JP5963743B2 - Ｃｎｓターゲティングａａｖベクターおよびその使用方法 - Google Patents

Ｃｎｓターゲティングａａｖベクターおよびその使用方法 Download PDF

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JP5963743B2
JP5963743B2 JP2013506330A JP2013506330A JP5963743B2 JP 5963743 B2 JP5963743 B2 JP 5963743B2 JP 2013506330 A JP2013506330 A JP 2013506330A JP 2013506330 A JP2013506330 A JP 2013506330A JP 5963743 B2 JP5963743 B2 JP 5963743B2
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raav
cns
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administration
vector
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JP2013531471A5 (enExample
JP2013531471A (ja
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ガオ，グアンピン
チャン，ホンウエイ
ワン，ホンイエン
シュイ，ツオシャン
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ユニバーシティ オブ マサチューセッツ
ユニバーシティ オブ マサチューセッツ
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JP2013506330A 2010-04-23 2011-04-22 Ｃｎｓターゲティングａａｖベクターおよびその使用方法 Active JP5963743B2 (ja)

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US32762710P 2010-04-23 2010-04-23
US61/327,627 2010-04-23
PCT/US2011/033616 WO2011133890A1 (en) 2010-04-23 2011-04-22 Cns targeting aav vectors and methods of use thereof

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JP2016000310A Division JP6235619B2 (ja) 2010-04-23 2016-01-04 Ｃｎｓターゲティングａａｖベクターおよびその使用方法
JP2016000309A Division JP6422897B2 (ja) 2010-04-23 2016-01-04 Ｃｎｓターゲティングａａｖベクターおよびその使用方法

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JP2013531471A JP2013531471A (ja) 2013-08-08
JP2013531471A5 JP2013531471A5 (enExample) 2014-08-21
JP5963743B2 true JP5963743B2 (ja) 2016-08-03

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JP2013506330A Active JP5963743B2 (ja) 2010-04-23 2011-04-22 Ｃｎｓターゲティングａａｖベクターおよびその使用方法
JP2016000309A Active JP6422897B2 (ja) 2010-04-23 2016-01-04 Ｃｎｓターゲティングａａｖベクターおよびその使用方法
JP2016000310A Active JP6235619B2 (ja) 2010-04-23 2016-01-04 Ｃｎｓターゲティングａａｖベクターおよびその使用方法
JP2018195573A Active JP6879988B2 (ja) 2010-04-23 2018-10-17 Ｃｎｓターゲティングａａｖベクターおよびその使用方法
JP2019005016A Active JP6941632B2 (ja) 2010-04-23 2019-01-16 Ｃｎｓターゲティングａａｖベクターおよびその使用方法
JP2021077161A Pending JP2021138702A (ja) 2010-04-23 2021-04-30 Ｃｎｓターゲティングａａｖベクターおよびその使用方法

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JP2016000309A Active JP6422897B2 (ja) 2010-04-23 2016-01-04 Ｃｎｓターゲティングａａｖベクターおよびその使用方法
JP2016000310A Active JP6235619B2 (ja) 2010-04-23 2016-01-04 Ｃｎｓターゲティングａａｖベクターおよびその使用方法
JP2018195573A Active JP6879988B2 (ja) 2010-04-23 2018-10-17 Ｃｎｓターゲティングａａｖベクターおよびその使用方法
JP2019005016A Active JP6941632B2 (ja) 2010-04-23 2019-01-16 Ｃｎｓターゲティングａａｖベクターおよびその使用方法
JP2021077161A Pending JP2021138702A (ja) 2010-04-23 2021-04-30 Ｃｎｓターゲティングａａｖベクターおよびその使用方法

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US (8) US9102949B2 (enExample)
EP (6) EP3567106A1 (enExample)
JP (6) JP5963743B2 (enExample)
CA (3) CA2833908C (enExample)
DK (2) DK2561073T3 (enExample)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019052150A (ja) * 2010-04-23 2019-04-04 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

Families Citing this family (228)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101014207B1 (ko) 2001-11-13 2011-02-14 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 아데노-결합 바이러스 (ａａｖ) 서열을 검출 및/또는 확인하는 방법 및 그 방법에 의해 확인된 신규한 서열을 분리하는 방법
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
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
US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
EP2561075B1 (en) 2010-04-23 2018-06-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
