CN111448321A - Sod1双表达载体及其用途 - Google Patents

Sod1双表达载体及其用途 Download PDF

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Publication number
CN111448321A
CN111448321A CN201880061583.5A CN201880061583A CN111448321A CN 111448321 A CN111448321 A CN 111448321A CN 201880061583 A CN201880061583 A CN 201880061583A CN 111448321 A CN111448321 A CN 111448321A
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nucleic acid
promoter
sod1
isolated nucleic
sequence
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CN201880061583.5A
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Chinese (zh)
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C·米勒
R·H·小布朗
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University of Massachusetts Amherst
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University of Massachusetts Amherst
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CN201880061583.5A 2017-09-22 2018-09-21 Sod1双表达载体及其用途 Pending CN111448321A (zh)

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CN202410170569.6A CN118006607A (zh) 2017-09-22 2018-09-21 Sod1双表达载体及其用途

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US201762561932P 2017-09-22 2017-09-22
US62/561,932 2017-09-22
PCT/US2018/052173 WO2019060686A1 (en) 2017-09-22 2018-09-21 NEW DUAL EXPRESSION VECTORS OF SOD1 AND USES THEREOF

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CN201880061583.5A Pending CN111448321A (zh) 2017-09-22 2018-09-21 Sod1双表达载体及其用途
CN202410170569.6A Pending CN118006607A (zh) 2017-09-22 2018-09-21 Sod1双表达载体及其用途

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US (3) US11739330B2 (enExample)
EP (1) EP3684937A4 (enExample)
JP (3) JP7397488B2 (enExample)
CN (2) CN111448321A (enExample)
AU (2) AU2018338188B2 (enExample)
CA (1) CA3075643A1 (enExample)
WO (1) WO2019060686A1 (enExample)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9226976B2 (en) 2011-04-21 2016-01-05 University Of Massachusetts RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
CA3276269A1 (en) 2014-03-18 2026-03-02 University Of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
EP3622073A4 (en) 2017-05-09 2021-01-06 University of Massachusetts METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
US11739330B2 (en) 2017-09-22 2023-08-29 University Of Massachusetts SOD1 dual expression vectors and uses thereof
AR123524A1 (es) * 2020-09-16 2022-12-14 Biogen Ma Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela) con aav-mir-sod1
EP4291654A2 (en) 2021-02-12 2023-12-20 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
US20250101424A1 (en) * 2023-09-25 2025-03-27 California Institute Of Technology Mirna circuits for controlled gene expression

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050288243A1 (en) * 2004-04-06 2005-12-29 University Of Massachusetts Methods and compositions for treating gain-of-function disorders using RNA interference
US20170114340A1 (en) * 2014-03-18 2017-04-27 University Of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis

