MX2018014152A - Composiciones y métodos para tratar la enfermedad de huntington. - Google Patents

Composiciones y métodos para tratar la enfermedad de huntington.

Info

Publication number
MX2018014152A
MX2018014152A MX2018014152A MX2018014152A MX2018014152A MX 2018014152 A MX2018014152 A MX 2018014152A MX 2018014152 A MX2018014152 A MX 2018014152A MX 2018014152 A MX2018014152 A MX 2018014152A MX 2018014152 A MX2018014152 A MX 2018014152A
Authority
MX
Mexico
Prior art keywords
disease
methods
compositions
treating huntington
sirna
Prior art date
Application number
MX2018014152A
Other languages
English (en)
Inventor
Deckert Jochen
Hossbach Markus
Wang Xin
Wen-Yee Sah Dinah
Hou Jinzhao
Zhou Pengcheng
Original Assignee
Voyager Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of MX2018014152A publication Critical patent/MX2018014152A/es

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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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Abstract

La presente invención se refiere a pequeñas moléculas de ARN interferente (ARNip) contra el gen HTT, vectores virales adenoasociados (AAV) que codifican moléculas de ARNsi y métodos para tratar la Enfermedad de Huntington (HD) utilizando las moléculas de ARNsi y los vectores AAV.
MX2018014152A 2016-05-18 2017-05-18 Composiciones y métodos para tratar la enfermedad de huntington. MX2018014152A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662338113P 2016-05-18 2016-05-18
US201762485049P 2017-04-13 2017-04-13
PCT/US2017/033281 WO2017201258A1 (en) 2016-05-18 2017-05-18 Compositions and methods of treating huntington's disease

Publications (1)

Publication Number Publication Date
MX2018014152A true MX2018014152A (es) 2019-03-28

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018014152A MX2018014152A (es) 2016-05-18 2017-05-18 Composiciones y métodos para tratar la enfermedad de huntington.

Country Status (13)

Country Link
US (1) US11951121B2 (es)
EP (1) EP3458589A4 (es)
JP (1) JP7220080B2 (es)
KR (2) KR20220108216A (es)
CN (1) CN109831916B (es)
AU (1) AU2017268382B2 (es)
BR (1) BR112018073472A2 (es)
CA (1) CA3024449A1 (es)
IL (2) IL297576B2 (es)
MX (1) MX2018014152A (es)
RU (1) RU2764587C2 (es)
SG (1) SG11201809643UA (es)
WO (1) WO2017201258A1 (es)

Families Citing this family (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2749882C2 (ru) 2014-11-14 2021-06-18 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
EP3218484A4 (en) 2014-11-14 2018-05-30 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
RU2758488C2 (ru) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
WO2018204803A1 (en) * 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2019060649A1 (en) * 2017-09-22 2019-03-28 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING HUNTINGTON'S DISEASE
AR113134A1 (es) * 2017-09-22 2020-01-29 Genzyme Corp Arni variante
EP4124658A3 (en) 2017-10-16 2023-04-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019213183A1 (en) * 2018-04-30 2019-11-07 The Trustees Of The University Of Pennsylvania In utero crispr-mediated therapeutic editing of genes
WO2019222444A2 (en) * 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Directed evolution
EP3807404A1 (en) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
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
US20210355454A1 (en) * 2018-07-24 2021-11-18 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
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
HRP20240586T1 (hr) 2018-10-09 2024-07-19 The University Of British Columbia Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima
CA3116701A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. Expression vectors for large-scale production of raav in the baculovirus/sf9 system
AU2019382824A1 (en) * 2018-11-19 2021-06-03 Uniqure Ip B.V. Method and means to deliver miRNA to target cells
US20220064671A1 (en) 2019-01-18 2022-03-03 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
WO2020172624A1 (en) * 2019-02-22 2020-08-27 Life Technologies Corporation Suspension system for adeno associated virus production
EP3962536A1 (en) 2019-04-29 2022-03-09 Voyager Therapeutics, Inc. Systems and methods for producing baculoviral infected insect cells (biics) in bioreactors
EP4010465A1 (en) 2019-08-09 2022-06-15 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
US20220364114A1 (en) 2019-08-26 2022-11-17 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2021072129A2 (en) 2019-10-08 2021-04-15 Trustees Of Boston College Proteins containing multiple, different unnatural amino acids and methods of making and using such proteins
CN112980837B (zh) * 2019-12-13 2023-07-04 深圳艾码生物科技有限公司 一种抑制HTT基因表达的siRNA及其前体和应用
CA3170369A1 (en) 2020-03-05 2022-04-14 Michal Shahar Methods and compositions for treating cancer with immune cells
AU2021265832A1 (en) 2020-04-29 2022-11-24 The Broad Institute, Inc. Machine learning accelerated protein engineering through fitness prediction
BR112023001648A2 (pt) 2020-07-27 2023-04-04 Anjarium Biosciences Ag Moléculas de dna de fita dupla, veículo de entrega e método para preparar uma molécula de dna com extremidade em grampo
WO2022032153A1 (en) 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
US20230340041A1 (en) 2020-08-17 2023-10-26 Massachusetts Institute Of Technology Shank3 gene therapy approaches
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
EP4396201A1 (en) * 2021-09-03 2024-07-10 BioMarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023060264A1 (en) 2021-10-08 2023-04-13 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