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
US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
WO2013006514A2 (en) * 2011-07-01 2013-01-10 University Of South Florida Recombinant adeno-associated virus-mediated expression of fractalkine for treatment of neuroinflammatory and neurodegenerative diseases
BR112014020325A2 (pt) * 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
JP6189415B2 (ja) * 2012-04-02 2017-08-30 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． 細胞質および細胞骨格タンパク質の産生のための修飾ポリヌクレオチド
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
EP2855684A1 (en) * 2012-05-25 2015-04-08 Commissariat à l'Énergie Atomique et aux Énergies Alternatives Vector for the selective silencing of a gene in astrocytes
US20150182637A1 (en) * 2012-06-21 2015-07-02 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
BR112015002168A2 (pt) * 2012-08-01 2017-11-07 Nationwide Childrens Hospital liberação intratecal de vírus 9 adeno-associado recombinante
US12024568B2 (en) * 2012-09-13 2024-07-02 Cornell University Treatment of brain cancers using central nervous system mediated gene transfer of monoclonal antibodies
JP2015529685A (ja) 2012-09-17 2015-10-08 ザ・リサーチ・インスティテュート・アット・ネイションワイド・チルドレンズ・ホスピタルＴｈｅ Ｒｅｓｅａｒｃｈ Ｉｎｓｔｉｔｕｔｅ Ａｔｎａｔｉｏｎｗｉｄｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ 筋萎縮性側索硬化症の処置のための組成物および方法
EP2922554B1 (en) * 2012-11-26 2022-02-23 ModernaTX, Inc. Terminally modified rna
RS64664B1 (sr) 2013-02-15 2023-11-30 Bioverativ Therapeutics Inc Gen optimizovanog faktora viii
AU2014244167A1 (en) * 2013-03-13 2015-10-08 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
PT2956056T (pt) 2013-03-14 2019-09-20 Univ Laval Sistema de eletrorretinografia (erg) para avaliação de distúrbios psiquiátricos
LT2984166T (lt) 2013-03-15 2020-08-10 The Trustees Of The University Of Pennsylvania Kompozicijos, skirtos mpsi gydymui
KR20240093939A (ko) * 2013-05-15 2024-06-24 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
MX2016001044A (es) 2013-07-22 2016-09-09 The Children's Hospital Of Philadelphia Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos.
MX373332B (es) * 2013-07-26 2020-05-21 Univ Iowa Res Found UNA PARTÍCULA rAAV Y UN AGENTE DE INMUNOSUPRESIÓN PARA USARSE EN EL TRATAMIENTO DE ENFERMEDADES.
LT3039146T (lt) 2013-08-27 2020-07-27 Research Institute at Nationwide Children`s Hospital Produktai ir būdai, skirti amiotropinės lateralinės sklerozės gydymui
EP3561062A1 (en) 2013-09-13 2019-10-30 California Institute of Technology Selective recovery
CN105745326A (zh) * 2013-10-24 2016-07-06 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
WO2015069647A1 (en) 2013-11-05 2015-05-14 The Research Institute At Nationwide Children's Hospital COMPOSITIONS AND METHODS FOR INHIBITING NF- kB AND SOD-1 TO TREAT AMYOTROPHIC LATERAL SCLEROSIS
CN105765066B (zh) 2013-11-29 2018-12-14 宝生物工程株式会社 用于定量腺伴随病毒的方法
SG11201604705UA (en) * 2013-12-11 2016-07-28 Sumitomo Chemical Co Method for manufacturing ciliary marginal zone-like structure
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
US10280418B2 (en) 2014-03-18 2019-05-07 Univeristy Of Massachusetts RAAV-based compositions and methods for treating amyotrophic lateral sclerosis
WO2015164723A1 (en) * 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
JP6741591B2 (ja) 2014-05-02 2020-08-19 ジェンザイム・コーポレーション 網膜およびｃｎｓ遺伝子治療用ａａｖベクター
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
JP6842410B2 (ja) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるａａｖベクター
AU2015335923B2 (en) 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
SG10202007103TA (en) * 2014-11-05 2020-09-29 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CN112375760A (zh) 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
KR20230169197A (ko) 2014-11-14 2023-12-15 보이저 테라퓨틱스, 인크. 근위축성 측삭 경화증(als)을 치료하는 조성물 및 방법
AU2015349759B2 (en) * 2014-11-21 2022-01-06 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
US10876134B2 (en) 2014-12-16 2020-12-29 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
EA201791805A1 (ru) 2015-02-10 2018-05-31 Джензим Корпорейшн ВАРИАНТ СРЕДСТВА ДЛЯ RNAi
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
EA036051B1 (ru) * 2015-03-10 2020-09-18 Зе Трастис Оф Коламбия Юниверсити Ин Зе Сити Оф Нью-Йорк РЕКОМБИНАНТНЫЙ АДЕНОАССОЦИИРОВАННЫЙ ВЕКТОР (rAAV), СПОСОБНЫЙ ПРОНИКАТЬ СКВОЗЬ ГЕМАТОЭНЦЕФАЛИЧЕСКИЙ БАРЬЕР (ГЭБ), И ЕГО ПРИМЕНЕНИЕ ДЛЯ ВОССТАНОВЛЕНИЯ ЭКСПРЕССИИ Glut1
WO2016172008A1 (en) 2015-04-24 2016-10-27 University Of Massachusetts Modified aav constructions and uses thereof
US20170067028A1 (en) * 2015-05-15 2017-03-09 Douglas J. Ballon Radiolabeling of adeno associated virus
RU2021134920A (ru) * 2015-05-29 2022-04-15 Дзе Трастиз Оф Дзе Юнивёрсити Оф Пенсильвания Композиции и способы деградации неправильно упакованных белков
CN108138159A (zh) 2015-06-23 2018-06-08 费城儿童医院 经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途
US10801040B2 (en) 2015-07-07 2020-10-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the peripheral nervous system of a subject
US10047377B2 (en) * 2015-09-22 2018-08-14 Loyola University Of Chicago Methods for modulating KLHL1 levels, methods for modulating current activity in T-type calcium channels, molecules therefor, and methods for identifying molecules therefor
US10597655B2 (en) * 2015-09-22 2020-03-24 Loyola University Chicago Methods for modulating KLHL1 levels, methods for modulating current activity in T-type calcium channels, molecules therefor, and methods for identifying molecules therefor
US10745447B2 (en) 2015-09-28 2020-08-18 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
WO2017060510A1 (en) * 2015-10-09 2017-04-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for the treatment of alzheimer's disease
CA3002980A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
ES2978086T3 (es) 2015-10-22 2024-09-05 Univ Massachusetts Terapia génica con aspartoacilasa en el tratamiento de enfermedad de Canavan
CN105420275A (zh) * 2015-11-27 2016-03-23 中国科学院苏州生物医学工程技术研究所 制备外源功能基因定点整合的人神经干细胞的方法
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
ES2918998T3 (es) 2015-12-11 2022-07-21 Univ Pennsylvania Método de purificación escalable para AAVrh10
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
SG11201804713VA (en) 2015-12-11 2018-07-30 California Inst Of Techn TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
PH12018501628B1 (en) 2016-02-01 2023-11-10 Bioverativ Therapeutics Inc Optimized factor viii genes
US11826433B2 (en) 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
JP7360241B2 (ja) 2016-02-03 2023-10-12 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 ムコ多糖症ｉ型を治療するための遺伝子治療
EP4094780A3 (en) * 2016-02-12 2023-02-08 University of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
MX394648B (es) * 2016-03-31 2025-03-24 Univ Cincinnati Composiciones que contienen vectores modificados de virus adeno-asociados para usarse en el tratamiento de esclerosis lateral amiotrófica.