Family Cites Families (145)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
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
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
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
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
US8202846B2 (en) 2000-03-16 2012-06-19 Cold Spring Harbor Laboratory Methods and compositions for RNA interference
ATE450621T2 (de) 2000-03-30 2009-12-15 Whitehead Biomedical Inst Mediatoren von rns-interferenz, die rns- sequenzspezifisch sind
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
AU2002315393A1 (en) 2001-06-21 2003-01-08 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
US20050019915A1 (en) 2001-06-21 2005-01-27 Bennett C. Frank Antisense modulation of superoxide dismutase 1, soluble expression
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
ES2609014T3 (es) 2001-07-12 2017-04-18 University Of Massachusetts Producción in vivo de ARN de interferencia pequeños que median el silenciamiento génico
JP2004534543A (ja) 2001-07-13 2004-11-18 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン シュードタイプ化アデノ関連ウイルスおよびその使用
SG10202108118RA (en) 2001-11-13 2021-08-30 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)
US20060292117A1 (en) 2002-04-17 2006-12-28 Loiler Scott A 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
WO2003093460A1 (en) 2002-04-29 2003-11-13 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
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
WO2004014933A1 (en) 2002-08-07 2004-02-19 University Of Massachusetts Compositions for rna interference and methods of use thereof
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
JP2006507841A (ja) 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ
AU2004208031B2 (en) 2003-01-28 2009-10-08 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
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
ES2712695T3 (es) 2003-06-02 2019-05-14 Univ Massachusetts Métodos y composiciones para controlar la eficacia de la silenciación del ARN
EP2530157B1 (en) 2003-07-31 2016-09-28 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of miRNAs
HUE054805T2 (hu) 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik
US20060134787A1 (en) 2004-12-22 2006-06-22 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA
WO2005062937A2 (en) 2003-12-22 2005-07-14 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended sirna
KR101246862B1 (ko) 2004-03-05 2013-03-27 베니텍 리미티드 ＲＮＡｉ 제제의 동시 전달을 위한 다중 프로모터 발현카세트
EP1752536A4 (en) 2004-05-11 2008-04-16 Alphagen Co Ltd POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME
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
AU2005316476A1 (en) 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
WO2006066203A2 (en) 2004-12-16 2006-06-22 Alsgen, Llc Small interfering rna (sirna) molecules for modulating superoxide dismutase (sod)
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
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
ES2564823T3 (es) 2005-05-27 2016-03-29 Ospedale San Raffaele S.R.L. Vector génico que comprende miARN
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
WO2007027775A2 (en) 2005-08-29 2007-03-08 Isis Pharmaceuticals, Inc. Methods for use in modulating mir-122a
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
DK2514823T3 (en) 2006-03-03 2018-07-23 Promis Neurosciences Inc Methods and compositions for treating and detecting failed SOD1-mediated diseases
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
DK2548438T3 (en) 2006-11-08 2015-10-19 Veritas Bio LLC IN VIVO SUBMITTING DOUBLE-STRENGTH RNA TO A CELL
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
EP2160477B1 (en) 2007-05-23 2013-08-07 Dharmacon, Inc. Micro-RNA scaffolds and non-naturally occurring micro-RNAs
WO2008150897A2 (en) 2007-05-31 2008-12-11 University Of Iowa Research Foundation Reduction of off-target rna interference toxicity
WO2009079592A2 (en) 2007-12-17 2009-06-25 California Institute Of Technology Modulating immune system development and function through microrna mir-146
EP2247729B1 (en) 2008-02-11 2019-05-01 Phio Pharmaceuticals Corp. Modified rnai polynucleotides and uses thereof
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
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
EP2297185A1 (en) 2008-06-17 2011-03-23 Amsterdam Molecular Therapeutics (AMT) B.V. Parvoviral capsid with incorporated gly-ala repeat region
WO2010005850A1 (en) 2008-07-08 2010-01-14 The J. David Gladstone Institutes Methods and compositions for modulating angiogenesis
CA2744804C (en) 2008-09-04 2019-02-05 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
WO2010099383A2 (en) 2009-02-26 2010-09-02 The Johns Hopkins University Compositions and methods for ex vivo hepatic nucleic acid delivery
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US20120270930A1 (en) 2009-10-29 2012-10-25 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Methods and compositions for dysferlin exon-skipping
EP2498825B1 (en) 2009-11-09 2017-03-29 Genepod Therapeutics Ab Novel viral vector construct for neuron specific continuous dopa synthesis in vivo
EP2508603B1 (en) 2009-11-19 2015-05-13 National University Corporation Okayama University System for increasing gene expression, and vector supporting said system
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
ES2680915T3 (es) 2010-01-28 2018-09-11 The Children's Hospital Of Philadelphia Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica
CA2791974A1 (en) 2010-03-01 2011-09-09 The Children's Hospital Of Philadelphia Nucleic acids for targeting multiple regions of the hcv genome
EP3567106A1 (en) 2010-04-23 2019-11-13 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US9272053B2 (en) 2010-04-23 2016-03-01 University Of Massachusetts AAV-based treatment of cholesterol-related disorders
CA2833905C (en) 2010-04-23 2019-09-10 University Of Massachusetts Multicistronic expression constructs