AU2023211606A1 (en) * 2022-01-27 2024-07-11 Asklepios Biopharmaceutical, Inc. Compositions for treating neurological disease
WO2023164545A1 (en) 2022-02-23 2023-08-31 Massachusetts Institute Of Technology Methods for upregulating shank3 expression
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2024129992A1 (en) * 2022-12-15 2024-06-20 President And Fellows Of Harvard College Enhancers driving expression in motor neurons
WO2024137931A1 (en) * 2022-12-21 2024-06-27 Icahn School Of Medicine At Mount Sinai Inhibition of zbtb7a
WO2024145474A2 (en) 2022-12-29 2024-07-04 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt

Family Cites Families (433)

* 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
WO1995028493A1 (en) 1994-04-13 1995-10-26 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
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
CA2207927A1 (en) 1994-12-06 1996-06-13 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
WO1997008298A1 (en) 1995-08-30 1997-03-06 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
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
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
US20020037867A1 (en) 1999-02-26 2002-03-28 James M. Wilson 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
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
WO1998010087A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Chimpanzee adenovirus vectors
AU723497C (en) 1996-09-06 2001-10-11 Trustees Of The University Of Pennsylvania, The Method for recombinant adeno-associated virus-directed gene therapy
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
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
ATE550429T1 (de) 1996-11-20 2012-04-15 Crucell Holland Bv Adenovirus-zusammensetzungen erhältlich durch ein verbessertes produktions- und reinigungsverfahren
AU741605B2 (en) 1996-12-18 2001-12-06 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
CA2303768C (en) 1997-09-19 2009-11-24 The Trustees Of The University Of 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
CA2304131A1 (en) 1997-09-19 1999-04-01 James M. Wilson Method for gene transfer using bcl2 and compositions useful therein
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
NZ505325A (en) 1997-12-23 2003-07-25 Crucell Holland B 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
WO1999043360A1 (en) 1998-02-26 1999-09-02 The Trustees Of The University Of Pennsylvania 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
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
ATE402254T1 (de) 1998-05-28 2008-08-15 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
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
WO2000022152A1 (en) 1998-10-13 2000-04-20 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
JP2002528087A (ja) 1998-10-27 2002-09-03 クルセル ホランド ベー ヴェー 改良ａａｖベクター産生
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
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
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
JP2002542805A (ja) 1999-04-30 2002-12-17 ユニバーシティ オブ フロリダ アデノ随伴ウイルス送達リボザイム組成物および使用方法
EP1183380A1 (en) 1999-06-02 2002-03-06 The Trustees Of The University Of Pennsylvania Compositions and methods useful for production of recombinant viruses which require helper viruses
WO2000075365A2 (en) 1999-06-08 2000-12-14 University Of Iowa Research Foundation Compounds and methods to enhance raav transduction
CA2382483A1 (en) 1999-08-20 2001-03-01 Johns Hopkins University School Of Medicine Methods and compositions for the construction and use of fusion libraries
CA2384814A1 (en) 1999-09-29 2001-04-05 The Trustees Of The University Of Pennsylvania Methods for rapid peg-modification of viral vectors, compositions for enhanced gene transduction, compositions with enhanced physical stability, and uses therefor
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
US7241447B1 (en) 1999-10-07 2007-07-10 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
WO2001025465A1 (en) 1999-10-07 2001-04-12 University Of Iowa Research Foundation Adeno-associated viruses 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
AU3642601A (en) 1999-11-05 2001-05-30 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
AU1954001A (en) 1999-12-10 2001-06-18 Ariad Gene Therapeutics, Inc. Methods for high level expression of genes in primates
US7638120B2 (en) 2000-03-14 2009-12-29 Thomas Jefferson University High transgene expression of a pseudotyped adeno-associated virus type
CA2373110A1 (en) 2000-03-14 2001-09-20 Neurologix, Inc. Production of chimeric capsid vectors
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
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
US7048920B2 (en) 2000-03-24 2006-05-23 Cell Genesys, Inc. Recombinant oncolytic adenovirus for human melanoma
DK1309726T4 (en) 2000-03-30 2019-01-28 Whitehead Inst Biomedical Res RNA Sequence-Specific Mediators of RNA Interference
GB0009887D0 (en) 2000-04-20 2000-06-07 Btg Int Ltd Cytotoxic agents
AU2001255575B2 (en) 2000-04-28 2006-08-31 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
US7125705B2 (en) 2000-04-28 2006-10-24 Genzyme Corporation Polynucleotides for use in recombinant adeno-associated virus virion production
US20020106381A1 (en) 2000-06-13 2002-08-08 High Katherine A. Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus
WO2002006483A1 (fr) 2000-07-18 2002-01-24 Takeda Chemical Industries, Ltd. Nouveau peptide actif sur le plan physiologique et utilisation associee
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
ES2288993T3 (es) 2000-08-17 2008-02-01 Keiya Ozawa Distribucion de factores angiogenicos mediada por virus adenoasociados.