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
SG10201912761UA (en) 2016-04-15 2020-02-27 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
GB201608046D0 (en) 2016-05-09 2016-06-22 Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The Treatment of complement-mediated disorders
CN116333057A (zh) 2016-05-13 2023-06-27 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
CN117904112A (zh) 2016-05-18 2024-04-19 沃雅戈治疗公司 调节性多核苷酸
WO2017218852A1 (en) 2016-06-15 2017-12-21 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
US20190160187A1 (en) * 2016-07-26 2019-05-30 Cornell University Gene therapy for the treatment of aldehyde dehydrogenase deficiency
KR20240172773A (ko) 2016-08-15 2024-12-10 젠자임 코포레이션 Aav의 검출 방법
EP3506817A4 (en) 2016-08-30 2020-07-22 The Regents of The University of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
US11111271B2 (en) 2016-09-28 2021-09-07 Cohbar, Inc. Therapeutic peptides
US20200048332A1 (en) 2016-10-12 2020-02-13 Bioverativ Usa Inc. Anti-c1s antibodies and methods of use thereof
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
CN110139933B (zh) 2016-11-09 2024-03-08 英特瑞克斯顿股份有限公司 共济蛋白表达构建体
US11117930B2 (en) 2017-02-23 2021-09-14 Adrx, Inc. Peptide inhibitors of transcription factor aggregation
TWI808079B (zh) 2017-04-03 2023-07-11 美商編碼製藥公司 組織選擇性轉基因表現
US10898585B2 (en) 2017-04-14 2021-01-26 Ptc Therapeutics .Inc. Gene therapy for AADC deficiency
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN119491003A (zh) 2017-05-05 2025-02-21 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
JP7327803B2 (ja) 2017-05-09 2023-08-16 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（ａｌｓ）を処置する方法
CA3066077A1 (en) 2017-06-07 2018-12-13 Adrx, Inc. Tau aggregation inhibitors
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
SG11201912631PA (en) 2017-07-06 2020-01-30 Univ Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
PT3684423T (pt) 2017-09-20 2023-06-09 4D Molecular Therapeutics Inc Cápsides variantes de vírus adeno-associado e métodos de utilização das mesmas
AU2018338188B2 (en) 2017-09-22 2025-02-20 University Of Massachusetts SOD1 dual expression vectors and uses thereof
KR20250161673A (ko) 2017-09-22 2025-11-17 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
WO2019060726A1 (en) * 2017-09-22 2019-03-28 Genzyme Corporation Variant rnai
AU2018338728B2 (en) * 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
CA3077057A1 (en) * 2017-10-02 2019-04-11 Research Institute At Nationwide Children's Hospital Mirna detargeting system for tissue specific interference
MX2020003965A (es) 2017-10-03 2020-10-05 Prevail Therapeutics Inc Terapias genicas para los trastornos lisosomales.
MX2020004005A (es) * 2017-10-03 2020-10-05 Prevail Therapeutics Inc Terapias genicas para los trastornos lisosomales.
TWI823868B (zh) 2017-10-11 2023-12-01 美商生物維瑞提夫美國公司 誘導補體活性之方法
WO2019075320A1 (en) * 2017-10-12 2019-04-18 The Trustees Of Columbia University In The City Of New York INCARN OF SLC2A1 AS BIOLOGICAL SUBSTANCE, TREATMENTS AND ASSOCIATED METHODS
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
AU2018352236B2 (en) 2017-10-16 2025-04-10 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
BR112020010674A2 (pt) 2017-11-27 2020-11-10 4D Molecular Therapeutics Inc. capsídeos de variante de vírus adeno-associado e uso para inibir a angiogênese
EP3717652A4 (en) 2017-11-30 2021-11-24 The Trustees of the University of Pennsylvania GENE THERAPY FOR MUCOPOLYSACCHARIDOSIS TYPE IIIB
BR112020010735A2 (pt) 2017-11-30 2020-11-10 The Trustees Of The University Of Pennsylvania terapia genética para mucopolissacaridose iii a
MA50942A (fr) 2017-12-01 2020-10-07 Encoded Therapeutics Inc Protéines de liaison à l'adn modifiées
US12194108B2 (en) 2018-03-23 2025-01-14 University Of Massachusetts Gene therapeutics for treating bone disorders
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
MX2020010464A (es) 2018-04-03 2021-01-29 Vectores de virus que evitan anticuerpos.
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
WO2019200016A1 (en) 2018-04-10 2019-10-17 President And Fellows Of Harvard College Aav vectors encoding clarin-1 or gjb2 and uses thereof
US12460226B2 (en) 2018-04-16 2025-11-04 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
AU2019257755A1 (en) 2018-04-27 2020-11-12 Voyager Therapeutics, Inc. Methods for measuring the potency of AADC viral vectors
