JP2017533715A5 - - Google Patents

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JP2017533715A5
JP2017533715A5 JP2017525592A JP2017525592A JP2017533715A5 JP 2017533715 A5 JP2017533715 A5 JP 2017533715A5 JP 2017525592 A JP2017525592 A JP 2017525592A JP 2017525592 A JP2017525592 A JP 2017525592A JP 2017533715 A5 JP2017533715 A5 JP 2017533715A5
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JP6863891B2 (ja
JP2017533715A (ja
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Priority claimed from PCT/US2015/060564 external-priority patent/WO2016077689A1/en
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Priority to JP2021062812A priority Critical patent/JP7256223B2/ja
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Priority to JP2023055041A priority patent/JP7469538B2/ja
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JP2017525592A 2014-11-14 2015-11-13 調節性ポリヌクレオチド Active JP6863891B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2021062812A JP7256223B2 (ja) 2014-11-14 2021-04-01 調節性ポリヌクレオチド
JP2023055041A JP7469538B2 (ja) 2014-11-14 2023-03-30 調節性ポリヌクレオチド

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201462079590P 2014-11-14 2014-11-14
US62/079,590 2014-11-14
US201562212004P 2015-08-31 2015-08-31
US62/212,004 2015-08-31
US201562234477P 2015-09-29 2015-09-29
US62/234,477 2015-09-29
PCT/US2015/060564 WO2016077689A1 (en) 2014-11-14 2015-11-13 Modulatory polynucleotides

Related Child Applications (1)

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JP2021062812A Division JP7256223B2 (ja) 2014-11-14 2021-04-01 調節性ポリヌクレオチド

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JP2017533715A JP2017533715A (ja) 2017-11-16
JP2017533715A5 true JP2017533715A5 (enExample) 2018-11-15
JP6863891B2 JP6863891B2 (ja) 2021-04-21

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JP2017525592A Active JP6863891B2 (ja) 2014-11-14 2015-11-13 調節性ポリヌクレオチド
JP2021062812A Active JP7256223B2 (ja) 2014-11-14 2021-04-01 調節性ポリヌクレオチド
JP2023055041A Active JP7469538B2 (ja) 2014-11-14 2023-03-30 調節性ポリヌクレオチド

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JP2023055041A Active JP7469538B2 (ja) 2014-11-14 2023-03-30 調節性ポリヌクレオチド

Country Status (16)

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US (4) US10570395B2 (enExample)
EP (2) EP3218386B1 (enExample)
JP (3) JP6863891B2 (enExample)
KR (2) KR102584655B1 (enExample)
CN (5) CN119876138A (enExample)
AU (3) AU2015346164B2 (enExample)
BR (1) BR112017010088A2 (enExample)
CA (1) CA2975583A1 (enExample)
DK (1) DK3218386T3 (enExample)
ES (1) ES2878451T3 (enExample)
IL (2) IL284949B2 (enExample)
MX (3) MX2017006217A (enExample)
RU (2) RU2719192C2 (enExample)
SG (2) SG11201703419UA (enExample)
WO (1) WO2016077689A1 (enExample)
ZA (1) ZA202004557B (enExample)

Families Citing this family (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
CA2975583A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
IL262784B2 (en) * 2016-05-18 2023-10-01 Voyager Therapeutics Inc modulatory polynucleotides
WO2018204786A1 (en) * 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
EP3619310A4 (en) * 2017-05-05 2021-01-27 Voyager Therapeutics, Inc. Modulatory polynucleotides
MX2019013172A (es) * 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
US20200377887A1 (en) * 2017-09-22 2020-12-03 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
US11434502B2 (en) * 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
US12194108B2 (en) 2018-03-23 2025-01-14 University Of Massachusetts Gene therapeutics for treating bone disorders
EP3822350A4 (en) * 2018-05-31 2022-06-22 Korea University Research and Business Foundation Rna interference-inducing nucleic acid inhibiting noncanonical targets of micro rna, and use for same
EP3807404A1 (en) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
WO2020010035A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Cannula system
JP7565218B2 (ja) * 2018-07-02 2024-10-10 ボイジャー セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症および脊髄に関連する障害の治療
JP2021530548A (ja) 2018-07-24 2021-11-11 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics, Inc. 遺伝子治療製剤を生産するための系および方法
WO2020023767A1 (en) * 2018-07-26 2020-01-30 Joslin Diabetes Center Targeting micro-rnas for exosomal delivery or cellular retention
SG11202103151RA (en) * 2018-09-28 2021-04-29 Voyager Therapeutics Inc Frataxin expression constructs having engineered promoters and methods of use thereof
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
CN113166731A (zh) 2018-10-05 2021-07-23 沃雅戈治疗公司 编码aav生产蛋白的工程化核酸构建体
US20210371470A1 (en) * 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3867389A1 (en) 2018-10-15 2021-08-25 Voyager Therapeutics, Inc. Expression vectors for large-scale production of raav in the baculovirus/sf9 system
CA3125770A1 (en) 2019-01-18 2020-07-23 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
EP3917566A4 (en) 2019-01-31 2022-10-26 Oregon Health & Science University METHODS OF USING TRANSCRIPTION-DEPENDENT DIRECTED EVOLUTION OF AAV CAPSIDS
SG11202111195VA (en) * 2019-04-12 2021-11-29 Encoded Therapeutics Inc Compositions and methods for administration of therapeutics
WO2020223274A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
WO2021000928A1 (zh) * 2019-07-04 2021-01-07 中美瑞康核酸技术(南通)研究院有限公司 激活atoh1基因的寡聚核酸分子及其应用
US20220290182A1 (en) 2019-08-09 2022-09-15 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
TW202122582A (zh) 2019-08-26 2021-06-16 美商航海家醫療公司 病毒蛋白之控制表現
CN111100873B (zh) * 2019-11-22 2020-12-22 昆明学院 激活rna调控启动子克服转基因沉默效应的方法
EP4192514A1 (en) 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
WO2022174000A2 (en) 2021-02-12 2022-08-18 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
EP4301768A2 (en) 2021-03-03 2024-01-10 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022225353A1 (ko) * 2021-04-21 2022-10-27 이화여자대학교 산학협력단 타겟 유전자 조절을 위한 다기능성 핵산 구조체 및 이의 용도
EP4430191A2 (en) * 2021-11-08 2024-09-18 University Of Massachusetts Oligonucleotides for sod1 modulation
WO2023097268A2 (en) * 2021-11-23 2023-06-01 Mantra Bio, Inc. Extracellular vesicles comprising non-naturally occurring modular rna hairpins and uses thereof
EP4499833A4 (en) * 2022-03-30 2025-11-05 Mirimus Inc COMPOSITIONS AND METHODS FOR GENERING A NEW AMIARN
JP2025522433A (ja) 2022-06-15 2025-07-15 アローヘッド ファーマシューティカルズ インコーポレイテッド スーパーオキシドジスムターゼ1(SOD1)の発現を阻害するためのRNAi剤、その組成物、及び使用の方法
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
AU2023392154A1 (en) * 2022-12-14 2025-06-26 King's College London Compositions and methods for treating neurological diseases
KR20250129743A (ko) 2022-12-29 2025-08-29 보이저 테라퓨틱스, 인크. Mapt를 조절하기 위한 조성물 및 방법
WO2024226761A2 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
CN120202298A (zh) * 2023-08-29 2025-06-24 石药集团中奇制药技术(石家庄)有限公司 一种抑制sod1基因表达的dsRNA分子及其应用
WO2025122644A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2025126153A2 (en) * 2023-12-14 2025-06-19 Aviadobio Ltd. Compositions and methods for treating sod1-mediated neurological diseases

Family Cites Families (520)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2640638B1 (fr) 1988-12-20 1991-02-15 Commissariat Energie Atomique Bioreacteur et dispositif pour la culture de cellules animales
WO1991018088A1 (en) 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5693531A (en) 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
ATE386131T1 (de) 1994-04-13 2008-03-15 Univ Rockefeller Aav-vermittelte überbringung von dna in zellen des nervensystems
