SG11201909870SA - Compositions and methods of treating amyotrophic lateral sclerosis (als) - Google Patents

Compositions and methods of treating amyotrophic lateral sclerosis (als)

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Publication number
SG11201909870SA
SG11201909870SA SG11201909870SA SG11201909870SA SG 11201909870S A SG11201909870S A SG 11201909870SA SG 11201909870S A SG11201909870S A SG 11201909870SA SG 11201909870S A SG11201909870S A SG 11201909870SA
Authority
SG
Singapore
Prior art keywords
international
methods
als
lateral sclerosis
amyotrophic lateral
Prior art date
Application number
Inventor
Dinah Sah
Qingmin Chen
Jinzhao Hou
Original Assignee
Voyager Therapeutics Inc
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Publication date
Application filed by Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of SG11201909870SA publication Critical patent/SG11201909870SA/en

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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
    • C12N15/1137—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 against enzymes
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    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0089—Oxidoreductases (1.) acting on superoxide as acceptor (1.15)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
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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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    • C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 08 November 2018 (08.11.2018) WIPO I PCT ~ill IIIIIIIIImillion ono o Holonmoinoiliflom oimIE (10) International Publication Number WO 2018/204786 Al (51) International Patent Classification: A61K 31/713 (2006.01) Cl 2N 15/11 (2006.01) A61K 48/00 (2006.01) Cl 2N 15/113 (2010.01) A61P 25/28 (2006.01) Cl 2N 15/86 (2006.01) (21) International Application Number: PCT/US2018/031089 (22) International Filing Date: 04 May 2018 (04.05.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/501,788 05 May 2017 (05.05.2017) US 62/507,927 18 May 2017 (18.05.2017) US 62/520,100 15 June 2017 (15.06.2017) US 62/566,609 02 October 2017 (02.10.2017) US (71) Applicant: VOYAGER THERAPEUTICS, INC. [US/US]; 75 Sidney Street, Cambridge, MA 02139 (US). (72) Inventors: SAH, Dinah, Wen-Yee; 75 Sidney Street, Cam- bridge, MA 02139 (US). CHEN, Qingmin; 75 Sidney Street, Cambridge, MA 02139 (US). HOU, Jinzhao; 75 Sidney Street, Cambridge, MA 02139 (US). MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Published: — with international search report (Art. 21(3)) — before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) with sequence listing part of description (Rule 5.2(a)) Agent: WARD, Donna T.; DT Ward, PC, 142A Main Street, Groton, MA 01824 (US). Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, = (54) Title: COMPOSITIONS AND METHODS OF TREATING AMYOTROPHIC LATERAL SCLEROSIS (ALS) 00 N O 00 O O (57) : The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS). The present invention relates to compositions, methods and processes for the design, preparation, manufacture, use and/or formulation of AAV particles comprising modulatory polynucleotides, e.g., polynucleotides en- codin g at least one small interfering RNA (siRNA) molecules which target the superoxide dismutase 1 (SOD1) gene. Methods for using the AAV particles to inhibit the expression of the SOD1 gene in a subject with a neurodegenerative disease (e.g., amyotrophic lateral sclerosis (ALS)) are also disclosed. 130 110 120
SG11201909870S 2017-05-05 2018-05-04 Compositions and methods of treating amyotrophic lateral sclerosis (als) SG11201909870SA (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762501788P 2017-05-05 2017-05-05
US201762507927P 2017-05-18 2017-05-18
US201762520100P 2017-06-15 2017-06-15
US201762566609P 2017-10-02 2017-10-02
PCT/US2018/031089 WO2018204786A1 (en) 2017-05-05 2018-05-04 Compositions and methods of treating amyotrophic lateral sclerosis (als)

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SG11201909870SA true SG11201909870SA (en) 2019-11-28

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US (2) US11603542B2 (en)
EP (1) EP3618839A4 (en)
JP (2) JP2020518258A (en)
CN (1) CN110913866A (en)
AU (1) AU2018261790A1 (en)
CA (1) CA3061652A1 (en)
SG (1) SG11201909870SA (en)
WO (1) WO2018204786A1 (en)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
KR20220077933A (en) 2014-04-01 2022-06-09 바이오젠 엠에이 인코포레이티드 Compositions for modulating sod-1 expression
WO2016077689A1 (en) * 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
US11499165B2 (en) 2015-12-11 2022-11-15 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
MX2018014154A (en) * 2016-05-18 2019-05-06 Voyager Therapeutics Inc Modulatory polynucleotides.