TW202015742A (zh) 2018-05-15 2020-05-01 美商航海家醫療公司 投遞腺相關病毒（ａａｖ）之組成物和方法
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
US12163129B2 (en) 2018-06-08 2024-12-10 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
EP3807404A1 (en) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
US12297241B2 (en) 2018-07-06 2025-05-13 The Regents Of The University Of California Method to engineer transplantable human tissues
JP2021530218A (ja) * 2018-07-11 2021-11-11 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッドThe Brigham and Women’s Hospital, Inc. 血液脳関門を超える薬剤の送達のための方法および組成物
AU2019310459A1 (en) 2018-07-24 2021-02-18 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
AU2019315445A1 (en) * 2018-07-31 2021-03-18 Cornell University Gene therapy methods to control organ function
WO2020028751A2 (en) 2018-08-03 2020-02-06 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
AR114540A1 (es) 2018-08-03 2020-09-16 Genzyme Corp VARIANTE DE ARNi CONTRA a-SINUCLEÍNA
US12508327B2 (en) 2018-08-30 2025-12-30 Rowan University Methods of treating or preventing amyotrophic lateral sclerosis
JP7624724B2 (ja) 2018-09-26 2025-01-31 カリフォルニア インスティテュート オブ テクノロジー 標的遺伝子療法のためのアデノ随伴ウイルス組成物
SG11202103151RA (en) 2018-09-28 2021-04-29 Voyager Therapeutics Inc Frataxin expression constructs having engineered promoters and methods of use thereof
AU2019354995B2 (en) 2018-10-02 2024-12-12 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
TW202035689A (zh) 2018-10-04 2020-10-01 美商航海家醫療公司 測量病毒載體粒子的效價及強度之方法
JP2022512621A (ja) 2018-10-05 2022-02-07 ボイジャー セラピューティクス インコーポレイテッド Ａａｖ産生タンパク質をコードする操作された核酸コンストラクト
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
TW202028468A (zh) 2018-10-15 2020-08-01 美商航海家醫療公司 用於桿狀病毒/Sf9系統中rAAV之大規模生產的表現載體
US12454701B2 (en) 2018-12-05 2025-10-28 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
JOP20210160A1 (ar) * 2018-12-21 2023-01-30 Univ Pennsylvania تركيبات لخفض التعبير عن الجين الناقل بشكل انتقائي لـ drg
MX2021008979A (es) 2019-01-28 2021-11-12 Cohbar Inc Peptidos terapeuticos.
US20220154211A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
SG11202108044YA (en) 2019-02-25 2021-09-29 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
AU2020233396B2 (en) 2019-03-04 2025-09-11 Duke University Compositions and methods for the diagnosis and treatment of retinopathies
SG11202110146VA (en) 2019-03-21 2021-10-28 Stridebio Inc Recombinant adeno-associated virus vectors
MX2021012184A (es) 2019-04-10 2022-01-24 Prevail Therapeutics Inc Terapias genicas para trastornos lisosomales.
CN113924313A (zh) * 2019-04-10 2022-01-11 马萨诸塞大学 因子h载体及其用途
WO2020210592A1 (en) * 2019-04-12 2020-10-15 University Of Massachusetts Recombinant aav gene therapy for ngyl1 deficiency
MX2021013268A (es) * 2019-04-29 2021-11-17 Univ Pennsylvania Nuevas capsides de aav y composiciones que las contienen.
WO2020223280A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
US20220243225A1 (en) 2019-04-29 2022-08-04 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
US20240124889A1 (en) 2019-05-07 2024-04-18 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
EP3976798B1 (en) 2019-05-29 2025-12-17 Encoded Therapeutics, Inc. Compositions and methods for selective gene regulation
CN121221803A (zh) * 2019-05-31 2025-12-30 巴塞罗那自治大学 胰岛素基因疗法
US20220307024A1 (en) * 2019-06-17 2022-09-29 Alnylam Pharmaceuticals, Inc. Delivery of oligonucleotides to the striatum
US11446084B2 (en) 2019-07-12 2022-09-20 Neuralink Corp. Laser drilling of pia mater
WO2021025995A1 (en) 2019-08-02 2021-02-11 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
US20220290182A1 (en) 2019-08-09 2022-09-15 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
GB2587319A (en) * 2019-08-12 2021-03-31 Healing Genes Llc Genetic construct
US20220364114A1 (en) 2019-08-26 2022-11-17 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CN115087738A (zh) * 2019-08-29 2022-09-20 小利兰·斯坦福大学托管委员会 用于穿越人血脑屏障的腺相关病毒载体
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
CN115279896A (zh) 2019-09-30 2022-11-01 比奥维拉迪维治疗股份有限公司 慢病毒载体配制品
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
WO2021126991A1 (en) * 2019-12-17 2021-06-24 Nikegen Limited Parvoviral vectors and methods of making and use thereof
KR20220127847A (ko) * 2020-01-10 2022-09-20 더 브리검 앤드 우먼즈 하스피털, 인크. 뇌암을 치료하기 위해 혈액-뇌 장벽을 통해 면역요법제를 전달하기 위한 방법 및 조성물