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
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
JP2004532822A (ja) 2001-03-14 2004-10-28 アビジェン， インコーポレイテッド ビリオンの管逆行感染による組換えアデノ随伴ウイルス媒介遺伝子移入
US20060217331A1 (en) * 2001-05-18 2006-09-28 Sirna Therapeutics, Inc. Chemically modified double stranded nucleic acid molecules that mediate RNA interference
ATE530672T1 (de) 2001-06-22 2011-11-15 Univ Pennsylvania Rekombinante adenoviren mit affen-adenovirus proteinen und verwendung davon.
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
US7319002B2 (en) 2001-08-08 2008-01-15 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
EP1430128B1 (en) 2001-09-28 2018-04-25 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Micro-rna 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
WO2003042361A2 (en) 2001-11-09 2003-05-22 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
NZ578982A (en) 2001-11-13 2011-03-31 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
EP3108899A1 (en) 2001-11-21 2016-12-28 The Trustees of the University of Pennsylvania Simian adenovirus adsv1 nucleic acid and amino acid sequences, vectors containing same, and methods of use
EP1453536A4 (en) 2001-12-12 2009-08-26 Mayne Pharma Int Pty Ltd COMPOSITION FOR PRESERVING VIRUSES
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
JP4769417B2 (ja) 2001-12-17 2011-09-07 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９の配列、それを含むベクターおよびその使用
US7112321B2 (en) 2001-12-19 2006-09-26 Genzyme Corporation Adeno-associated virus-mediated delivery of GDNF to skeletal muscles
JP2005538929A (ja) 2002-01-16 2005-12-22 ダイナル バイオテック エイエスエイ 単一サンプルからの核酸及びタンパク質の単離方法
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
JP3943048B2 (ja) 2002-04-29 2007-07-11 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 組織の細胞ｄｎａからの組込みウイルスの直接レスキュー及び増幅の方法
WO2003093463A1 (en) 2002-04-30 2003-11-13 Oncolytics Biotech Inc. Improved viral purification methods
NZ561656A (en) 2002-05-01 2009-03-31 Univ Florida Improved rAAV expression systems for genetic modification of specific capsid proteins
SI1504126T1 (sl) 2002-05-03 2014-08-29 Duke University Office Of Science And Technology Postopek regulacije izraĹľanja genov
PT1506287E (pt) 2002-05-14 2007-07-17 Merck & Co Inc Métodos de purificação de adenovírus
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
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O 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
US20050255086A1 (en) 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
EP1546344A4 (en) 2002-09-18 2007-10-03 Isis Pharmaceuticals Inc EFFICIENT REDUCTION OF TARGET RNA USING OLIGOMERIC COMPOUNDS WITH SINGLE AND DOUBLE STRAPS
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
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
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
ATE490307T1 (de) 2003-05-21 2010-12-15 Genzyme Corp Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind
EP1633890B2 (en) * 2003-06-02 2020-11-18 University of Massachusetts METHODS AND COMPOSITIONS FOR ENHANCING THE EFFICACY AND SPECIFICITY OF FNAi
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
SI2657248T1 (sl) 2003-06-19 2017-07-31 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in njihova uporaba
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
WO2005013901A2 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
EP2821085B1 (en) 2003-09-12 2020-04-29 University of Massachusetts Rna interference for the treatment of gain-of-function disorders
ES2648241T3 (es) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
CN1926551B (zh) 2003-10-27 2010-06-16 罗斯塔生化科技有限责任公司 用于基因沉默的siRNA的设计方法
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
CA2569244C (en) 2004-06-01 2017-02-14 Avigen, Inc. Compositions and methods to prevent aav vector aggregation
EP1773401B1 (en) 2004-06-21 2013-01-02 Medtronic, Inc. Medical systems and methods for delivering compositions to cells
MX2007003850A (es) 2004-10-05 2007-11-21 Genzyme Corp Canula escalonada.