US20020159979A1 (en) 1994-06-06 2002-10-31 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
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
US5625048A (en) 1994-11-10 1997-04-29 The Regents Of The University Of California Modified green fluorescent proteins
EP0796339A1 (en) 1994-12-06 1997-09-24 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US5652224A (en) 1995-02-24 1997-07-29 The Trustees Of The University Of Pennsylvania Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism
US5741657A (en) 1995-03-20 1998-04-21 The Regents Of The University Of California Fluorogenic substrates for β-lactamase and methods of use
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US5688676A (en) 1995-06-07 1997-11-18 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6676935B2 (en) 1995-06-27 2004-01-13 Cell Genesys, Inc. Tissue specific adenoviral vectors
US6197293B1 (en) 1997-03-03 2001-03-06 Calydon, Inc. Adenovirus vectors specific for cells expressing androgen receptor and methods of use thereof
CA2230655C (en) 1995-08-30 2008-06-17 Genzyme Corporation Chromatographic purification of adenovirus and aav
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
US5846528A (en) 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US5952221A (en) 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
US7026468B2 (en) 1996-07-19 2006-04-11 Valentis, Inc. Process and equipment for plasmid purification
US6083716A (en) 1996-09-06 2000-07-04 The Trustees Of The University Of Pennsylvania Chimpanzee adenovirus vectors
IL128779A0 (en) 1996-09-06 2000-01-31 Univ Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
AU722375B2 (en) 1996-09-06 2000-08-03 Trustees Of The University Of Pennsylvania, The Methods using cre-lox for production of recombinant adeno-associated viruses
US5866552A (en) 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
US20020037867A1 (en) 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
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
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
US7732129B1 (en) 1998-12-01 2010-06-08 Crucell Holland B.V. Method for the production and purification of adenoviral vectors
BR9713368A (pt) 1996-11-20 2001-09-18 Introgen Therapeutics Inc Processo melhorado para a produção e a purificação de vetores adenovirais
CA2270285A1 (en) 1996-12-18 1998-06-25 Targeted Genetics Corporation Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6710036B2 (en) 1997-07-25 2004-03-23 Avigen, Inc. Induction of immune response to antigens expressed by recombinant adeno-associated virus
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
CA2304131A1 (en) 1997-09-19 1999-04-01 James M. Wilson Method for gene transfer using bcl2 and compositions useful therein
WO1999014354A1 (en) 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
AU9397098A (en) 1997-09-19 1999-04-12 Trustees Of The University Of Pennsylvania, The Methods and cell line useful for production of recombinant adeno-associated viruses
US6642051B1 (en) 1997-10-21 2003-11-04 Targeted Genetics Corporation Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors
IT1297074B1 (it) 1997-11-21 1999-08-03 Angeletti P Ist Richerche Bio Forme ormone-dipendenti delle proteine rep del virus adeno-associato (aav-2), sequenze di dna codificanti per esse, vettori che le
AU759573B2 (en) 1997-12-23 2003-04-17 Crucell Holland B.V. Adeno-associated virus and adenovirus chimeric recombinant viruses useful for the integration of foreign genetic information into the chromosomal DNA of target cells
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
AU2882899A (en) 1998-02-26 1999-09-15 Trustees Of The University Of Pennsylvania, The Stable protection from dystrophic sarcolemmal degeneration and restoration of the sarcoglycan complex
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
US6521426B1 (en) 1998-04-08 2003-02-18 Istituto Di Ricerche Di Biologia Molecolare P. Angeletti S.P.A. Preparation of recombinant adenovirus carrying a rep gene of adeno-associated virus
FR2778413B1 (fr) 1998-05-07 2000-08-04 Immunotech Sa Nouveaux reactifs et methode de lyse des erythrocytes
WO1999058700A1 (en) 1998-05-11 1999-11-18 Ariad Gene Therapeutics, Inc. Multiviral compositions and uses thereof
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
EP1849872A1 (en) 1998-05-20 2007-10-31 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
WO1999061643A1 (en) 1998-05-27 1999-12-02 University Of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient
JP2002516345A (ja) 1998-05-27 2002-06-04 セル ジェネシス インコーポレイテッド 第viii因子活性のアデノ随伴ウイルスベクター媒介性発現
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
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
GB2338236B (en) 1998-06-13 2003-04-09 Aea Technology Plc Microbiological cell processing
US6900049B2 (en) 1998-09-10 2005-05-31 Cell Genesys, Inc. Adenovirus vectors containing cell status-specific response elements and methods of use thereof
US6416992B1 (en) 1998-10-13 2002-07-09 Avigen, Inc. Compositions and methods for producing recombinant adeno-associated virus
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
AU764130B2 (en) 1998-10-27 2003-08-14 Crucell Holland B.V. Improved AAV vector production
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6689600B1 (en) 1998-11-16 2004-02-10 Introgen Therapeutics, Inc. Formulation of adenovirus for gene therapy
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
US6225113B1 (en) 1998-12-04 2001-05-01 Genvec, Inc. Use of trans-activation and cis-activation to modulate the persistence of expression of a transgene in an at least E4-deficient adenovirus
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
DE19905501B4 (de) 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
US6509150B1 (en) 1999-03-05 2003-01-21 Universite De Nantes Compositions and methods for recombinant Adeno-Associated Virus production
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
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
CA2368194A1 (en) 1999-04-30 2000-11-09 University Of Florida Adeno-associated virus-delivered ribozyme compositions and methods of use
CA2375098A1 (en) 1999-06-02 2000-12-14 Trustees Of The University Of Pennsylvania Compositions and methods useful for production of recombinant viruses which require helper viruses
JP4969002B2 (ja) 1999-06-08 2012-07-04 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 rAAV形質導入を増加するための化合物および方法
AU781478B2 (en) 1999-08-20 2005-05-26 Johns Hopkins University School Of Medicine, The Methods and compositions for the construction and use of fusion libraries
WO2001023001A2 (en) 1999-09-29 2001-04-05 The Trustees Of The University Of Pennsylvania Rapid peg-modification
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
EP1224313A1 (en) 1999-10-07 2002-07-24 University of Iowa Research Foundation Adeno-associated viruses and uses thereof
US7241447B1 (en) 1999-10-07 2007-07-10 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
WO2001032711A2 (en) 1999-10-21 2001-05-10 Board Of Trustees Of The University Of Arkansas Adeno-associated virus aav rep78 major regulatory protein, mutants thereof and uses thereof
WO2001036623A2 (en) 1999-11-05 2001-05-25 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
AU1775901A (en) 1999-11-17 2001-05-30 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
US20010049144A1 (en) 1999-12-10 2001-12-06 Victor Rivera Methods for high level expression of genes in primates
AU4565401A (en) 2000-03-14 2001-09-24 Thomas Jefferson University Production of chimeric capsid vectors