US20210261625A1 (en) 2020-01-29 2021-08-26 Genzyme Corporation Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof
WO2021177116A1 (ja) 2020-03-02 2021-09-10 国立大学法人群馬大学 ミクログリア選択的遺伝子発現ベクター
CA3173341A1 (en) 2020-03-26 2021-09-30 Diamentis Inc. Systems and methods for processing retinal signal data and identifying conditions
KR20230020394A (ko) 2020-04-15 2023-02-10 보이저 테라퓨틱스, 인크. Tau 결합 화합물
GB202005732D0 (en) * 2020-04-20 2020-06-03 Synpromics Ltd Regulatory nucleic acid sequences
JP2023524024A (ja) * 2020-04-28 2023-06-08 ファイザー・インコーポレイテッド アスパルトアシラーゼ（ａｓｐａ）をコードする修飾核酸及び遺伝子治療のためのベクター
IL298138A (en) 2020-05-12 2023-01-01 Univ Pennsylvania Compositions for drg-specific reduction of transgene expression
CA3182970A1 (en) 2020-05-13 2021-11-18 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
IL299333A (en) 2020-06-24 2023-02-01 Bioverativ Therapeutics Inc Methods for the purification of viral vectors
GB202010981D0 (en) 2020-07-16 2020-09-02 Ucl Business Ltd Gene therapy for neuromuscular and neuromotor disorders
EP4192518A4 (en) * 2020-08-05 2024-12-25 Hangzhou Exegenesis Bio Ltd. NUCLEIC ACID CONSTRUCTS AND THEIR USES FOR THE TREATMENT OF SPINAL MUSCULAR ATROPHY
EP4192514A1 (en) 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
CN116783298A (zh) * 2020-11-04 2023-09-19 贝林格尔.英格海姆国际有限公司 供治疗ild、pf-ild和ipf之用的病毒载体和核酸
US20240050593A1 (en) * 2020-12-18 2024-02-15 Duke University Compositions For and Methods of Enhancing Spinal Cord Tissue Regeneration
US20240141377A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022192708A1 (en) * 2021-03-11 2022-09-15 Temple University- Of The Commonwealth System Of Higher Education Noninvasive aav vectors for highly efficient gene delivery to the nervous system
US20240252679A1 (en) 2021-05-28 2024-08-01 Shanghai Regenelead Therapies Co., Ltd. Recombinant adeno-associated virus having variant capsid, and application thereof
US20250001008A1 (en) * 2021-07-01 2025-01-02 Bridgebio Gene Therapy Research, Inc. Gene therapy for galactosemia
EP4405396A2 (en) 2021-09-20 2024-07-31 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
AU2022366947A1 (en) 2021-10-12 2024-05-02 Bridgebio Gene Therapy Llc Methods and compositions for treating leukodystrophies
KR20240113624A (ko) 2021-11-02 2024-07-22 보이저 테라퓨틱스, 인크. Aav 캡시드 변이체 및 이의 용도
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
TW202325850A (zh) 2021-11-29 2023-07-01 大陸商上海瑞宏迪醫藥有限公司 Aadc、gdnf多核苷酸及其用於治療帕金森病
EP4469585A2 (en) 2022-01-25 2024-12-04 Voyager Therapeutics, Inc. Baculovirus expression system
CN119095531A (zh) 2022-02-09 2024-12-06 迪亚门蒂斯公司 用于视网膜刺激和/或收集视网膜信号数据的系统和方法
TW202342526A (zh) 2022-02-21 2023-11-01 大陸商上海瑞宏迪醫藥有限公司 Ｖｅｇｆ結合分子及其醫藥用途
CA3248481A1 (en) 2022-04-12 2023-10-19 Genzyme Corporation USE OF INTERLEUKIN 4 RECEPTOR-ASSOCIATED KINASE MODULATORS FOR GENE THERAPY
WO2023201274A1 (en) 2022-04-12 2023-10-19 Genzyme Corporation Use of an irak4 modulator for gene therapy
TW202405430A (zh) 2022-04-12 2024-02-01 美商健臻公司 對於基因療法藥劑的先天免疫原性之樹突細胞試驗
US20250269064A1 (en) * 2022-04-22 2025-08-28 The Children's Hospital Of Philadelphia Human ependyma-specific promoter and uses thereof
EP4522757A2 (en) 2022-05-13 2025-03-19 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
WO2023250388A1 (en) 2022-06-22 2023-12-28 Voyager Therapeutics, Inc. Tau binding compounds
WO2024027632A1 (en) * 2022-08-01 2024-02-08 Skyline Therapeutics (Shanghai) Co., Ltd. Novel plasmid backbone to reduce dna impurities in raav preparation
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
AU2023342086A1 (en) 2022-09-15 2025-03-13 Voyager Therapeutics, Inc. Tau binding compounds
GB2622629A (en) 2022-09-23 2024-03-27 Sania Rx Ltd Method
US20250197862A1 (en) 2023-12-15 2025-06-19 Genzyme Corporation ARTIFICIAL microRNAs TARGETING TAU
WO2025158385A1 (en) 2024-01-25 2025-07-31 Genzyme Corporation Pegylated il-2 for suppressing adaptive immune response to gene therapy
WO2025160429A1 (en) 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting snca
WO2025160434A1 (en) 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting huntington's disease
GB202406331D0 (en) 2024-05-07 2024-06-19 Sania Rx Ltd Capsid
WO2025235643A1 (en) 2024-05-08 2025-11-13 Genzyme Corporation Profiling of gene therapy agents

Family Cites Families (141)

* 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