WO2018039375A1 (en) 2016-08-23 2018-03-01 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
JP2020518258A (en) 2017-05-05 2020-06-25 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． Amyotrophic lateral sclerosis (ALS) treatment composition and method
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
JP2021502060A (en) 2017-10-16 2021-01-28 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． Treatment of amyotrophic lateral sclerosis (ALS)
EP3793686A1 (en) * 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Aav serotypes for brain specific payload delivery
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
CA3103963A1 (en) * 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
EP3826719A1 (en) 2018-07-24 2021-06-02 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
WO2020068990A1 (en) * 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
SG11202103425YA (en) 2018-10-05 2021-05-28 Voyager Therapeutics Inc Engineered nucleic acid constructs encoding aav production proteins
JP2022505106A (en) 2018-10-15 2022-01-14 ボイジャー セラピューティクス インコーポレイテッド Expression vector for large-scale production of rAAV in the baculovirus / Sf9 system
TW202035694A (en) * 2018-12-14 2020-10-01 美商百健Ｍａ公司 Compositions and methods for treating and preventing amyotrophic lateral sclerosis
US20220064671A1 (en) 2019-01-18 2022-03-03 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
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
TW202122582A (en) 2019-08-26 2021-06-16 美商航海家醫療公司 Controlled expression of viral proteins
WO2021168362A1 (en) 2020-02-21 2021-08-26 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
EP4192514A1 (en) 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
EP4301768A2 (en) 2021-03-03 2024-01-10 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022192687A1 (en) * 2021-03-12 2022-09-15 University Of Massachusetts Synthetic promoters for gene therapy and protein expression
WO2023196862A1 (en) * 2022-04-06 2023-10-12 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins

Family Cites Families (493)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5171680A (en) 1988-06-14 1992-12-15 Chiron Corporation Superoxide dismutase analogs having novel binding properties
ATE125566T1 (en) 1988-06-14 1995-08-15 Chiron Corp SUPEROXIDE DISMUTASE ANALOGS WITH NEW BINDING PROPERTIES.
FR2640638B1 (en) 1988-12-20 1991-02-15 Commissariat Energie Atomique BIOREACTOR AND DEVICE FOR THE CULTURE OF ANIMAL CELLS
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
DE69535703T2 (en) 1994-04-13 2009-02-19 The Rockefeller University AAV-mediated delivery of DNA to cells of the nervous system
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US20020159979A1 (en) 1994-06-06 2002-10-31 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
US5688676A (en) 1995-06-07 1997-11-18 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
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
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
US6143548A (en) 1995-08-30 2000-11-07 Genzyme Corporation Chromatographic purification of adeno-associated virus (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
JP4468488B2 (en) 1996-05-29 2010-05-26 コーネル・リサーチ・ファンデーション・インコーポレイテッド Detection of nucleic acid sequence differences using combined ligase detection and polymerase chain reaction
US7026468B2 (en) 1996-07-19 2006-04-11 Valentis, Inc. Process and equipment for plasmid purification
AU4255397A (en) 1996-09-06 1998-03-26 Trustees Of The University Of Pennsylvania, The Chimpanzee adenovirus vectors
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
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US20020037867A1 (en) 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
EP0950111A1 (en) 1996-09-06 1999-10-20 The Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
EP0932418B1 (en) 1996-09-06 2007-12-05 The Trustees Of The University Of Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
WO1998011244A2 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Aav4 vector and uses thereof
ATE348155T1 (en) 1996-11-20 2007-01-15 Introgen Therapeutics Inc AN IMPROVED METHOD FOR PRODUCTION AND PURIFICATION OF ADENOVIRAL VECTORS
US7732129B1 (en) 1998-12-01 2010-06-08 Crucell Holland B.V. Method for the production and purification of adenoviral vectors
EP0950091A2 (en) 1996-12-18 1999-10-20 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
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO1999015677A1 (en) 1997-09-19 1999-04-01 The Trustees Of The University Of Pennsylvania Method for gene transfer using bcl2 and compositions useful therein
AU9319198A (en) 1997-09-19 1999-04-05 Trustees Of The University Of Pennsylvania, The Methods and vector constructs useful for production of recombinant aav