US7638120B2 (en) 2000-03-14 2009-12-29 Thomas Jefferson University High transgene expression of a pseudotyped adeno-associated virus type
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
US7048920B2 (en) 2000-03-24 2006-05-23 Cell Genesys, Inc. Recombinant oncolytic adenovirus for human melanoma
PT1309726E (pt) 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
GB0009887D0 (en) 2000-04-20 2000-06-07 Btg Int Ltd Cytotoxic agents
AU2001257611A1 (en) 2000-04-28 2001-11-12 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US20030013189A1 (en) 2000-04-28 2003-01-16 Wilson James M. Compositions and methods useful for non-invasive delivery of therapeutic molecules to the bloodstream
AU6808001A (en) 2000-05-23 2001-12-03 Neurologix Inc Glutamic acid decarboxylase (gad) based delivery systems
AU2001268373A1 (en) 2000-06-13 2001-12-24 The Children's Hospital Of Philadelphia Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus
US7045299B2 (en) 2000-07-18 2006-05-16 Takeda Pharmaceutical Company Limited Physiologically active peptide and use thereof
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
AU2001294096A1 (en) 2000-08-17 2002-02-25 Uichi Ikeda Adeno-associated virus-mediated delivery of angiogenic factors
DE10066104A1 (de) 2000-09-08 2003-01-09 Medigene Ag Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung
FR2813891B1 (fr) 2000-09-14 2005-01-14 Immunotech Sa Reactif multifonctionnel pour erythrocytes mettant en jeu des carbamates et applications
GB0024550D0 (enExample) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
JP2002153278A (ja) 2000-11-22 2002-05-28 Hisamitsu Pharmaceut Co Inc ウイルスベクターの製造に用いられる細胞、その製法およびその細胞を用いたウイルスベクターの製造方法
WO2002070719A2 (en) 2001-01-19 2002-09-12 Trustees Of The University Of Pennsylvania Regulatable gene expression system
FR2821624B1 (fr) 2001-03-01 2004-01-02 Sod Conseils Rech Applic Nouveau polynucleotide utilisable pour moduler la proliferation des cellules cancereuses
US7588757B2 (en) 2001-03-14 2009-09-15 Genzyme Corporation Methods of treating Parkinson's disease using recombinant adeno-associated virus virions
US20030147853A1 (en) 2001-03-14 2003-08-07 Mcclelland Alan Recombinant adeno-associated virus-mediated gene transfer via retroductal infusion of virions
CA2450470C (en) 2001-06-22 2012-08-28 The Trustees Of The University Of Pennsylvania Method for rapid screening of bacterial transformants and novel simian adenovirus proteins
US20040136963A1 (en) 2001-06-22 2004-07-15 The Trustees Of The University Of Pennsylvania Simian adenovirus vectors and methods of use
EP1900815B1 (en) 2001-07-12 2016-09-07 University of Massachusetts In vivo production of small interfering RNAs that mediate gene silencing
CA2921821A1 (en) 2001-07-12 2003-01-23 University Of Massachusetts In vivo production of small interfering rnas that mediate gene silencing
US8241622B2 (en) 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
EP1279740A1 (en) 2001-07-26 2003-01-29 Vrije Universiteit Brussel Recombinant vector derived from adeno-associated virus for gene therapy
AU2002349877B2 (en) 2001-08-08 2007-10-11 The Trustees Of The University Of Pennsylvania Method for purification of viral vectors having proteins which bind sialic acid
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
EP2428569B1 (en) 2001-09-28 2018-05-23 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Microrna molecules
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
CA2467959C (en) 2001-11-09 2009-03-10 Robert M. Kotin Production of adeno-associated virus in insect cells
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
EP1944043A1 (en) 2001-11-21 2008-07-16 The Trustees of the University of Pennsylvania Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use
BR0214350A (pt) 2001-11-21 2005-05-10 Univ Pennsylvania Sequências de ácido nucleico e aminoácido de adenovìrus de sìmio, vetores contendo as mesmas e métodos de uso
EP1453537A1 (en) 2001-12-12 2004-09-08 FH Faulding & Co. Limited Composition for viral preservation
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
JP4810062B2 (ja) 2001-12-17 2011-11-09 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 アデノ随伴ウイルス(aav)血清型8の配列
AU2002359786A1 (en) 2001-12-19 2003-07-09 Hiroaki Mizukami Adeno-associated virus-mediated delivery of gdnf to skeletal muscles
WO2003062462A2 (en) 2002-01-16 2003-07-31 Dynal Biotech Asa Method for isolating nucleic acids and protein from a single sample
EP2348119B1 (en) 2002-02-01 2017-04-26 Oxford BioMedica (UK) Limited Multicistronic lentiviral vector
US20030180756A1 (en) 2002-03-21 2003-09-25 Yang Shi Compositions and methods for suppressing eukaryotic gene expression
GB0208390D0 (en) 2002-04-11 2002-05-22 Univ London Adeno-associated virus producer system
US20030198620A1 (en) 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
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.
MXPA04010603A (es) 2002-04-30 2004-12-13 Oncolytics Biotech Inc Metodos mejorados de purificacion viral.
NZ561656A (en) 2002-05-01 2009-03-31 Univ Florida Improved rAAV expression systems for genetic modification of specific capsid proteins
CA2524569C (en) 2002-05-03 2013-10-22 Duke University A method of regulating gene expression
WO2003097797A2 (en) 2002-05-14 2003-11-27 Merck & Co., Inc. Methods of adenovirus purification
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
WO2003104413A2 (en) 2002-06-05 2003-12-18 University Of Florida Production of pseudotyped recombinant aav virions
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
WO2004020605A2 (en) 2002-08-29 2004-03-11 The Board Of Trustees Of The Leland Stanford Junior University Circular nucleic acid vectors, and methods for making and using the same
WO2004027030A2 (en) 2002-09-18 2004-04-01 Isis Pharmaceuticals, Inc. Efficient reduction of target rna’s by single- and double-stranded oligomeric compounds
EP1418185A1 (en) 2002-11-11 2004-05-12 Aventis Pharma Deutschland GmbH Use of EDG2 receptor in an animal model of heart failure
US7169612B2 (en) 2002-11-11 2007-01-30 Sanofi-Aventis Deutschland Gmbh Use of EDG2 receptor in an animal model of heart failure
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US7618948B2 (en) 2002-11-26 2009-11-17 Medtronic, Inc. Devices, systems and methods for improving and/or cognitive function through brain delivery of siRNA
US20080318210A1 (en) 2003-08-27 2008-12-25 Rosetta Genomics Bioinformatically detectable group of novel regulatory viral and viral associated oligonucleotides and uses thereof
WO2005017127A2 (en) 2003-02-21 2005-02-24 The Penn State Research Foundation Rna interference compositions and methods
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
WO2004083441A2 (en) 2003-03-19 2004-09-30 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Random peptide library displayed on aav vectors
WO2004108922A2 (en) 2003-04-25 2004-12-16 The Trustees Of The University Of Pennsylvania Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver
WO2004101787A1 (ja) 2003-05-14 2004-11-25 Japan Science And Technology Agency ハンチンチン遺伝子の発現抑制
US8927269B2 (en) 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
PT1625210E (pt) 2003-05-21 2011-03-15 Genzyme Corp Métodos para produzir preparações de vírions de aav recombinantes substancialmente livres de capsídeos vazios
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
JP4888876B2 (ja) 2003-06-13 2012-02-29 田平 武 アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター
