつイントトで相同的組換えを誘発するためのメガヌクレアーゼの使用およびその応用
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
EP1633767B1 (en) 2003-06-02 2018-11-21 University of Massachusetts Methods and compositions for controlling efficacy of rna silencing
WO2008005562A2 (en) 2006-07-07 2008-01-10 University Of Massachusetts Rna silencing compositions and methods for the treatment of huntington's disease
US8680063B2 (en) * 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
HUE035567T2 (en) 2003-09-30 2018-05-28 Univ Pennsylvania Adeno-associated virus (AAV) clusters, sequences, vectors and applications containing them
NZ550284A (en) 2004-03-05 2009-04-30 Benitec Ltd Multiple promoter expression cassettes for simultaneous delivery of RNAi agents
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
US20060189564A1 (en) 2004-10-22 2006-08-24 Medtronic, Inc. Methods and sequences to suppress pro-inflamatory cytokine actions locally to treat pain
NZ555830A (en) 2004-12-15 2009-01-31 Univ North Carolina Chimeric vectors
EP1866422B1 (en) 2005-04-07 2016-04-06 The Trustees of The University of Pennsylvania Method of increasing the function of an aav vector
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
PL2002003T3 (pl) 2005-05-27 2016-06-30 Ospedale San Raffaele Srl Wektor genetyczny zawierający 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
EP2338991B1 (en) 2005-08-29 2017-01-18 Regulus Therapeutics Inc. Methods for use in modulating MIR-122a
US7977314B2 (en) 2005-12-02 2011-07-12 Amorfix Life Sciences Limited Methods and compositions to treat and detect misfolded-SOD1 mediated diseases
ES2531052T3 (es) 2006-01-05 2015-03-10 Univ Ohio State Res Found Métodos y composiciones basados en microARN para el diagnóstico de cánceres de mama
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
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US20080199961A1 (en) * 2006-08-25 2008-08-21 Avi Biopharma, Inc. ANTISENSE COMPOSITION AND METHOD FOR INHIBITION OF miRNA BIOGENESIS
ES2434169T3 (es) 2006-11-08 2013-12-13 Veritas Bio, Llc Entrega in vivo de ARN bicatenario a una célula objetivo
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
AU2008247427A1 (en) 2007-05-03 2008-11-13 Rosetta Inpharmatics Llc Compositions comprising miR34 therapeutic agents for treating cancer
AU2008256886B2 (en) 2007-05-23 2013-02-28 Dharmacon, Inc. Micro-RNA scaffolds and non-naturally occurring micro-RNAs
EP2530152B1 (en) 2007-05-31 2017-12-27 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
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
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
US8222221B2 (en) 2008-06-04 2012-07-17 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
EP2310507A4 (en) 2008-07-08 2013-03-20 David Gladstone Inst METHOD AND COMPOSITIONS FOR ANGIOGENIC MODULATION
JP5789513B2 (ja) 2008-09-04 2015-10-07 ガレニア コーポレーション シナプス小胞サイクリングアッセイおよびシステム
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
ES2786039T3 (es) * 2009-04-30 2020-10-08 Ospedale San Raffaele Srl Vector génico
DK3421603T3 (da) 2009-05-02 2022-01-10 Genzyme Corp Genterapi for neurodegenerative forstyrrelser
US8734809B2 (en) * 2009-05-28 2014-05-27 University Of Massachusetts 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
KR102173836B1 (ko) * 2009-09-11 2020-11-05 아이오니스 파마수티컬즈, 인코포레이티드 헌팅틴 발현의 조절
US20120270930A1 (en) 2009-10-29 2012-10-25 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Methods and compositions for dysferlin exon-skipping
MX2012005450A (es) 2009-11-09 2012-08-15 Genepod Therapeutics Ab Constructo de vector virico novedoso para sintesis dopa continua optimizada especificamente de neuronas 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
WO2011088081A1 (en) 2010-01-12 2011-07-21 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
CN102947453A (zh) 2010-01-28 2013-02-27 费城儿童医院 用于病毒载体纯化的可扩缩生产平台和用于基因治疗中的如此纯化的病毒载体
AU2011223820B2 (en) 2010-03-01 2016-01-14 The Children's Hospital Of Philadelphia Nucleic acids for targeting multiple regions of the HCV genome
JP5963743B2 (ja) 2010-04-23 2016-08-03 ユニバーシティ オブ マサチューセッツ Cnsターゲティングaavベクターおよびその使用方法