EP1015619A1 (en) 1997-09-19 2000-07-05 The Trustees Of The University Of Pennsylvania 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 (en) 1997-11-21 1999-08-03 Angeletti P Ist Richerche Bio HORMONE-DEPENDENT FORMS OF THE REP PROTEIN OF THE ADENUS ASSOCIATED VIRUS (AAV-2), DNA SEQUENCES CODING FOR THEM, VECTORS THAT
CA2316414A1 (en) 1997-12-23 1999-07-01 Introgene 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 (en) 1998-05-07 2000-08-04 Immunotech Sa NOVEL REAGENTS AND METHOD FOR LYSIS OF ERYTHROCYTES
EP1078096A1 (en) 1998-05-11 2001-02-28 Ariad Gene Therapeutics, Inc. Multiviral compositions and uses thereof
EP1849872A1 (en) 1998-05-20 2007-10-31 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
WO1999061643A1 (en) 1998-05-27 1999-12-02 University Of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient
CA2329143A1 (en) 1998-05-27 1999-12-02 Cell Genesys, Inc. Adeno-associated viral vector-mediated expression of factor viii activity
DE69939169D1 (en) 1998-05-28 2008-09-04 Us Gov Health & Human Serv AAV5 VECTORS AND THEIR USE
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
MXPA01004169A (en) 1998-10-27 2002-06-04 Crucell Holland Bv 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 (en) 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie A method of producing a recombinant adeno-associated virus, suitable compositions therefor, and use for the manufacture of a medicament
US6509150B1 (en) 1999-03-05 2003-01-21 Universite De Nantes Compositions and methods for recombinant Adeno-Associated Virus production
JP4693244B2 (en) 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 Compositions and methods for helperless production of recombinant adeno-associated virus
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 (en) 1999-04-30 2002-12-17 ユニバーシティ オブ フロリダ Adeno-associated virus delivery 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 (en) 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 Compounds and methods for increasing 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
JP2003523320A (en) 1999-09-29 2003-08-05 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 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
EP1224313A1 (en) 1999-10-07 2002-07-24 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
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
CA2392299A1 (en) 1999-12-10 2001-06-14 Victor Rivera Methods for expression of genes in primates
JP2003526377A (en) 2000-03-14 2003-09-09 ニユーロロジクス・インコーポレーテツド Production of chimeric capsid vector
US7638120B2 (en) 2000-03-14 2009-12-29 Thomas Jefferson University High transgene expression of a pseudotyped adeno-associated virus type
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
EP2028278B1 (en) 2000-03-30 2014-03-19 Whitehead Institute For Biomedical Research RNA sequence-specific mediators of RNA interference
GB0009887D0 (en) 2000-04-20 2000-06-07 Btg Int Ltd Cytotoxic agents
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
AU2001257611A1 (en) 2000-04-28 2001-11-12 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
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
DE60117550T2 (en) 2000-06-01 2006-12-07 University Of North Carolina At Chapel Hill DOUBLE-SIDED PARVOVIRUS VECTORS
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
EP1302542B1 (en) 2000-07-18 2007-06-13 Takeda Pharmaceutical Company Limited Novel physiologically active peptide and use thereof
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
US20020111324A1 (en) 2000-08-17 2002-08-15 Keiya Ozawa Adeno-associated virus-mediated delivery of angiogenic factors
DE10044384A1 (en) 2000-09-08 2002-04-18 Medigene Ag Host cells for packaging recombinant adeno-associated virus (rAAV), process for their preparation and their use
FR2813891B1 (en) 2000-09-14 2005-01-14 Immunotech Sa MULTIFUNCTIONAL REAGENT FOR ERYTHROCYTES INVOLVING CARBAMATES AND APPLICATIONS
JP3758164B2 (en) 2000-10-24 2006-03-22 三菱ウェルファーマ株式会社 A therapeutic agent for amyotrophic lateral sclerosis (ALS)
JP2002153278A (en) 2000-11-22 2002-05-28 Hisamitsu Pharmaceut Co Inc Cell for producing virus vector, method for producing the virus vector and method for producing virus vector using the cell
WO2002070719A2 (en) 2001-01-19 2002-09-12 Trustees Of The University Of Pennsylvania Regulatable gene expression system
US20030147853A1 (en) 2001-03-14 2003-08-07 Mcclelland Alan Recombinant adeno-associated virus-mediated gene transfer via retroductal infusion of virions
EP1404698A4 (en) 2001-06-21 2004-12-22 Isis Pharmaceuticals Inc Antisense modulation of superoxide dismutase 1, soluble expression
US20040136963A1 (en) 2001-06-22 2004-07-15 The Trustees Of The University Of Pennsylvania Simian adenovirus vectors and methods of use
ATE530672T1 (en) 2001-06-22 2011-11-15 Univ Pennsylvania RECOMBINANT ADENOVIRUSES WITH MONKEY ADENOVIRUS PROTEINS AND USE THEREOF.