EP2657248B1 (en) 2003-06-19 2017-03-22 Genzyme Corporation AAV virions with decreased immunoreactivity and uses therefor
US7291498B2 (en) 2003-06-20 2007-11-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
WO2005001103A2 (en) 2003-06-20 2005-01-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
WO2005012537A2 (en) 2003-07-25 2005-02-10 Genvec, Inc. Adenoviral vector-based vaccines
CA2533701A1 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
EP3760234B1 (en) 2003-09-12 2023-11-01 University of Massachusetts Rna interference for the treatment of gain-of-function disorders
US20050064489A1 (en) 2003-09-24 2005-03-24 Zhang Fang Liang Engineered U6 and H1 promoters
HUE035567T2 (en) 2003-09-30 2018-05-28 Univ Pennsylvania Adeno-associated virus (AAV) clusters, sequences, vectors and applications containing them
US7962316B2 (en) 2003-10-27 2011-06-14 Merck Sharp & Dohme Corp. Method of designing siRNAs for gene silencing
NZ550284A (en) 2004-03-05 2009-04-30 Benitec Ltd Multiple promoter expression cassettes for simultaneous delivery of RNAi agents
WO2006078279A2 (en) 2004-04-28 2006-07-27 The Trustees Of The University Of Pennsylvania Sequential delivery of immunogenic molecules via adenovirus and adeno-associated virus-mediated administrations
DK3290513T3 (da) 2004-06-01 2022-12-12 Genzyme Corp Sammensætninger og fremgangsmåder til forhindring af aav-vektoraggregering
JP4838255B2 (ja) 2004-10-05 2011-12-14 ジェンザイム・コーポレーション 階段状カニューレ
US7901921B2 (en) 2004-10-22 2011-03-08 Oncolytics Biotech Inc. Viral purification methods
AU2005307737C1 (en) 2004-11-18 2013-08-29 The Board Of Trustees Of The University Of Illinois Multicistronic siRNA constructs to inhibit tumors
CN1286981C (zh) 2004-11-30 2006-11-29 华中科技大学同济医学院附属同济医院 表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法
US20060229268A1 (en) 2004-12-16 2006-10-12 Alsgen, Llc Small interference RNA (siRNA) molecules for modulating superoxide dismutase (SOD)
AU2006210443B2 (en) 2005-02-03 2011-01-27 Benitec, Inc. RNAi expression constructs
US8614101B2 (en) 2008-05-20 2013-12-24 Rapid Pathogen Screening, Inc. In situ lysis of cells in lateral flow immunoassays
US7625570B1 (en) 2005-03-10 2009-12-01 The Regents Of The University Of California Methods for purifying adeno-associated virus
WO2006130201A1 (en) 2005-03-14 2006-12-07 Board Of Regents, The University Of Texas System Antigene oligomers inhibit transcription
WO2006102072A2 (en) 2005-03-23 2006-09-28 The Trustees Of The University Of Pennsylvania Use of a pa131 polypeptide in treatment of atherosclerosis
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
PL2420256T3 (pl) 2005-05-02 2016-12-30 Terapia genowa dla zaburzeń neurometabolicznych
EP2239328A3 (en) 2005-08-18 2011-01-05 Alnylam Pharmaceuticals Inc. Methods and compositions for treating neurological disease
CA2619882C (en) 2005-08-23 2015-05-26 The Regents Of The University Of California Reflux resistant cannula and system for chronic delivery of therapeutic agents using convection-enhanced delivery
EP1857552A1 (en) 2006-05-20 2007-11-21 Cargill Incorporated Thermostable xylose isomerase enzyme
PT3272872T (pt) 2005-10-20 2020-06-26 Uniqure Ip Bv Vetores aav melhorados produzidos em células de insetos
RU2448974C2 (ru) 2005-11-01 2012-04-27 Элнилэм Фармасьютикалз, Инк. РНКи-ИНГИБИРОВАНИЕ РЕПЛИКАЦИИ ВИРУСА ГРИППА
JP5066095B2 (ja) 2005-11-17 2012-11-07 ボード・オブ・リージエンツ,ザ・ユニバーシテイ・オブ・テキサス・システム 染色体dnaに標的化されるオリゴマーによる遺伝子発現の調節
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
CA3024953A1 (en) * 2006-04-03 2007-10-11 Roche Innovation Center Copenhagen A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
DK2012822T3 (da) 2006-04-28 2010-05-25 Univ Pennsylvania Modificeret adenovirushexonprotein og anvendelser deraf
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
EP2016174A2 (en) 2006-04-28 2009-01-21 The Trustees of the University of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
US20080003565A1 (en) 2006-05-02 2008-01-03 Government Of The Us, As Represented By The Secretary, Department Of Health And Human Services Viral nucleic acid microarray and method of use
JP5551432B2 (ja) 2006-05-25 2014-07-16 サンガモ バイオサイエンシーズ, インコーポレイテッド 遺伝子不活性化のための方法と組成物
AU2007261806B2 (en) 2006-06-21 2013-08-15 Uniqure Ip B.V. Vectors with modified initiation codon for the translation of AAV-Rep78 useful for production of AAV in insect cells
AU2007275010B2 (en) 2006-07-21 2013-06-20 California Institute Of Technology Targeted gene delivery for dendritic cell vaccination
ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
EP3146982B1 (en) 2006-10-03 2019-08-21 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
US8895309B2 (en) 2006-11-29 2014-11-25 Nationwide Children's Hospital Myostatin inhibition for enhancing muscle and/or improving muscle function
WO2008067398A2 (en) 2006-11-29 2008-06-05 University Of Iowa Research Foundation Alternative export pathways for vector expressed rna interference
US8071752B2 (en) 2007-01-29 2011-12-06 City Of Hope Multi-targeting short interfering RNAs
WO2008097503A2 (en) 2007-02-02 2008-08-14 Biogen Idec Ma Inc. Use of semaphorin 6a for promoting myelination and oligodendrocyte differentiation
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
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
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
WO2008128251A1 (en) 2007-04-17 2008-10-23 The Children's Hospital Of Philadelphia Humanized viral vectors and methods of use thereof
WO2008134646A2 (en) 2007-04-26 2008-11-06 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
WO2008145400A2 (en) 2007-05-31 2008-12-04 Medigene Ag Mutated structural protein of a parvovirus
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
EP2530152B1 (en) 2007-05-31 2017-12-27 University of Iowa Research Foundation Reduction of off-target RNA interference toxicity
JP5480132B2 (ja) 2007-06-15 2014-04-23 ジ・オハイオ・ステイト・ユニバーシティ・リサーチ・ファウンデイション Drosha媒介性のマイクロrnaプロセッシングを標的するための癌原性all−1融合タンパク質
US8841437B2 (en) 2008-06-20 2014-09-23 The Board Of Trustees Of The Leland Stanford Junior University Precursor miRNA loop-modulated target regulation
RU2491344C2 (ru) 2007-06-29 2013-08-27 Ф.Хоффманн-Ля Рош Аг Промотор
WO2009006450A1 (en) * 2007-06-29 2009-01-08 Boston Biomedical, Inc. Bacteria-mediated gene modulation via microrna machinery
CA3002933A1 (en) 2007-07-14 2009-01-22 Beverly L. Davidson Methods and compositions for treating brain diseases
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
CA2694406C (en) 2007-07-26 2019-03-26 Amsterdam Molecular Therapeutics (Amt) B.V. Baculoviral vectors comprising repeated coding sequences with differential codon biases
US20100286378A1 (en) * 2007-08-27 2010-11-11 Boston Biomedical, Inc. Composition of Asymmetric RNA Duplex As MicroRNA Mimetic or Inhibitor
WO2009030025A1 (en) 2007-09-04 2009-03-12 Her Majesty The Queen In Right Of Canada As Represented By The Minister Of Health Porcine adeno-associated viruses
ES2426091T3 (es) 2007-09-19 2013-10-21 Uniqure Ip B.V. Uso de maquinaria de replicación de AAV para producción de proteína mejorada
EP2205740A2 (en) 2007-10-02 2010-07-14 Rxi Pharmaceuticals Corp. Tripartite rnai constructs
CA2701639A1 (en) 2007-10-04 2009-04-09 Board Of Regents, The University Of Texas System Modulating gene expression with agrna and gapmers targeting antisense transcripts
EP2186283A4 (en) 2007-10-18 2011-03-09 Lg Electronics Inc METHOD AND SYSTEM FOR TRANSMITTING AND RECEIVING SIGNALS