WO2006043354A1 (ja) 2004-10-20 2006-04-27 National Institute Of Radiological Sciences 組込型の低線量放射線誘導性ベクター
US7901921B2 (en) 2004-10-22 2011-03-08 Oncolytics Biotech Inc. Viral purification methods
EP2189469B1 (en) 2004-11-18 2015-09-16 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
CA2596588C (en) 2005-01-31 2017-06-27 University Of Iowa Research Foundation Nucleic acid silencing of huntington's disease gene
CN101180394B (zh) 2005-02-03 2013-01-09 贝尼泰克有限公司 RNAi表达构建体
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
WO2006102072A2 (en) 2005-03-23 2006-09-28 The Trustees Of The University Of Pennsylvania Use of a pa131 polypeptide in treatment of atherosclerosis
US8999678B2 (en) 2005-04-07 2015-04-07 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
CN104306986A (zh) 2005-05-02 2015-01-28 建新公司 神经代谢疾病的基因治疗
US20070161591A1 (en) 2005-08-18 2007-07-12 University Of Massachusetts Methods and compositions for treating neurological disease
AU2006283189B2 (en) 2005-08-23 2013-01-31 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
EP2325315B1 (en) * 2005-10-28 2014-05-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of huntingtin gene
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
MX2008012219A (es) 2006-04-03 2008-10-02 Santaris Pharma As Composicion farmaceutica que comprende oligonucleotidos antisentido anti-miarn.
DE602007004470D1 (de) 2006-04-28 2010-03-11 Univ Pennsylvania Modifiziertes adenovirus-hexon-protein und anwendungen davon
JP2009535339A (ja) 2006-04-28 2009-10-01 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 低下されたキャプシド免疫原性を有する改変ａａｖベクターおよびその使用
US9198984B2 (en) 2006-04-28 2015-12-01 The Trustees Of The University Of Pennsylvania Scalable production method for AAV
WO2007130519A2 (en) 2006-05-02 2007-11-15 Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Viral nucleic acid microarray and method of use
WO2007139982A2 (en) 2006-05-25 2007-12-06 Sangamo Biosciences, Inc. Methods and compositions for gene inactivation
CN103849629B (zh) 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
DK2048955T3 (da) 2006-07-21 2013-09-02 California Inst Of Techn Målrettet gen-tilførsel til dendrit-cellevaccination
CN101501193B (zh) * 2006-08-11 2013-07-03 普罗森那技术公司 用于治疗与dna重复不稳定性相关的遗传病的方法和手段
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
CA2670967C (en) 2006-11-29 2016-05-10 University Of Iowa Research Foundation Alternative export pathways for vector expressed rna interference
CA2693178C (en) 2006-11-29 2018-12-04 Nationwide Children's Hospital, Inc. Myostatin inhibition for enhancing muscle and/or improving muscle function
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
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
WO2008128251A1 (en) 2007-04-17 2008-10-23 The Children's Hospital Of Philadelphia Humanized viral vectors and methods of use thereof
US20090036395A1 (en) 2007-04-26 2009-02-05 Davidson Beverly L Rna interference suppression of neurodegenerative diseases and methods of use thereof
US8258286B2 (en) 2007-04-26 2012-09-04 University Of Iowa Research Foundation Reduction of off-target RNA interference toxicity
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
US8841437B2 (en) 2008-06-20 2014-09-23 The Board Of Trustees Of The Leland Stanford Junior University Precursor miRNA loop-modulated target regulation
US9080183B2 (en) 2007-06-29 2015-07-14 Hoffmann-La Roche Inc. Promoter
ES2615180T3 (es) 2007-07-14 2017-06-05 University Of Iowa Research Foundation Métodos y composiciones para el tratamiento de enfermedades cerebrales
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