EP1900815B1 (en) 2001-07-12 2016-09-07 University of Massachusetts In vivo production of small interfering RNAs that mediate gene silencing
EP2345720A3 (en) 2001-07-12 2012-01-25 University of Massachusetts In vivo production of small interfering RNAs that mediate gene silencing
AU2007201330C1 (en) 2001-07-12 2011-06-09 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
EP2385123B1 (en) 2001-09-28 2018-04-25 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Microrna molecules
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
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
DE60209193T2 (en) 2001-11-13 2006-09-28 Trustees Of The University Of Pennsylvania Method for identifying adeno-associated virus (AAV) sequences and kit for carrying out the method
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
HU230364B1 (en) 2001-11-21 2016-03-29 The Trustees Of The University Of Pennsylvania Simian adeniovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use
EP1453537A1 (en) 2001-12-12 2004-09-08 FH Faulding & Co. Limited Composition for viral preservation
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
EP2573170B1 (en) 2001-12-17 2017-12-20 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 9 sequences, vectors containing same, and uses therefor
CA2469658A1 (en) 2001-12-19 2003-07-03 Lijun Wang Adeno-associated virus-mediated delivery of gdnf to skeletal muscles
AU2003202026A1 (en) 2002-01-16 2003-09-02 Dynal Biotech Asa Method for isolating nucleic acids and protein from a single sample
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
WO2003093460A1 (en) 2002-04-29 2003-11-13 The Trustees Of The University Of Pennsylvania Method for direct rescue and amplification of integrated viruses from cellular dna of tissues
DE60327069D1 (en) 2002-04-30 2009-05-20 Oncolytics Biotech Inc IMPROVED CLEANING METHOD FOR VIRUSES
CA2483624A1 (en) 2002-05-01 2004-04-01 University Of Florida Research Foundation, Inc. Improved raav expression systems for genetic modification of specific capsid proteins
ES2465574T3 (en) 2002-05-03 2014-06-06 Duke University A method to regulate gene expression
EP1506287B1 (en) 2002-05-14 2007-04-25 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
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
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
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US7897380B2 (en) 2002-08-29 2011-03-01 The Board Of Trustees Of The Leland Stanford Junior University Circular nucleic acid vectors, and methods for making and using the same
AU2003273336A1 (en) 2002-09-18 2004-04-08 Isis Pharmaceuticals, Inc. Efficient reduction of target rna's by single- and double-stranded oligomeric compounds
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
US20060041022A1 (en) 2002-11-06 2006-02-23 Pasinetti Giulio M Treatment of amyotrophic lateral sclerosis with nimesulide
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
ES2440284T3 (en) 2002-11-14 2014-01-28 Thermo Fisher Scientific Biosciences Inc. SiRNA directed to tp53
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
CA2516425A1 (en) 2003-02-21 2005-02-24 The Penn State Research Foundation Rna interference compositions and methods
WO2004075861A2 (en) 2003-02-26 2004-09-10 Children's Hospital, Inc. 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
CN1829736A (en) * 2003-04-10 2006-09-06 希龙公司 The severe acute respiratory syndrome coronavirus
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
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
EP2781596A1 (en) 2003-05-21 2014-09-24 Genzyme Corporation Methods for producing prepartions of recombinant AAVvirions substantially free of empty capsids
ES2712695T3 (en) 2003-06-02 2019-05-14 Univ Massachusetts Methods and compositions to control the efficiency of RNA silencing
US8309704B2 (en) 2003-06-02 2012-11-13 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNAi
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