US8906607B2 (en) 2010-04-30 2014-12-09 Cellectis Method for modulating double-strand break-induced homologous recombination
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
WO2012109667A1 (en) 2011-02-12 2012-08-16 University Of Iowa Research Foundation Therapeutic compounds
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
CA2846307C (en) 2011-08-31 2020-03-10 University College Cardiff Consultants Limited Method for diagnosing a neurodegenerative disease
EP2766482B1 (en) 2011-10-11 2016-12-07 The Brigham and Women's Hospital, Inc. Micrornas in neurodegenerative disorders
US20130142861A1 (en) 2011-12-05 2013-06-06 National Yang Ming University Compositions And Method For Detecting And Treating Abnormal Liver Homeostasis And Hepatocarcinogenesis
KR102057540B1 (ko) 2012-02-17 2019-12-19 더 칠드런스 호스피탈 오브 필라델피아 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
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
RU2730677C2 (ru) 2012-10-15 2020-08-24 Ионис Фармасьютикалз, Инк. Соединение для модуляции экспрессии гена c9orf72 и его применение
WO2014062736A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
IL239317B (en) 2012-12-12 2022-07-01 Broad Inst Inc Providing, engineering and optimizing systems, methods and compositions for sequence manipulation and therapeutic applications
CA2897932A1 (en) 2013-01-14 2014-07-17 Recombinetics, Inc. Hornless livestock
WO2014114303A1 (en) 2013-01-22 2014-07-31 Deutsches Zentrum Für Neurodegenerative Erkrankungen Dipeptide-repeat proteins as therapeutic target in neurodegenerative diseases with hexanucleotide repeat expansion
CA2905952A1 (en) 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
AU2014227766B2 (en) 2013-03-15 2018-10-04 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV 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
ES3029138T3 (en) 2013-06-05 2025-06-23 Univ Duke Rna-guided gene editing and gene regulation
MX2015017312A (es) 2013-06-17 2017-04-10 Broad Inst Inc Suministro y uso de composiciones, vectores y sistemas crispr-cas para la modificación dirigida y terapia hepáticas.
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
EP3030665B9 (en) 2013-08-05 2023-10-04 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
RS63942B1 (sr) 2013-08-27 2023-02-28 Res Inst Nationwide Childrens Hospital Proizvodi i postupci za lečenje amiotrofične lateralne skleroze
SG10201808903UA (en) 2013-10-11 2018-11-29 Ionis Pharmaceuticals Inc Compositions for modulating c9orf72 expression
US20160230172A1 (en) 2013-10-14 2016-08-11 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
BR112016013547A2 (pt) 2013-12-12 2017-10-03 Broad Inst Inc Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos
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
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
WO2015150922A2 (en) 2014-03-17 2015-10-08 The Hospital For Sick Children β-HEXOSAMINIDASE PROTEIN VARIANTS AND ASSOCIATED METHODS FOR TREATING GM2 GANGLIOSIDOSES
US10975391B2 (en) 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
EP3137497B2 (en) 2014-05-02 2025-03-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
ES2759317T3 (es) 2014-05-20 2020-05-08 Univ Iowa Res Found Compuestos terapéuticos para la enfermedad de Huntington
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
EP3674408A1 (en) 2014-06-16 2020-07-01 The Johns Hopkins University Compositions and methods for the expression of crispr guide rnas
US10590420B2 (en) * 2014-07-31 2020-03-17 Association Institut De Myologie Treatment of amyotrophic lateral sclerosis
EP3200830B1 (en) 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
RU2020140209A (ru) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
WO2016112132A1 (en) 2015-01-06 2016-07-14 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
EP4491733A3 (en) 2015-04-23 2025-03-26 University of Massachusetts Modulation of aav vector transgene expression
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
EP3289081B1 (en) 2015-04-27 2019-03-27 Genethon Compositions and methods for the treatment of nucleotide repeat expansion disorders
US10016514B2 (en) * 2015-05-15 2018-07-10 New Hope Research Foundation Polynucleotides, vectors and methods for insertion and expression of transgenes
RU2021132397A (ru) 2015-05-16 2022-02-24 Джензим Корпорейшн Генное редактирование глубоких интронных мутаций
EP3302677A4 (en) 2015-06-01 2019-02-20 University of Massachusetts CATHETER ARRANGEMENTS
US10758545B2 (en) 2015-06-25 2020-09-01 University Of Southern California Methods to treat neurological diseases
US20180214576A1 (en) 2015-07-28 2018-08-02 University Of Massachusetts Transgenic expression of dnasei in vivo delivered by an adeno-associated virus vector
US20190000940A1 (en) 2015-07-31 2019-01-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
MX2018007840A (es) 2015-12-23 2019-05-02 Crispr Therapeutics Ag Materiales y metodos para el tratamiento de la esclerosis lateral amiotrofica y/o la degeneracion lobar frontotemporal.
EP3518667B1 (en) 2016-09-30 2024-10-30 Regeneron Pharmaceuticals, Inc. Rodents having a hexanucleotide repeat expansion in a c9orf72 locus
EP3622073A4 (en) 2017-05-09 2021-01-06 University of Massachusetts METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
US11739330B2 (en) 2017-09-22 2023-08-29 University Of Massachusetts SOD1 dual expression vectors and uses thereof

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050288243A1 (en) * 2004-04-06 2005-12-29 University Of Massachusetts Methods and compositions for treating gain-of-function disorders using RNA interference
US20170114340A1 (en) * 2014-03-18 2017-04-27 University Of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
CHRISTIAN MUELLER等: "Sustained miRNA-mediated Knockdown of Mutant AAT With Simultaneous Augmentation of Wild-type AAT Has Minimal Effect on Global Liver miRNA Profiles", 《MOL THER.》 *
LORELEI STOICA等: "Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model", 《ANN NEUROL.》 *

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