BRPI0814459B1 (pt) 2007-07-26 2023-01-24 Uniqure Ip B.V Método para produzir um vírion parvoviral recombinante em uma célula de inseto, e, construção de ácido nucleico
JP2010537640A (ja) 2007-08-27 2010-12-09 ボストン バイオメディカル， インコーポレイテッド マイクロｒｎａ模倣剤または阻害剤としての非対称性ｒｎａ二重鎖の組成物
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
WO2009038462A1 (en) 2007-09-19 2009-03-26 Amsterdam Molecular Therapeutics B.V. Use of aav replication machinery for improved protein production
KR101614369B1 (ko) 2007-11-28 2016-04-21 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 유인원 아과 ｃ 아데노바이러스 ｓａｄｖ-40, -31, 및 -34 및 그것의 사용
PL2220242T3 (pl) 2007-11-28 2017-09-29 The Trustees Of The University Of Pennsylvania Podrodziny b adenowirusów małpich sadv-28,27,-29,-32,-33, oraz-35 oraz ich wykorzystanie
US8685387B2 (en) 2007-11-28 2014-04-01 The Trustees Of The University Of Pennsylvania Simian E adenoviruses SAdV-39, -25.2, -26, -30, -37, and -38
CA2713338C (en) 2008-01-29 2021-10-26 Applied Genetic Technologies Corporation Recombinant virus production using mammalian cells in suspension
WO2009104964A1 (en) 2008-02-19 2009-08-27 Amsterdam Molecular Therapeutics B.V. Optimisation of expression of parvoviral rep and cap proteins in insect cells
US8470310B2 (en) 2008-03-04 2013-06-25 The Trustees Of The University Of Pennsylvania Simian adenoviruses SAdV-36, -42.1, -42.2, and -44 and uses thereof
US8632764B2 (en) 2008-04-30 2014-01-21 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
RU2496502C2 (ru) * 2008-05-09 2013-10-27 Маунт Синай Скул Оф Медсин Способы профилактики и лечения нейродегенеративных заболеваний
CA2762118A1 (en) 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US20110171262A1 (en) 2008-06-17 2011-07-14 Andrew Christian Bakker Parvoviral capsid with incorporated gly-ala repeat region
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
WO2010014857A2 (en) 2008-07-30 2010-02-04 University Of Massachusetts Chromosome therapy
AU2009297243B2 (en) 2008-09-29 2015-10-29 Proyecto De Biomedicina Cima S.L. 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
ES2552688T3 (es) 2008-10-31 2015-12-01 The Trustees Of The University Of Pennsylvania Adenovirus de simio con proteínas de la cápside hexónica de SAdV-46 y usos del mismo
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
MX2011009193A (es) 2009-03-04 2011-12-16 Deutsches Krebsforsch Proteina activadora del ensamblaje (aap) y su uso para la fabricacion de particulas de parvovirus que consisten esencialmente de vp3.
CN102448501A (zh) 2009-03-27 2012-05-09 西马生物医学计划公司 治疗肝硬化和肝纤维化的方法和组合物
TR201906398T4 (tr) 2009-04-30 2019-05-21 Univ Pennsylvania Salgı bezleriyle ilişkili virüs yapılarını içeren iletken hava yolu hücrelerinin hedeflenmesine yönelik bileşimler.
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2010136549A2 (en) 2009-05-28 2010-12-02 Deutsches Krebsforschungszentrum Modified aav capsid polypeptides
US8846031B2 (en) 2009-05-29 2014-09-30 The Trustees Of The University Of Pennsylvania Simian adenovirus 41 and uses thereof
SG176283A1 (en) 2009-06-16 2012-01-30 Genzyme Corp Improved methods for purification of recombinant aav vectors
CA2767225A1 (en) 2009-07-06 2011-01-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for enhancing production of a biological product
EP2451476A4 (en) 2009-07-06 2013-07-03 Alnylam Pharmaceuticals Inc BIOTRAITEMENT BASED ON CELLS
RU2015133046A (ru) 2009-07-15 2018-12-24 Кэлиммьюн Инк. Двойной вектор для подавления вируса иммунодефицита человека
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
BR112012010866A2 (pt) 2009-11-09 2016-11-29 Genepod Therapeutics Ab "nova construção de vetor viral para síntese específica otimizada contínua de dopa por neurônios in vivo".