JP4888876B2 (en) 2003-06-13 2012-02-29 田平 武 Recombinant adeno-associated virus vector for the treatment of Alzheimer's disease
PL2657247T3 (en) 2003-06-19 2017-09-29 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
EP1636370B1 (en) 2003-06-20 2014-04-16 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
WO2005007875A2 (en) 2003-07-18 2005-01-27 University Of Massachusetts Enhanced promoters for synthesis of small hairpin rna
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
EP1668369B1 (en) 2003-08-20 2016-01-06 ProMIS Neurosciences Inc. Epitope protection assay and method for detecting protein conformations
US7947658B2 (en) 2003-09-12 2011-05-24 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
PT2292780T (en) 2003-09-30 2017-11-28 Univ Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses thereof
US7962316B2 (en) 2003-10-27 2011-06-14 Merck Sharp & Dohme Corp. Method of designing siRNAs for gene silencing
WO2005062937A2 (en) 2003-12-22 2005-07-14 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended sirna
DE602005027820D1 (en) 2004-03-05 2011-06-16 Benitec Inc MULTIPLE PROMOTER EXPRESSION CASSETTES FOR THE SIMULTANEOUS INTRODUCTION OF RNAI AGENTS
WO2005096781A2 (en) 2004-04-06 2005-10-20 University Of Massachusetts Methods and compositions for treating gain-of-function disorders using rna interference
CA2563538C (en) 2004-04-28 2013-12-17 The Trustees Of The University Of Pennsylvania Sequential delivery of immunogenic molecules via adenovirus and adeno-associated virus-mediated administrations
EP1751275B1 (en) 2004-06-01 2017-08-16 Avigen, Inc. Compositions and methods to prevent aav vector aggregation
EP1807009B1 (en) 2004-10-05 2014-11-26 Genzyme Corporation Stepped cannula
US7901921B2 (en) 2004-10-22 2011-03-08 Oncolytics Biotech Inc. Viral purification methods
CA2587854C (en) 2004-11-18 2014-01-21 The Board Of Trustees Of The University Of Illinois Multicistronic sirna constructs to inhibit tumors
CN1286981C (en) 2004-11-30 2006-11-29 华中科技大学同济医学院附属同济医院 Combination adeno-associated virus of expression human CYP2J2 antigene and its preparation method
AU2005316476A1 (en) 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
US20060229268A1 (en) 2004-12-16 2006-10-12 Alsgen, Llc Small interference RNA (siRNA) molecules for modulating superoxide dismutase (SOD)
JP4292416B2 (en) 2005-01-12 2009-07-08 住友電気工業株式会社 Superconducting cable terminal structure
CA2596711C (en) 2005-02-03 2015-12-15 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
WO2006102072A2 (en) 2005-03-23 2006-09-28 The Trustees Of The University Of Pennsylvania Use of a pa131 polypeptide in treatment of atherosclerosis
DE23193601T1 (en) 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania METHOD FOR INCREASE THE FUNCTION OF AN AAV VECTOR
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
PT3058959T (en) 2005-05-02 2019-05-24 Genzyme Corp Gene therapy for neurometabolic disorders
EP1937066A4 (en) 2005-08-18 2008-12-24 Alnylam Pharmaceuticals Inc Methods and compositions for treating neurological disease
JP5238503B2 (en) 2005-08-23 2013-07-17 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 Anti-reflux cannula and system for chronic delivery of therapeutic agents using enhanced convection delivery
EP1857552A1 (en) 2006-05-20 2007-11-21 Cargill Incorporated Thermostable xylose isomerase enzyme
WO2007046703A2 (en) 2005-10-20 2007-04-26 Amsterdam Molecular Therapeutics B.V. Improved aav vectors produced in insect cells
US7887803B2 (en) 2005-12-02 2011-02-15 Amorfix Life Sciences Methods and compositions to treat misfolded-SOD1 mediated diseases
US7794692B2 (en) 2005-12-02 2010-09-14 Amorfix Life Sciences Ltd. Methods and compositions for detecting amyotrophic lateral sclerosis
WO2007089607A2 (en) 2006-01-26 2007-08-09 University Of Massachusetts Rna silencing agents for use in therapy and nanotransporters for efficient delivery of same
WO2007089632A2 (en) 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
WO2008016391A2 (en) 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