US5043270A (en) 1989-03-31 1991-08-27 The Board Of Trustees Of The Leland Stanford Junior University Intronic overexpression vectors
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
JPH10501686A (ja) 1994-04-13 1998-02-17 ザ ロックフェラー ユニヴァーシティ 神経系の細胞へのｄｎａのａａｖ仲介送達
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
US6391607B1 (en) 1996-06-14 2002-05-21 Genentech, Inc. Human DNase I hyperactive variants
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
US6083716A (en) 1996-09-06 2000-07-04 The Trustees Of The University Of Pennsylvania Chimpanzee adenovirus vectors
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
US6475469B1 (en) 1996-11-21 2002-11-05 Applied Dental Sciences, Inc. Compositions for removing tooth stains
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
CA2304168A1 (en) 1997-09-19 1999-04-01 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
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
CA2335390A1 (en) 1998-07-22 2000-02-03 Immusol Incorporated Substantially complete ribozyme libraries
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
US6365394B1 (en) 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
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
US6821512B1 (en) 1999-12-03 2004-11-23 The Trustees Of The University Of Pennsylvania Compositions and methods for increasing packaging and yield of recombinant adenoviruses using multiple packaging signals
US7638120B2 (en) 2000-03-14 2009-12-29 Thomas Jefferson University High transgene expression of a pseudotyped adeno-associated virus type
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
EP1409748B1 (en) 2001-06-22 2011-10-26 The Trustees of The University of Pennsylvania Recombinant Adenoviruses comprising simian adenovirus proteins and uses thereof.
US20040136963A1 (en) 2001-06-22 2004-07-15 The Trustees Of The University Of Pennsylvania Simian adenovirus vectors and methods of use
EP1402043A1 (en) * 2001-07-03 2004-03-31 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods of administering vectors to synaptically connected neurons
EP1419245A4 (en) 2001-07-13 2006-04-05 Univ Iowa Res Found ADENO-ASSOCIATED PSEUDOTYPE VIRUSES AND USES THEREOF
US20030092161A1 (en) 2001-09-19 2003-05-15 The Trustees Of The University Of Pennsylvania Compositions and methods for production of recombinant viruses, and uses therefor
KR101014207B1 (ko) * 2001-11-13 2011-02-14 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 아데노-결합 바이러스 (ａａｖ) 서열을 검출 및/또는 확인하는 방법 및 그 방법에 의해 확인된 신규한 서열을 분리하는 방법
CA2852277C (en) 2001-11-21 2018-02-20 Guangping Gao Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use
CA2469785C (en) 2001-12-17 2014-04-22 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
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)
AU2003221733A1 (en) 2002-04-17 2003-11-03 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
ATE348153T1 (de) 2002-04-29 2007-01-15 Univ Pennsylvania Methode für die direkte gewinnung und amplifikation von integrierten viren aus zellulärer gewebe-dna
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
CA2514417A1 (en) 2003-01-28 2004-08-12 Cellectis Custom-made meganuclease and use thereof
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
PL1633767T3 (pl) 2003-06-02 2019-07-31 University Of Massachusetts Sposoby i kompozycje do kontrolowania wydajności wyciszania rna
EP2298926A1 (en) 2003-09-30 2011-03-23 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
WO2005087926A2 (en) 2004-03-05 2005-09-22 Benitec, Inc. Multiple promoter expression cassettes for simultaneous delivery of rnai agents
US7498316B2 (en) * 2004-04-06 2009-03-03 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
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
CA2596711C (en) 2005-02-03 2015-12-15 Benitec, Inc. Rnai expression constructs
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
CN101203613B (zh) 2005-04-07 2012-12-12 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
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
AU2006242371A1 (en) * 2005-04-29 2006-11-09 The University Of North Carolina At Chapel Hill Methods and compositions for regulated expression of nucleic acid at post-transcriptional level
KR20130114758A (ko) * 2005-05-27 2013-10-17 오스페달레 산 라파엘 에스.알.엘. Ｍｉ-ｒｎａ를 포함하는 유전자 벡터
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