US9493776B2 (en) 2009-11-19 2016-11-15 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
EP2524037B1 (en) 2010-01-12 2018-05-16 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
US9228174B2 (en) 2010-03-11 2016-01-05 Uniqure Ip B.V. Mutated rep encoding sequences for use in AAV production
WO2011117258A2 (en) 2010-03-22 2011-09-29 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
CA2793633A1 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9315825B2 (en) 2010-03-29 2016-04-19 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
US9272053B2 (en) 2010-04-23 2016-03-01 University Of Massachusetts AAV-based treatment of cholesterol-related disorders
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
CA2833905C (en) 2010-04-23 2019-09-10 University Of Massachusetts Multicistronic expression constructs
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
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
WO2012046727A1 (ja) 2010-10-05 2012-04-12 タカラバイオ株式会社 ウイルスベクターの製造方法
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
WO2012064920A1 (en) 2010-11-11 2012-05-18 University Of Miami Methods, compositions, cells, and kits for treating ischemic injury
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
WO2012109667A1 (en) 2011-02-12 2012-08-16 University Of Iowa Research Foundation Therapeutic compounds
HUE043921T2 (hu) 2011-02-17 2019-09-30 Univ Pennsylvania Készítmények és módszerek szövetspecifitás módosítására és AAV9-mediált géntranszfer javítására
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
WO2012144446A1 (ja) 2011-04-18 2012-10-26 独立行政法人国立精神・神経医療研究センター 薬剤送達粒子及びその製造方法
WO2012145572A1 (en) 2011-04-20 2012-10-26 The Trustees Of The University Of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
RS62795B1 (sr) 2011-04-22 2022-02-28 Univ California Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu
WO2012149646A1 (en) 2011-05-05 2012-11-08 Sunnybrook Research Institute Mirna inhibitors and their uses
US9249425B2 (en) 2011-05-16 2016-02-02 The Trustees Of The University Of Pennslyvania Proviral plasmids and 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
EP2718713B1 (en) 2011-06-06 2019-07-17 Koninklijke Philips N.V. Selective lysis of cells by ionic surfactants
US20140275211A1 (en) * 2011-06-21 2014-09-18 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
CA2842392C (en) 2011-07-27 2020-09-15 Genethon Improved baculovirus expression systems
AU2012304993B2 (en) 2011-09-08 2017-09-14 Uniqure Ip B.V. Removal of contaminating viruses from AAV preparations
WO2013036889A1 (en) 2011-09-09 2013-03-14 University Of Washington Retrograde transport peptide and use of same for delivery to central nervous system
EP2771455B1 (en) 2011-10-28 2016-10-05 The University of North Carolina At Chapel Hill Cell line for production of adeno-associated virus
WO2013078400A1 (en) 2011-11-22 2013-05-30 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
AU2013221615B2 (en) 2012-02-14 2018-02-22 The Regents Of The University Of California Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions
ES2752191T3 (es) 2012-02-17 2020-04-03 Childrens Hospital Philadelphia Composiciones y métodos con vectores de AAV para la transferencia de genes a células, órganos y tejidos
AU2013225950B2 (en) 2012-02-29 2018-02-15 Sangamo Therapeutics, Inc. Methods and compositions for treating huntington's disease
MY172457A (en) 2012-04-18 2019-11-26 Childrens Hospital Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
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
JP6178843B2 (ja) 2012-05-08 2017-08-09 メルク・シャープ・アンド・ドーム・コーポレーションＭｅｒｃｋ Ｓｈａｒｐ ＆ Ｄｏｈｍｅ Ｃｏｒｐ． 透過性グリコシダーゼインヒビターおよびその用途
US9677088B2 (en) 2012-05-09 2017-06-13 Oregon Health & Science University Adeno associated virus plasmids and vectors
TWI702955B (zh) 2012-05-15 2020-09-01 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）
BR112014028684A2 (pt) 2012-05-18 2017-07-25 Univ Pennsylvania subfamília e adenovírus de símio a1302, a1320, a1331 e a1337 e usos dos mesmos
EP2871239B1 (en) 2012-07-06 2018-08-29 Takara Bio Inc. Cell capable of producing adeno-associated virus vector
WO2014007858A1 (en) 2012-07-06 2014-01-09 University Of Iowa Research Foundation Modified adeno-associated virus vector compositions
CA2879514C (en) 2012-07-17 2020-04-14 Universite De Geneve Nucleic acids for down-regulation of gene expression
DK2879719T3 (en) 2012-08-01 2018-09-03 Nationwide Childrens Hospital INTRATEKAL ADMINISTRATION OF RECOMBINANT ADENOASSOCATED VIRUSES
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
US9938541B2 (en) 2012-12-25 2018-04-10 Takara Bio Inc. AAV variant
CN105143450A (zh) 2013-01-08 2015-12-09 贝尼泰克生物制药有限公司 年龄相关性黄斑变性治疗
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
JP2016514152A (ja) 2013-03-13 2016-05-19 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア アデノ随伴ウイルスベクターおよびその使用の方法
WO2014143932A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
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
BR112015021884A8 (pt) 2013-03-15 2019-11-26 Childrens Hospital Philadelphia vetores de plasmídeo recombinantes, partícula viral avv ou pluralidade de partículas virais, seus métodos de produção e seus usos, e composição farmacêutica
US9828587B2 (en) 2013-04-08 2017-11-28 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
US10494645B2 (en) 2013-04-18 2019-12-03 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
CA2909807C (en) 2013-04-20 2023-08-08 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted u7snrna polynucleotide constructs