DK2514823T3 (en) 2006-03-03 2018-07-23 Promis Neurosciences Inc Methods and compositions for treating and detecting failed SOD1-mediated diseases
EP1993611A4 (en) 2006-03-16 2013-05-22 Alnylam Pharmaceuticals Inc RNAi MODULATION OF TGF-BETA AND THERAPEUTIC USES THEREOF
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
WO2008010864A2 (en) 2006-04-28 2008-01-24 The Trustees Of The University Of Pennsylvania Modified adenovirus hexon protein and uses thereof
CN101495624A (en) 2006-04-28 2009-07-29 宾夕法尼亚州立大学托管会 Modified AAV vectors having reduced capsid immunogenicity and use thereof
ES2400235T3 (en) 2006-04-28 2013-04-08 The Trustees Of The University Of Pennsylvania AAV scalable production method
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
AU2007253909B2 (en) 2006-05-05 2012-08-09 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of GCCR
CA2651499C (en) 2006-05-25 2015-06-30 Sangamo Biosciences, Inc. Methods and compositions for ccr-5 gene inactivation
CN101506369B (en) 2006-06-21 2014-02-12 尤尼克尔生物制药股份有限公司 Vectors with modified initiation codon for translation of AAV-REP78 useful for production of AAV in insect cells
SG173337A1 (en) 2006-07-21 2011-08-29 California Inst Of Techn Targeted gene delivery for dendritic cell vaccination
ES2385679T3 (en) 2006-08-24 2012-07-30 Virovek, Inc. Expression of gene insect cells with overlapping open reading frames, methods and compositions thereof
PT2066791E (en) 2006-10-03 2012-12-05 Genzyme Corp Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
US7794443B2 (en) 2006-10-24 2010-09-14 Medtronic, Inc. System and method for intraparenchymal drug infusion
EP2799547B1 (en) 2006-11-08 2016-12-21 Veritas Bio, LLC In Vivo Delivery of RNA to a Target Cell
WO2008067480A2 (en) 2006-11-29 2008-06-05 Nationwide Children's Hospital Myostatin inhibition for enhancing muscle and/or improving muscle function
US8227592B2 (en) 2006-11-29 2012-07-24 University Of Iowa Research Foundation Alternative export pathways for vector expressed RNA interference
WO2008086079A2 (en) 2007-01-03 2008-07-17 Medtronic, Inc. Compositions comprising rnai and a neurotrophic factor and uses thereof
US8173614B2 (en) 2007-01-03 2012-05-08 Medtronic, Inc. Therapeuting compositions comprising an RNAi agent and a neurotrophic factor and methods of use thereof
NZ578816A (en) 2007-02-02 2012-06-29 Biogen Idec 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
ES2714007T3 (en) 2007-04-09 2019-05-24 Univ Florida Compositions of rAAV vectors that have tyrosine modified capsid proteins and methods for their use
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
US20100203083A1 (en) 2007-05-31 2010-08-12 Medigene Ag Mutated structural protein of a parvovirus
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
WO2008150897A2 (en) 2007-05-31 2008-12-11 University Of Iowa Research Foundation Reduction of off-target rna interference toxicity
US8841437B2 (en) 2008-06-20 2014-09-23 The Board Of Trustees Of The Leland Stanford Junior University Precursor miRNA loop-modulated target regulation
BRPI0813003A2 (en) 2007-06-29 2014-10-14 Hoffmann La Roche DISTRICT ATTORNEY
CA2728571A1 (en) 2007-06-29 2009-01-08 Boston Biomedical, Inc. Bacteria-mediated gene modulation via microrna machinery
EP3121190A1 (en) 2007-07-14 2017-01-25 University of Iowa Research Foundation 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
CN101868547B (en) 2007-07-26 2016-03-16 尤尼克尔Ip股份有限公司 Comprise the baculovirus vector of the repeated encoding sequence with difference codon bias
EP2193140B1 (en) 2007-08-27 2016-11-02 1Globe Health Institute LLC Compositions of asymmetric interfering rna and uses thereof
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 (en) 2007-09-19 2013-10-21 Uniqure Ip B.V. Use of AAV replication machinery for improved protein production
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
BRPI0819783A2 (en) 2007-11-28 2015-06-23 Univ Pennsylvania Simian subfamily and adenovirus sadv-39, -25.2, -26, -30, -37 and -38 and uses thereof.