KR101614362B1 (ko) 2007-11-28 2016-04-22 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 유인원 아과 b 아데노바이러스 sadv-28,27,-29,-32,-33, 및 -35 및 그것의 사용
DK2220241T3 (en) 2007-11-28 2017-01-09 Univ Pennsylvania Adenovirus comprising a simian adenovirus E-SAdV-39-capsidhexonprotein and uses thereof
JP5758124B2 (ja) 2007-11-28 2015-08-05 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 サルサブファミリーCアデノウイルスSAdV−40、−31および−34ならびにそれらの用途
CA2710953A1 (en) 2007-12-28 2009-07-09 The Regents Of The University Of California Methods and compositions for increasing gene expression
US8133488B2 (en) 2008-01-18 2012-03-13 Genentech, Inc. Methods and compositions for targeting polyubiquitin
EP2242840B1 (en) 2008-01-29 2019-07-24 Applied Genetic Technologies Corporation Recombinant adeno-associated virus production using bhk cells in suspension
US10131904B2 (en) 2008-02-11 2018-11-20 Rxi Pharmaceuticals Corporation Modified RNAi polynucleotides and uses thereof
AU2009215987B2 (en) 2008-02-19 2015-01-22 Uniqure Ip B.V. Optimisation of expression of parvoviral rep and cap proteins in insect cells
EP2250255A2 (en) 2008-03-04 2010-11-17 The Trustees of the University of Pennsylvania Simian adenoviruses sadv-36,-42.1, -42.2, and -44 and uses thereof
WO2009137006A2 (en) 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
WO2009134681A2 (en) 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
WO2010011404A2 (en) 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
JP5689413B2 (ja) 2008-05-21 2015-03-25 ライニッシュ フリードリッヒ−ウィルヘルムズ−ユニバーシタット ボン 平滑末端を有する5’三リン酸オリゴヌクレオチドおよびその使用
EP2299812B1 (en) 2008-05-27 2017-07-12 Yale University Targeting tgf-beta as a therapy for alzheimer's disease
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
WO2009151620A2 (en) 2008-06-13 2009-12-17 Cornell Research Foundation, Inc. Wmc Pain treatment using erk2 inhibitors
US20110171262A1 (en) 2008-06-17 2011-07-14 Andrew Christian Bakker Parvoviral capsid with incorporated gly-ala repeat region
WO2010002851A1 (en) 2008-06-30 2010-01-07 Alnylam Pharmaceuticals, Inc. Silencing and rig-1 activation by dual function oligonucleotides
US8945885B2 (en) 2008-07-03 2015-02-03 The Board Of Trustees Of The Leland Stanford Junior University Minicircle DNA vector preparations and methods of making and using the same
WO2010011346A1 (en) 2008-07-24 2010-01-28 Rxi Pharmaceuticals Corporation Rnai constructs and uses therof
WO2010014857A2 (en) 2008-07-30 2010-02-04 University Of Massachusetts Chromosome therapy
CA2753338A1 (en) 2008-09-22 2010-03-25 Rxi Pharmaceuticals Corporation Neutral nanotransporters
CA2738969A1 (en) 2008-09-29 2010-04-01 Amsterdam Molecular Therapeutics (Amt) B.V. Porphobilinogen deaminase gene therapy
CN104689316A (zh) 2008-10-22 2015-06-10 弗·哈夫曼-拉罗切有限公司 轴突变性的调节
EP2774985B1 (en) 2008-10-31 2016-12-14 The Trustees Of The University Of Pennsylvania Simian adenovirus SAdV-43 and uses thereof
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
ES2634118T3 (es) 2009-02-11 2017-09-26 The University Of North Carolina At Chapel Hill Vectores de virus modificados y métodos para fabricar y utilizar los mismos
CN102341406B (zh) 2009-03-04 2016-06-08 德国癌症研究中心 组装活化蛋白(aap)及其用于制备基本上由vp3组成的细小病毒颗粒的应用
AU2010227419A1 (en) 2009-03-27 2011-10-20 Proyecto De Biomedicina Cima, S.L. Methods and compositions for the treatment of cirrhosis and liver fibrosis
US10920244B2 (en) 2009-04-30 2021-02-16 The Trustees Of The University Of Pennsylvania Compositions for targeting conducting airway cells comprising adeno-associated virus constructs
EP2772542B1 (en) 2009-05-28 2016-12-28 Deutsches Krebsforschungszentrum Modified AAV capsid polypeptides
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2010138675A1 (en) 2009-05-29 2010-12-02 The Trustees Of The University Of Pennsylvania Simian adenovirus 41 and uses thereof
US20120142764A1 (en) 2009-06-05 2012-06-07 Dai-Wu Seol Multi-Cistronic shRNA Expression Cassette for Suppressing Single or Multiple Target Genes
HUE054940T2 (hu) 2009-06-16 2021-10-28 Genzyme Corp Javított eljárások rekombináns AAV-vektorok tisztítására
CA2767225A1 (en) 2009-07-06 2011-01-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for enhancing production of a biological product
CA2767231A1 (en) 2009-07-06 2011-01-13 Alnylam Pharmaceuticals, Inc. Cell-based bioprocessing
EP2453735B1 (en) 2009-07-15 2015-07-08 Calimmune Inc. Dual Vector for Inhibition of Human Immunodeficiency Virus
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
WO2011038187A1 (en) 2009-09-25 2011-03-31 The Trustees Of The University Of Pennsylvania Controlled adeno-associated virus (aav) diversification and libraries prepared therefrom
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
WO2011069529A1 (en) 2009-12-09 2011-06-16 Curevac Gmbh Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids
WO2011088081A1 (en) 2010-01-12 2011-07-21 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
EP2531604B1 (en) 2010-02-05 2017-04-05 The University of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
WO2011101869A1 (en) * 2010-02-22 2011-08-25 Transgene Biotek Ltd. Adeno-associated virus 2/8 - micro rna-101 therapy for liver cancer
EP2545165B1 (en) 2010-03-11 2020-07-29 uniQure IP B.V. Mutated rep encoding sequences for use in aav production
US9546112B2 (en) 2010-03-22 2017-01-17 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
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
CA2833912C (en) 2010-04-23 2021-09-21 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
US9839696B2 (en) 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
AU2011285909B2 (en) 2010-08-02 2016-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of catenin (cadherin-associated protein), beta 1 (CTNNB1) gene expression using short interfering nucleic acid (siNA)
WO2012018881A2 (en) 2010-08-03 2012-02-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for the regulation of rna
US8808684B2 (en) 2010-09-10 2014-08-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Epidermal growth factor receptor (EGFR) and methods of use in adenoviral-associated virus type 6 (AAV6) transduction
CN101972476B (zh) * 2010-09-14 2012-12-19 中国人民解放军第二军医大学 利用MicroRNA-155的核酸疫苗佐剂及其构建方法
US9102943B2 (en) 2010-10-05 2015-08-11 Takara Bio Inc. Method for producing virus vector
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
JP5704361B2 (ja) 2010-10-27 2015-04-22 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
EP2637702A4 (en) 2010-11-11 2014-11-26 Univ Miami METHODS, COMPOSITIONS, CELLS AND KITS FOR TREATING ISCHEMIC INJURIES
AU2011332025B2 (en) 2010-11-23 2015-06-25 The Trustees Of The University Of Pennsylvania Subfamily E simian adenoviruses A1321, A1325, A1295, A1309 and A1322 and uses thereof
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
WO2012115980A1 (en) 2011-02-22 2012-08-30 California Institute Of Technology Delivery of proteins using adeno-associated virus (aav) vectors
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
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
JP6007463B2 (ja) 2011-04-18 2016-10-12 国立研究開発法人国立精神・神経医療研究センター 薬剤送達粒子及びその製造方法
EP2699688A1 (en) 2011-04-20 2014-02-26 The Trustees Of The University Of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
US9226976B2 (en) 2011-04-21 2016-01-05 University Of Massachusetts RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
MX349138B (es) 2011-04-22 2017-07-13 Univ California Variones de virus adeno-asociados con capside variante y sus metodos de uso.