WO2015012924A2 (en) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
SG11201509419QA (en) 2013-05-15 2015-12-30 Univ Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
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
US11136557B2 (en) 2013-05-31 2021-10-05 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
EP3008191A2 (en) 2013-06-13 2016-04-20 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
EP3024498B1 (en) 2013-07-22 2019-12-04 The Children's Hospital of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
EP3024497B1 (en) 2013-07-26 2021-01-13 University of Iowa Research Foundation Methods and compositions for treating brain diseases
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
WO2015031686A1 (en) 2013-08-30 2015-03-05 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
EP3561062A1 (en) 2013-09-13 2019-10-30 California Institute of Technology Selective recovery
US20160354489A1 (en) 2013-09-26 2016-12-08 Universitat Autònome de Barcelona Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
CA3182790A1 (en) 2013-10-11 2015-04-16 Massachusetts Eye & Ear Infirmary Ancestral adeno-associated virus sequences and uses thereof
US20160243260A1 (en) 2013-10-24 2016-08-25 Uniqure Ip B.V. Treatment of neurological diseases using adeno-associated virus (AAV) comprising AAV-5 capsid proteins
EP3066195A4 (en) 2013-11-08 2017-06-28 The Board of Trustees of the University of Arkansas Adeno-associated virus "x" oncogene
CN105934524A (zh) 2013-11-11 2016-09-07 桑格摩生物科学股份有限公司 用于治疗亨廷顿氏病的方法和组合物
JP6649265B2 (ja) 2013-11-26 2020-02-19 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ 糖原病の処置のためのアデノ随伴ウイルスベクター
US10161011B2 (en) 2013-11-29 2018-12-25 Takara Bio Inc. Method for quantifying adeno-associated virus
US20170002350A1 (en) 2013-12-03 2017-01-05 Agency For Science, Technology And Research Tailed mirtron effectors for rnai-mediated gene silencing
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
WO2015106273A2 (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
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
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
US20170007720A1 (en) 2014-02-21 2017-01-12 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
EP3450571B1 (en) 2014-02-24 2023-04-05 Celgene Corporation Methods of using an activator of cereblon for neural cell expansion and the treatment of central nervous system disorders
CA2941640A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
UA120923C2 (uk) 2014-03-10 2020-03-10 Юнікьюре Айпі Б.В. Молекула нуклеїнової кислоти, яка кодує капсидний протеїн aav5
EP4410805A2 (en) 2014-03-18 2024-08-07 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
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
JP6741590B2 (ja) 2014-04-25 2020-08-19 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 コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用
HUE054768T2 (hu) 2014-05-02 2021-09-28 Genzyme Corp AAV vektorok retina és CNS génterápiára
WO2015171932A1 (en) 2014-05-08 2015-11-12 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
US20170088819A1 (en) 2014-05-16 2017-03-30 Vrije Universiteit Brussel Genetic correction of myotonic dystrophy type 1
CN107075514A (zh) 2014-05-20 2017-08-18 衣阿华大学研究基金会 亨廷顿氏病的治疗化合物
EP3160980B1 (en) 2014-05-28 2020-05-20 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
WO2015192063A1 (en) 2014-06-13 2015-12-17 Yasuhiro Ikeda Methods and materials for increasing viral vector infectivity
WO2015196179A1 (en) 2014-06-20 2015-12-23 University Of Florida Research Foundation, Inc. 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
WO2016006658A1 (ja) 2014-07-10 2016-01-14 タカラバイオ株式会社 非エンベロープウイルス粒子の製造方法
WO2016019364A1 (en) 2014-08-01 2016-02-04 The Trustees Of The University Of Pennsylvania Compositions and methods for self-regulated inducible gene expression
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
US10711270B2 (en) 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
EP3209311B1 (en) 2014-10-21 2024-03-06 University of Massachusetts Recombinant aav variants and uses thereof
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
RU2749882C2 (ru) 2014-11-14 2021-06-18 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
EP3218484A4 (en) 2014-11-14 2018-05-30 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
RU2727015C2 (ru) 2014-11-21 2020-07-17 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Векторы aav, нацеленные на центральную нервную систему
EP3221456B1 (en) 2014-11-21 2021-09-22 University of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
PL3237618T3 (pl) 2014-12-24 2019-09-30 Uniqure Ip B.V. Supresja genu huntingtyny indukowana rnai
KR102618947B1 (ko) 2014-12-30 2023-12-27 유니버시티 오브 아이오와 리써치 파운데이션 뇌 질환을 치료하기 위한 방법 및 조성물
WO2016115382A1 (en) 2015-01-14 2016-07-21 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
WO2016115503A1 (en) 2015-01-16 2016-07-21 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
WO2016122791A1 (en) 2015-01-30 2016-08-04 The Regents Of The University Of California Spinal subpial gene delivery system
WO2016126857A1 (en) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
HUE057272T2 (hu) 2015-02-10 2022-04-28 Genzyme Corp Vírusrészecskék fokozott bevitele a csíkolt testbe és az agykéregbe
CA2976075A1 (en) 2015-02-10 2016-08-18 Genzyme Corporation Variant rnai
EP3262162A4 (en) 2015-02-23 2018-08-08 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