CA2707029A1 (en) 2007-11-28 2009-08-27 Soumitra Roy Simian subfamily c adenoviruses sadv-40, -31, and -34 and uses thereof
PL2220242T3 (en) 2007-11-28 2017-09-29 The Trustees Of The University Of Pennsylvania Simian subfamily b adenoviruses sadv-28,27,-29,-32,-33, and -35 and uses thereof
US8480626B2 (en) 2007-11-30 2013-07-09 Medtronic, Inc. Infusion catheter assembly with reduced backflow
WO2009126350A2 (en) 2008-01-18 2009-10-15 Genentech, Inc. Methods and compositions for targeting polyubiquitin
ES2751999T3 (en) 2008-01-29 2020-04-02 Applied Genetic Tech Corporation Recombinant production of adeno-associated viruses using suspension BHK cells
US10131904B2 (en) 2008-02-11 2018-11-20 Rxi Pharmaceuticals Corporation Modified RNAi polynucleotides and uses thereof
ES2929031T3 (en) 2008-02-19 2022-11-24 Uniqure Ip Bv Optimization of parvoviral rep and cap protein expression in insect cells
CN102016011B (en) 2008-03-04 2013-12-11 宾夕法尼亚大学托管会 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
WO2010011404A2 (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
US8951979B2 (en) 2008-06-13 2015-02-10 Cornell University Pain treatment using ERK2 inhibitors
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
JP5328244B2 (en) 2008-07-07 2013-10-30 株式会社ニュージェン・ファーマ A therapeutic agent for amyotrophic lateral sclerosis
CA2731779A1 (en) 2008-07-24 2010-01-28 Rxi Pharmaceuticals Corporation Rnai constructs and uses therof
US8212019B2 (en) 2008-07-30 2012-07-03 University Of Massachusetts Nucleic acid silencing sequences
AU2009293658A1 (en) 2008-09-22 2010-03-25 James Cardia Reduced size self-delivering RNAi compounds
MX2011003229A (en) 2008-09-29 2011-09-06 Proyecto Biomedicina Cima Sl Porphobilinogen deaminase gene therapy.
JP5851838B2 (en) 2008-10-22 2016-02-03 ジェネンテック， インコーポレイテッド Regulation of axonal degeneration
US8940290B2 (en) 2008-10-31 2015-01-27 The Trustees Of The University Of Pennsylvania Simian adenoviruses SAdV-43, -45, -46, -47, -48, -49, and -50 and uses thereof
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
BRPI1013364A2 (en) 2009-03-04 2016-03-29 Deutsches Krebsforsch assembly activating protein (aap) and its use for the manufacture of parvovirus particles consisting essentially of vp3.
MX2011010097A (en) 2009-03-27 2011-10-19 Proyecto Biomedicina Cima Sl Methods and compositions for the treatment of cirrhosis and liver fibrosis.