US7977314B2 (en) * 2005-12-02 2011-07-12 Amorfix Life Sciences Limited Methods and compositions to treat and detect misfolded-SOD1 mediated diseases
US9150882B2 (en) 2006-01-31 2015-10-06 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
HUE041928T2 (hu) * 2006-02-08 2019-06-28 Genzyme Corp Génterápia A-típusú Niemann-Pick-betegségre
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
US8524679B2 (en) 2006-11-08 2013-09-03 Veritas Bio, Llc In vivo delivery of double stranded RNA to a target cell
EP2114460B1 (en) 2007-02-15 2014-07-16 National Jewish Health Compositions for the disruption of biofilms
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
EP2140004B1 (en) 2007-04-12 2022-01-12 The Provost, Fellows, Foundation Scholars, & the other members of Board, of the College of the Holy & Undiv. Trinity of Queen Elizabeth near Dublin Genetic suppression and replacement
US20090131355A1 (en) 2007-05-23 2009-05-21 Adrian Ion Bot Multicistronic vectors and methods for their design
CA2687336C (en) 2007-05-23 2014-12-23 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
EP3252161B1 (en) 2007-06-06 2021-11-17 Genzyme Corporation Gene therapy for lysosomal storage diseases
EP2019143A1 (en) * 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
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
WO2009146178A1 (en) * 2008-04-15 2009-12-03 President And Fellows Of Harvard College Angiogenin and amyotrophic lateral sclerosis
CA2722238C (en) 2008-04-22 2017-11-28 Life Sciences Research Partners Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2009149182A1 (en) 2008-06-04 2009-12-10 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
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
PL4342992T3 (pl) * 2009-05-02 2025-09-22 Genzyme Corporation Terapia genowa dla zaburzeń neurodegeneracyjnych
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
CN102695526A (zh) 2009-11-09 2012-09-26 吉恩波多治疗股份公司 用于神经元特异性的体内连续dopa合成的新型病毒载体构建体
JP5697042B2 (ja) 2009-11-19 2015-04-08 国立大学法人 岡山大学 遺伝子発現を上昇させるシステム及び該システムを保持したベクター
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
JP2013517798A (ja) 2010-01-28 2013-05-20 ザ チルドレンズ ホスピタル オブ フィラデルフィア ウイルスベクター精製における拡張可能な製造プラットフォームおよび遺伝子治療における使用のための高純度ウイルスベクター
CA2791974A1 (en) 2010-03-01 2011-09-09 The Children's Hospital Of Philadelphia Nucleic acids for targeting multiple regions of the hcv genome
EP2561075B1 (en) 2010-04-23 2018-06-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
WO2011133890A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
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
SG10201601110VA (en) 2011-02-17 2016-03-30 Univ Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
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
WO2013055865A1 (en) 2011-10-11 2013-04-18 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
BR112014020325A2 (pt) 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
US20150182637A1 (en) 2012-06-21 2015-07-02 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
MX2015009122A (es) 2013-01-14 2015-10-05 Recombinetics Inc Ganado sin cuernos.
AU2014244167A1 (en) 2013-03-13 2015-10-08 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
CN105163764B (zh) 2013-03-15 2019-11-12 北卡罗来纳-查佩尔山大学 双重聚糖结合aav载体的方法和组合物
DK2986635T3 (en) 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
BR112015031608A2 (pt) 2013-06-17 2017-08-22 Massachusetts Inst Technology Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos
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
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
LT3039146T (lt) 2013-08-27 2020-07-27 Research Institute at Nationwide Children`s Hospital Produktai ir būdai, skirti amiotropinės lateralinės sklerozės gydymui
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
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
JP6741591B2 (ja) 2014-05-02 2020-08-19 ジェンザイム・コーポレーション 網膜およびｃｎｓ遺伝子治療用ａａｖベクター
RU2711147C2 (ru) 2014-05-20 2020-01-15 Юниверсити Оф Айова Рисерч Фаундейшн Терапевтические соединения для лечения болезни хантингтона
AU2015335923B2 (en) 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
WO2017023724A1 (en) 2015-07-31 2017-02-09 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019052150A (ja) * 2010-04-23 2019-04-04 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

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