WO2012149646A1 (en) * 2011-05-05 2012-11-08 Sunnybrook Research Institute Mirna inhibitors and their uses
WO2012158757A1 (en) 2011-05-16 2012-11-22 The Trustees Of The University Of Pennsylvania Proviral plasmids for production of recombinant adeno-associated virus
WO2012159006A2 (en) 2011-05-18 2012-11-22 University Of Florida Research Foundation, Inc. Polypeptides and vectors for targeting her2/neu expressing cells and uses thereof
WO2012168003A1 (en) 2011-06-06 2012-12-13 Biocartis S.A. Selective lysis of cells by ionic surfactants
CA3106285A1 (en) 2011-07-25 2013-01-31 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
ES2623780T3 (es) 2011-07-27 2017-07-12 Genethon Sistemas de expresión de baculovirus mejorados
US8852911B2 (en) 2011-08-04 2014-10-07 The Regents Of The University Of California Method of producing dicer
US20130129668A1 (en) 2011-09-01 2013-05-23 The Regents Of The University Of California Diagnosis and treatment of arthritis using epigenetics
CA2847604A1 (en) 2011-09-08 2013-03-14 Uniqure Ip B.V. Removal of contaminating viruses from aav preparations
US9464322B2 (en) 2011-09-09 2016-10-11 University Of Kentucky Research Foundation Methods for diagnosing and treating alzheimer's disease (AD) using the molecules that stabilize intracellular calcium (Ca2+) release
WO2013036889A1 (en) 2011-09-09 2013-03-14 University Of Washington Retrograde transport peptide and use of same for delivery to central nervous system
EP2758433B1 (en) 2011-09-19 2017-10-18 Axon Neuroscience SE Protein-based therapy and diagnosis of tau-mediated pathology in alzheimer's disease
EP2771455B1 (en) 2011-10-28 2016-10-05 The University of North Carolina At Chapel Hill Cell line for production of adeno-associated virus
AU2012340567B2 (en) 2011-11-22 2017-11-23 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
WO2013078199A2 (en) 2011-11-23 2013-05-30 Children's Medical Center Corporation Methods for enhanced in vivo delivery of synthetic, modified rnas
US9611305B2 (en) 2012-01-06 2017-04-04 Mayo Foundation For Medical Education And Research Treating cardiovascular or renal diseases
WO2013113696A1 (en) 2012-01-30 2013-08-08 Vib Vzw Means and method for diagnosis and treatment of alzheimer's disease
EP2814513B1 (en) 2012-02-14 2017-12-20 The Regents of The University of California Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions
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
WO2013122605A1 (en) 2012-02-17 2013-08-22 Evernote Corporation Site memory processing
US20150148405A1 (en) 2012-02-21 2015-05-28 The Johns Hopkins University Expression Construct for a Lin28-Resistant Let-7 Precursor MicroRNA
EP2820159B1 (en) 2012-02-29 2019-10-23 Sangamo Therapeutics, Inc. Methods and compositions for treating huntington's disease
HUE054087T2 (hu) 2012-04-18 2021-09-28 Childrens Hospital Philadelphia Készítmények és eljárások nagy hatékonyságú géntranszferre AAV kapszidvariánsok alkalmazásával
EP2660325A3 (en) 2012-05-02 2014-02-12 Christian Medical College AAV vectors and corresponding nucleotide sequences and methods
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
BR112014027724B1 (pt) 2012-05-08 2022-05-03 Merck Sharp & Dohme Corp. E Alectos Therapeutics Inc Composto, e, composição farmacêutica
WO2013170078A1 (en) 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
IN2014KN02672A (enExample) 2012-05-18 2015-05-08 Univ Iowa Res Found
WO2013173702A2 (en) 2012-05-18 2013-11-21 The Trustees Of The University Of Pennsylvania Subfamily e simian adenoviruses a1302, a1320, a1331 and a1337 and uses thereof
WO2013177367A2 (en) 2012-05-23 2013-11-28 The Johns Hopkins University Compounds and methods of use thereof for treating neurodegenerative disorders
EP2871239B1 (en) 2012-07-06 2018-08-29 Takara Bio Inc. Cell capable of producing adeno-associated virus vector
JP6366581B2 (ja) 2012-07-06 2018-08-08 ユニバーシティー オブ アイオワ リサーチ ファウンデーション 改変されたアデノ随伴ウイルスベクター組成物
EP2875133B1 (en) * 2012-07-17 2018-01-10 Université de Genève Nucleic acids for down-regulation of gene expression
US20150252384A1 (en) 2012-08-01 2015-09-10 National Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
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
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
AU2013204200B2 (en) 2012-10-11 2016-10-20 Brandeis University Treatment of amyotrophic lateral sclerosis
US9733237B2 (en) 2012-10-31 2017-08-15 The Trustees Of Columbia University In The City Of New York Methods for identifying candidates for the treatment of neurodegenerative diseases
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
CN104937100B (zh) 2012-12-25 2020-04-03 宝生物工程株式会社 Aav 变体
EP3521420A1 (en) 2013-01-08 2019-08-07 Genzyme Corporation Use of inos inhibitors to increase viral yield in culture
US10000753B2 (en) 2013-01-08 2018-06-19 Benitec Biopharma Limited Age-related macular degeneration treatment
US9066966B2 (en) * 2013-02-01 2015-06-30 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
DK2954051T3 (da) 2013-02-08 2019-07-08 Univ Pennsylvania Modificeret kapsid til genoverførsel til behandling af nethinden
US9726009B2 (en) 2013-03-12 2017-08-08 Halliburton Energy Services, Inc. Wellbore servicing tools, systems and methods utilizing near-field communication
AU2014244167A1 (en) 2013-03-13 2015-10-08 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
WO2014144844A1 (en) 2013-03-15 2014-09-18 The Board Of Trustees Of The Leland Stanford Junior University tRNA DERIVED SMALL RNAs (tsRNAs) INVOLVED IN CELL VIABILITY
US9447433B2 (en) 2013-03-15 2016-09-20 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
CA2904156C (en) 2013-03-15 2023-01-10 The Children's Hospital Of Philadelphia Vectors comprising stuffer/filler polynucleotide sequences and methods of use
CA3174963A1 (en) 2013-04-08 2014-10-16 University Of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
DK2986635T3 (en) 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
WO2014172669A1 (en) 2013-04-20 2014-10-23 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted u7snrna polynucleotide constructs
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
CN115120746A (zh) 2013-05-15 2022-09-30 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
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
CA2907799A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
WO2014201308A1 (en) 2013-06-12 2014-12-18 Washington University Endothelial-targeted adenoviral vectors, methods and uses therefor
US20160122727A1 (en) 2013-06-13 2016-05-05 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
SI3024498T1 (sl) 2013-07-22 2020-07-31 The Children's Hospital Of Philadelphia Različica AAV in sestavki, postopki in uporabe za prenos genov v celice, organe in tkiva