JP2018510160A (ja) 2015-03-20 2018-04-12 ブルーバード バイオ， インコーポレイテッド ベクター製剤
JP6836999B2 (ja) 2015-03-24 2021-03-03 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Ｔｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
WO2016161388A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna
PT3277814T (pt) * 2015-04-03 2020-07-28 Univ Massachusetts Compostos de oligonucleótidos para visar marn de huntingtina
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
WO2016168728A2 (en) 2015-04-16 2016-10-20 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
WO2016172155A1 (en) 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
CA3021949C (en) 2015-04-24 2023-10-17 University Of Massachusetts Modified aav constructs and uses thereof
CA2985223A1 (en) 2015-05-07 2016-11-10 Massachusetts Eye And Ear Infirmary Methods of delivering an agent to the eye
EP3294398A4 (en) 2015-05-11 2019-01-09 Alcyone Lifesciences, Inc. SYSTEMS AND METHODS FOR DRUG DELIVERY
EP3294894B8 (en) 2015-05-12 2019-09-25 The U.S.A. 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
WO2016191418A1 (en) 2015-05-26 2016-12-01 Salk Institute For Biological Studies Motor neuron-specific expression vectors
US11027024B2 (en) 2015-05-29 2021-06-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
US11266748B2 (en) 2015-07-02 2022-03-08 University Of Florida Research Foundation, Incorporated 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
ES2895652T3 (es) 2015-07-07 2022-02-22 Inst Nat Sante Rech Med Métodos y composiciones farmacéuticas para expresar un polinucleótido de interés en el sistema nervioso periférico de un sujeto
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
US20180214576A1 (en) 2015-07-28 2018-08-02 University Of Massachusetts Transgenic expression of dnasei in vivo delivered by an adeno-associated virus vector
WO2017019994A2 (en) 2015-07-30 2017-02-02 Massachusetts Eye And Ear Infirmary Ancestral virus sequences and uses thereof
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
WO2017053753A1 (en) 2015-09-23 2017-03-30 Sangamo Biosciences, Inc. Htt repressors and uses thereof
WO2017058892A2 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
CA3001238C (en) 2015-10-09 2024-06-25 Genzyme Corporation Early post-transfection isolation of cells (epic) for biologics production
US11473084B2 (en) 2015-10-09 2022-10-18 The Children's Hospital Of Philadelphia Compositions and methods for treating Huntington's disease and related disorders
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
CA3002980A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
US11253576B2 (en) 2015-10-22 2022-02-22 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
WO2017070476A2 (en) 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
US20180236105A1 (en) 2015-10-23 2018-08-23 University Of Iowa Research Foundation Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection
EP4316512A3 (en) 2015-10-28 2024-04-24 The Trustees of The University of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
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
RU2766583C2 (ru) 2015-12-01 2022-03-15 Спарк Терапьютикс, Инк. Масштабируемые способы получения рекомбинантного вектора на основе аденоассоциированного вируса (aav) в системе бессывороточной суспензионной культуры клеток, подходящего для клинического применения
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
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
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
EP3383896A1 (en) 2015-12-03 2018-10-10 Genethon Compositions and methods for improving viral vector efficiency
WO2017100704A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
WO2017100671A1 (en) 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
ES2934848T3 (es) 2015-12-11 2023-02-27 Univ Pennsylvania Método de purificación escalable para AAV8
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
BR112018011881A2 (pt) 2015-12-14 2018-12-04 The University Of North Carolina At Chapel Hill proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus
CA3011939A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
AU2017229347A1 (en) 2016-03-07 2018-11-01 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
EP3235516B1 (en) 2016-04-22 2019-06-26 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts Universitätsmedizin Regulatable adeno-associated virus (aav) vector
EP3448875A4 (en) 2016-04-29 2020-04-08 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
AU2017261249B2 (en) 2016-05-03 2021-05-06 Children's Medical Research Institute Adeno-associated virus polynucleotides, polypeptides and virions
WO2017192750A1 (en) 2016-05-04 2017-11-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
RU2758488C2 (ru) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
WO2018204803A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
WO2018204797A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2018220211A1 (en) 2017-06-02 2018-12-06 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
AR113134A1 (es) 2017-09-22 2020-01-29 Genzyme Corp Arni variante
EP4124658A3 (en) 2017-10-16 2023-04-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
US20210355454A1 (en) 2018-07-24 2021-11-18 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
WO2021016505A1 (en) 2019-07-24 2021-01-28 Voyager Therapeutics, Inc. Compositions and methods for treating huntington's disease

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