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
CA2833912C (en) 2010-04-23 2021-09-21 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
ES2739804T3 (es) 2011-02-12 2020-02-04 Univ Iowa Res Found Compuestos terapéuticos
SG10201601110VA (en) 2011-02-17 2016-03-30 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
EP2699258A4 (en) 2011-04-15 2014-10-29 Us Sec Dep Of Health And Human Services AAV-mediated CTLA-4 gene transfer for the treatment of SJÖGREN 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
KR20140074997A (ko) 2011-10-11 2014-06-18 더 브리검 앤드 우먼즈 하스피털, 인크. 신경병성 질환에서 마이크로rna
US20130142861A1 (en) 2011-12-05 2013-06-06 National Yang Ming University Compositions And Method For Detecting And Treating Abnormal Liver Homeostasis And Hepatocarcinogenesis
RU2653444C2 (ru) 2012-02-17 2018-05-08 Дзе Чилдрен'З Хоспитал Оф Филадельфия Композиции вектора aav и способы переноса генов в клетки, органы и ткани
WO2013170078A1 (en) 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
CN104704123A (zh) 2012-06-21 2015-06-10 肌肉学研究协会 基因治疗载体的广泛的基因递送
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
AU2013359199C1 (en) 2012-12-12 2021-06-17 Massachusetts Institute Of Technology Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
EP2943060A4 (en) 2013-01-14 2016-11-09 Recombinetics Inc LIVESTOCK WITHOUT HORN
AU2014244167A1 (en) 2013-03-13 2015-10-08 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
WO2014144229A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav vectors
DK2986635T3 (en) 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
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
ES2991293T3 (es) 2013-06-05 2024-12-03 Univ Duke Edición génica guiada por ARN y regulación génica
SG11201510284XA (en) 2013-06-17 2016-01-28 Broad Inst 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
CA2919995A1 (en) 2013-08-05 2015-02-12 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
EP4219727A3 (en) 2013-08-27 2023-09-06 Research Institute at Nationwide Children's Hospital Products and methods for treatment of amyotrophic lateral sclerosis
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
WO2015150922A2 (en) 2014-03-17 2015-10-08 The Hospital For Sick Children β-HEXOSAMINIDASE PROTEIN VARIANTS AND ASSOCIATED METHODS FOR TREATING GM2 GANGLIOSIDOSES
US10280418B2 (en) 2014-03-18 2019-05-07 Univeristy Of Massachusetts RAAV-based compositions and methods for treating amyotrophic lateral sclerosis
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
PT3137497T (pt) 2014-05-02 2021-07-12 Genzyme Corp Vetores de aav para terapia genética na retina e snc
ES2759317T3 (es) * 2014-05-20 2020-05-08 Univ Iowa Res Found Compuestos terapéuticos para la enfermedad de Huntington
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
LT3237618T (lt) * 2014-12-24 2019-07-10 Uniqure Ip B.V. Rnri sukeltas hantingtino geno slopinimas
HUE050441T2 (hu) * 2015-04-03 2020-12-28 Univ Massachusetts Huntingtin-mRNS célzására szolgáló oligonukleotid-vegyületek
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

Similar Documents

Publication Publication Date Title
JP2019533439A5 (enExample)
JP2025063053A5 (enExample)
JP2018108113A5 (enExample)
JP2011101646A5 (enExample)
JP2020019772A5 (enExample)
RU2019111648A (ru) Средство лечения болезни гентингтона на основе aav
JP6366581B2 (ja) 改変されたアデノ随伴ウイルスベクター組成物
HRP20241180T1 (hr) Modificirani faktor ix, te pripravci, postupci i upotreba za prijenos gena u stanice, organe i tkiva
ES2732023T3 (es) Supresión del gen de la huntingtina inducida por ARNi
JP2020522269A5 (enExample)
FI3684423T3 (fi) Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä
JP2014012022A5 (enExample)
RU2017116576A (ru) Композиции и способы лечения бокового амиотрофического склероза (als)
JP2017535266A5 (enExample)
JP2017509632A5 (enExample)
JP2018522529A5 (enExample)
JP2020532981A5 (enExample)
JP2017510298A5 (enExample)
JP2017533715A5 (enExample)
JP2015521465A5 (enExample)
JP2018506304A5 (enExample)
HRP20231077T1 (hr) Genska terapija za pigmentni retinitis
JP2020519284A5 (enExample)
JP2013509168A5 (enExample)
JP2020519294A5 (enExample)