RU2664471C2 (ru) 2013-07-26 2018-08-17 Юниверсити Оф Айова Рисерч Фаундейшн Способы и композиции для лечения болезней мозга
ITTO20130669A1 (it) 2013-08-05 2015-02-06 Consiglio Nazionale Ricerche Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari
EP4219727A3 (en) 2013-08-27 2023-09-06 Research Institute at Nationwide Children's Hospital Products and methods for treatment of amyotrophic lateral sclerosis
EP3039129B1 (en) 2013-08-30 2018-06-27 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
EP3044318B1 (en) 2013-09-13 2019-05-01 California Institute of Technology Selective recovery
EP3049527A4 (en) 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
WO2015044292A1 (en) 2013-09-26 2015-04-02 Universitat Autonoma De Barcelona Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease
EP3459965B1 (en) 2013-10-11 2020-12-02 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
WO2015060722A1 (en) 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
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
US20160272687A1 (en) 2013-11-08 2016-09-22 The Board Of Trustees Of The University Of Arkansas Adeno-associated virus "x" oncogene
EP3068905A4 (en) 2013-11-11 2017-07-05 Sangamo BioSciences, Inc. Methods and compositions for treating huntington's disease
CN105934515B (zh) 2013-11-26 2020-08-04 美国政府(由卫生和人类服务部的部长所代表) 用于治疗糖原贮积病的腺相关病毒载体
EP3075849B1 (en) 2013-11-29 2019-03-13 Takara Bio Inc. Method for quantifying adeno-associated virus
SG11201604505QA (en) 2013-12-03 2016-07-28 Agency Science Tech & Res Tailed Mirtron Effectors For RNAi-Mediated Gene Silencing
US9732345B2 (en) 2013-12-09 2017-08-15 Baylor College Of Medicine Hippo and dystrophin complex signaling in cardiomyocyte renewal
AU2014362245A1 (en) 2013-12-12 2016-06-16 Massachusetts Institute Of Technology Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
US20150197809A1 (en) 2014-01-13 2015-07-16 Trustees Of Boston University Methods and assays relating to huntingtons disease and parkinson'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
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
RU2016136989A (ru) 2014-02-19 2018-03-22 Ф.Хоффманн-Ля Рош Аг Шаттл для гематоэнцефалического барьера
WO2015124546A1 (en) 2014-02-19 2015-08-27 Fundación Centro Nacional De Investigaciones Cardiovasculares Carlos Iii- Cnic Aav vectors for the treatment of ischemic and non-ischemic heart disease
US20170007720A1 (en) 2014-02-21 2017-01-12 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
EP3110974A4 (en) 2014-02-24 2018-01-24 Celgene Corporation Methods of using an activator of cereblon for neural cell expansion and the treatment of central nervous system disorders
EP3113787B1 (en) 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
AU2015226911B2 (en) 2014-03-07 2018-03-01 The Arizona Board Of Regents On Behalf Of The University Of Arizona Non-narcotic CRMP2 peptides targeting sodium channels for chronic pain
PL3117005T3 (pl) 2014-03-10 2024-11-04 Uniqure Ip B.V. Dodatkowo ulepszone wektory aav produkowane w komórkach owadzich
US10280418B2 (en) 2014-03-18 2019-05-07 Univeristy Of Massachusetts RAAV-based compositions and methods for treating amyotrophic lateral sclerosis
PL3757214T3 (pl) 2014-04-01 2022-09-12 Biogen Ma Inc. Kompozycje do modulowania ekspresji sod-1
WO2015157070A2 (en) 2014-04-09 2015-10-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
EP3134431B1 (en) 2014-04-25 2021-04-07 The Trustees Of The University Of Pennsylvania Ldlr variants and their use in compositions for reducing cholesterol levels
EP3139966B1 (en) 2014-05-08 2020-11-18 Sangamo Therapeutics, Inc. Compositions for use in treating huntington's disease
US20170088819A1 (en) 2014-05-16 2017-03-30 Vrije Universiteit Brussel Genetic correction of myotonic dystrophy type 1
ES2759317T3 (es) 2014-05-20 2020-05-08 Univ Iowa Res Found Compuestos terapéuticos para la enfermedad de Huntington
WO2015183667A1 (en) 2014-05-28 2015-12-03 The Regents Of The University Of California HYBRID tRNA/pre-miRNA MOLECULES AND METHODS OF USE
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US20150376612A1 (en) 2014-06-10 2015-12-31 The General Hospital Corporation CCCTC-Binding Factor (CTCF) RNA Interactome
US11207424B2 (en) 2014-06-13 2021-12-28 Mayo Foundation For Medical Education And Research Methods and materials for increasing viral vector infectivity
US10781459B2 (en) 2014-06-20 2020-09-22 University Of Florida Research Foundation, Incorporated Methods of packaging multiple adeno-associated virus vectors
US10526583B2 (en) 2014-07-02 2020-01-07 University Of Florida Research Foundation, Incorporated Compositions and methods for purifying recombinant adeno-associated virus
US10023846B2 (en) 2014-07-10 2018-07-17 Takara Bio Inc. Production method for non-enveloped virus particles
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
CA2955285C (en) 2014-07-31 2023-09-26 Association Institut De Myologie Treatment of amyotrophic lateral sclerosis
WO2016019364A1 (en) 2014-08-01 2016-02-04 The Trustees Of The University Of Pennsylvania Compositions and methods for self-regulated inducible gene expression
WO2016040347A2 (en) 2014-09-08 2016-03-17 University Of Iowa Research Foundation Microrna inhibitor system and methods of use thereof
WO2016054554A1 (en) 2014-10-03 2016-04-07 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
US10842886B2 (en) 2014-10-10 2020-11-24 Research Institute At Nationwide Children's Hospital Guided injections for AAV gene transfer to muscle
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
WO2016073693A2 (en) 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
AU2015342997B2 (en) 2014-11-05 2021-11-18 Research Institute At Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
CA2975583A1 (en) * 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
CA2967393A1 (en) 2014-11-21 2016-05-26 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
WO2016081927A2 (en) 2014-11-21 2016-05-26 University Of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
US11021762B2 (en) 2014-11-28 2021-06-01 Uniqure Ip B.V. DNA impurities in a composition comprising a parvoviral virion
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
LT3237618T (lt) 2014-12-24 2019-07-10 Uniqure Ip B.V. Rnri sukeltas hantingtino geno slopinimas
KR102618947B1 (ko) 2014-12-30 2023-12-27 유니버시티 오브 아이오와 리써치 파운데이션 뇌 질환을 치료하기 위한 방법 및 조성물
EP3245220B1 (en) 2015-01-14 2023-09-20 The University of North Carolina at Chapel Hill Methods and compositions for targeted gene transfer
MX2017009336A (es) 2015-01-16 2017-11-15 Voyager Therapeutics Inc Polinucleótidos dirigidos al sistema nervioso central.
JP2018506530A (ja) 2015-01-30 2018-03-08 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 脊柱軟膜下遺伝子送達システム
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
HK1247641A1 (zh) 2015-02-10 2018-09-28 Genzyme Corporation 变体RNAi
LT3256594T (lt) 2015-02-10 2022-02-10 Genzyme Corporation Patobulintas virusinių dalelių tiekimas į dryžuotąjį kūną ir smegenų žievę
EP3262162A4 (en) 2015-02-23 2018-08-08 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
EP3270960A4 (en) 2015-03-20 2018-08-08 Bluebird Bio, Inc. Vector formulations
CA2979065A1 (en) 2015-03-24 2016-09-29 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
HUE050441T2 (hu) 2015-04-03 2020-12-28 Univ Massachusetts Huntingtin-mRNS célzására szolgáló oligonukleotid-vegyületek
JP6892433B2 (ja) 2015-04-03 2021-06-23 ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts 十分に安定化された非対称sirna
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
ES2763551T3 (es) 2015-04-16 2020-05-29 Univ Emory Promotores y vectores recombinantes para la expresión de proteínas en el hígado y uso de los mismos
EP4491733A3 (en) 2015-04-23 2025-03-26 University of Massachusetts Modulation of aav vector transgene expression
WO2016172008A1 (en) 2015-04-24 2016-10-27 University Of Massachusetts Modified aav constructions and uses thereof
WO2016179038A1 (en) 2015-05-01 2016-11-10 Spark Therapeutics, Inc. ADENO-ASSOCIATED VIRUS-MEDIATED CRISPR-Cas9 TREATMENT OF OCULAR DISEASE
AU2016256894B2 (en) 2015-05-07 2020-11-26 Massachusetts Eye And Ear Infirmary Methods of delivering an agent to the eye
EP3294398A4 (en) 2015-05-11 2019-01-09 Alcyone Lifesciences, Inc. SYSTEM AND METHOD FOR THE ADMINISTRATION OF MEDICAMENTS
CA2985786A1 (en) 2015-05-12 2016-11-17 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
US20170067028A1 (en) 2015-05-15 2017-03-09 Douglas J. Ballon Radiolabeling of adeno associated virus
US10729790B2 (en) 2015-05-26 2020-08-04 Salk Institute For Biological Studies Motor neuron-specific expression vectors
IL322085A (en) 2015-05-29 2025-09-01 Univ Pennsylvania Compositions and methods for reducing unfolded proteins
WO2016196507A1 (en) 2015-05-29 2016-12-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
WO2017004514A1 (en) 2015-07-02 2017-01-05 University Of Florida Research Foundation, Inc. Recombinant adeno-associated virus vectors to target medullary thyroid carcinoma
US20170007669A1 (en) 2015-07-07 2017-01-12 Mayo Foundation For Medical Education And Research Peptide-mediated delivery of active agents across the blood-brain barrier
EP3320101B1 (en) 2015-07-07 2021-08-11 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
WO2017015102A1 (en) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
CA2988471A1 (en) 2015-07-28 2017-02-02 University Of Massachusetts Transgenic expression of dnase i in vivo delivered by an adeno-associated virus vector
JP7166168B2 (ja) 2015-07-30 2022-11-07 マサチューセッツ アイ アンド イヤー インファーマリー 祖先ウイルス配列およびその使用
US20190000940A1 (en) 2015-07-31 2019-01-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
US20180201937A1 (en) 2015-08-04 2018-07-19 The University Of Chicago Inhibitors of cacna1a/alpha1a subunit internal ribosomal entry site (ires) and methods of treating spinocerebellar ataxia type 6
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
WO2017053753A1 (en) 2015-09-23 2017-03-30 Sangamo Biosciences, Inc. Htt repressors and uses thereof
CA2996420A1 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
AU2016335572B2 (en) 2015-10-09 2022-12-08 The Children's Hospital Of Philadelphia Compositions and methods for treating Huntington's disease and related disorders
EP4567425A3 (en) 2015-10-09 2025-10-08 Genzyme Corporation Early post-transfection isolation of cells (epic) for biologics production
US10123969B2 (en) 2015-10-15 2018-11-13 Wisconsin Alumni Research Foundation Osmotic enhancement of drug/therapeutic delivery to the brain following infusion or injection into the cerebrospinal fluid
EP3364996B1 (en) 2015-10-22 2021-08-25 University of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
DK3364997T5 (da) 2015-10-22 2024-09-30 Univ Massachusetts Aspartoacylase genterapi til behandling af canavans sygdom
CN116650668A (zh) 2015-10-23 2023-08-29 衣阿华大学研究基金会 使用基因疗法以推迟疾病发生和发展同时提供认知保护来治疗神经变性疾病的方法
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
WO2017075119A1 (en) 2015-10-28 2017-05-04 The Trustees Of The Univeresity Of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
WO2017079768A1 (en) 2015-11-08 2017-05-11 Genentech, Inc. Methods of screening for multispecific antibodies
US10633662B2 (en) 2015-11-10 2020-04-28 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for modulating AAV infection
WO2017096039A1 (en) 2015-12-01 2017-06-08 Spark Therapeutics, Inc. Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use
US20170151416A1 (en) 2015-12-01 2017-06-01 Invivo Therapeutics Corporation Methods and Systems for Delivery of a Trail of a Therapeutic Substance into an Anatomical Space
EP3384034B1 (en) 2015-12-02 2020-07-08 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
CA3004971A1 (en) 2015-12-03 2017-06-08 Universite D'evry Val D'essonne Compositions and methods for improving viral vector efficiency
EP3387118B1 (en) 2015-12-11 2022-04-06 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
EP3387117B1 (en) 2015-12-11 2022-11-23 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
CA3007495C (en) 2015-12-11 2023-04-11 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (aavs)
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
CA3008013A1 (en) 2015-12-14 2017-06-22 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
WO2017112948A1 (en) 2015-12-24 2017-06-29 University Of Florida Research Foundation, Inc. Improved aav production using suspension adapted cells
WO2017122789A1 (ja) 2016-01-15 2017-07-20 学校法人自治医科大学 てんかん治療のためのアデノ随伴ウイルスビリオン
EP3411059A4 (en) 2016-02-02 2019-10-16 University Of Massachusetts METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM
WO2017136202A1 (en) 2016-02-05 2017-08-10 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
CN109415704B (zh) 2016-02-16 2022-02-25 利兰斯坦福初级大学董事会 对先已存在的人中和抗体有抗性的新型重组腺相关病毒衣壳
WO2017147477A1 (en) 2016-02-26 2017-08-31 University Of Florida Research Foundation, Inc. Aav heparin mutants that display significantly improved eye and brain transduction
EP3957331A1 (en) 2016-03-03 2022-02-23 University Of Massachusetts Closed-ended linear duplex dna for non-viral gene transfer
WO2017155973A1 (en) 2016-03-07 2017-09-14 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
AU2017234929B2 (en) 2016-03-18 2024-05-02 The Children's Hospital Of Philadelphia Therapeutic for treatment of diseases including the central nervous system
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
EP3436476A4 (en) 2016-03-28 2020-04-29 The Regents of The University of California ANTI-RYK ANTIBODIES AND METHOD FOR USE THEREOF
LT3436593T (lt) 2016-03-28 2023-03-10 Ultragenyx Pharmaceutical Inc. Adenoviruso šiluminio inaktyvavimo būdai
BR112018070250A2 (pt) 2016-03-30 2019-01-29 Spark Therapeutics Inc linhagem celular para proteína recombinante e/ou produção de vetores virais
WO2017173283A1 (en) 2016-03-31 2017-10-05 Spark Therapeutics, Inc. Column-based fully scalable raav manufacturing process
CN109661470A (zh) 2016-04-15 2019-04-19 宾夕法尼亚州大学信托人 新型aav8突变衣壳和含有其的组合物
ES2989468T3 (es) 2016-04-16 2024-11-26 Univ Florida Métodos para mejorar la potencia biológica de virus adenoasociados recombinantes producidos en un sistema baculovírico
NZ744418A (en) 2016-04-21 2025-10-31 Virovek Inc Aav production in insect cells, methods and compositions therefor
EP3235516B1 (en) 2016-04-22 2019-06-26 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts Universitätsmedizin Regulatable adeno-associated virus (aav) vector
WO2017190031A1 (en) 2016-04-28 2017-11-02 Indiana University Research And Technology Corporation Methods and compositions for resolving components of a virus preparation
WO2017189963A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3452495B1 (en) 2016-05-03 2021-06-23 Children's Medical Research Institute Adeno-associated virus polynucleotides, polypeptides and virions
US11866462B2 (en) 2016-05-04 2024-01-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
IL262784B2 (en) 2016-05-18 2023-10-01 Voyager Therapeutics Inc modulatory polynucleotides
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
US10898585B2 (en) 2017-04-14 2021-01-26 Ptc Therapeutics .Inc. Gene therapy for AADC deficiency
EP3619310A4 (en) 2017-05-05 2021-01-27 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
EP3635121A1 (en) 2017-06-02 2020-04-15 Institut National de la Sante et de la Recherche Medicale (INSERM) Viral vector combining gene therapy and genome editing approaches for gene therapy of genetic disorders
GB201714027D0 (en) 2017-09-01 2017-10-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of huntington's disease
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
JP7565218B2 (ja) 2018-07-02 2024-10-10 ボイジャー セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症および脊髄に関連する障害の治療

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