ES2724122T3 (en) 2009-04-30 2019-09-06 Univ Pennsylvania Compositions for directing conductive 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
CN102575232B (en) 2009-05-29 2015-07-22 宾夕法尼亚大学托管会 Simian adenovirus 41 and uses thereof
SG176283A1 (en) 2009-06-16 2012-01-30 Genzyme Corp Improved methods for purification of recombinant aav vectors
EP2451823A4 (en) 2009-07-06 2013-07-03 Alnylam Pharmaceuticals Inc Compositions and methods for enhancing production of a biological product
JP5828838B2 (en) 2009-07-15 2015-12-09 カリミューン， インコーポレーティッド Dual vector for human immunodeficiency virus inhibition
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
EP2498825B1 (en) 2009-11-09 2017-03-29 Genepod Therapeutics Ab Novel viral vector construct for neuron specific continuous dopa synthesis in vivo
AU2010322723B9 (en) 2009-11-19 2015-03-05 Momotaro-Gene Inc. 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
ES2683695T3 (en) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Inverse restrictive terminal repeats for viral vectors
US9169492B2 (en) 2010-02-05 2015-10-27 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
US9163261B2 (en) 2010-02-22 2015-10-20 Koteswara Rao KOLLIPARA 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
WO2011117258A2 (en) 2010-03-22 2011-09-29 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
SG10201502270TA (en) 2010-03-29 2015-05-28 Univ 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
EP3444346B1 (en) 2010-04-23 2022-07-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
CA3066596A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US9839696B2 (en) 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US9856210B2 (en) 2010-09-02 2018-01-02 Kyoto University Pharmaceutical composition for prevention and treatment of amyotrophic lateral sclerosis
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 (en) 2010-10-05 2012-04-12 タカラバイオ株式会社 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
CN107828820B (en) 2010-10-27 2022-06-07 学校法人自治医科大学 Adeno-associated virus particles for gene transfer into nervous system cells
US20130236433A1 (en) 2010-11-11 2013-09-12 Keith A. Webster 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
SG10202007803QA (en) 2011-02-17 2020-09-29 Univ Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
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 (en) 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 Drug delivery particle and method for producing the same
EP3699286A1 (en) 2011-04-20 2020-08-26 The Trustees of the University of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
SG194583A1 (en) 2011-04-22 2013-12-30 Univ California Adeno-associated virus virions with variant capsid and methods of use thereof
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
CA2831969A1 (en) 2011-06-06 2012-12-30 Biocartis S.A. Selective lysis of cells by ionic surfactants
US20130019580A1 (en) 2011-07-20 2013-01-24 Anderson Noel W Bidirectional harvesting system
IN2014CN00688A (en) 2011-07-27 2015-04-03 Genethon
US8852911B2 (en) 2011-08-04 2014-10-07 The Regents Of The University Of California Method of producing dicer
US9840694B2 (en) 2011-09-08 2017-12-12 Uniqure Ip B.V. Removal of contaminating viruses from AAV preparations
US9056892B2 (en) 2011-09-09 2015-06-16 University Of Washington Retrograde transport peptide and use of same for delivery to central nervous system
CN104520421B (en) 2011-10-28 2017-05-24 北卡罗来纳-查佩尔山大学 Cell line for production of adeno-associated virus
US10640785B2 (en) 2011-11-22 2020-05-05 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
JP2013143917A (en) 2012-01-13 2013-07-25 Mie Univ Preventive or therapeutic agent for fibrosis
HUE036640T2 (en) 2012-02-14 2018-07-30 Univ California Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions
MX367100B (en) 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Aav vector compositions and methods for gene transfer to cells, organs and tissues.
SG11201406776TA (en) 2012-04-18 2015-03-30 Philadelphia Children Hospital Composition and methods for highly efficient gene transfer using aav capsid variants
US20140162319A2 (en) 2012-05-02 2014-06-12 Sangeetha Hareendran Nucleotide sequences, methods, kit and a recombinant cell thereof
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
WO2013170078A1 (en) 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
TWI775096B (en) 2012-05-15 2022-08-21 澳大利亞商艾佛蘭屈澳洲私營有限公司 Treatment of amd using aav sflt-1
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
CN105473723A (en) 2012-05-18 2016-04-06 宾夕法尼亚大学托管会 Subfamily E simian adenoviruses A1302, A1320, A1331 and A1337 and uses thereof
US9539259B2 (en) 2012-05-23 2017-01-10 The Johns Hopkins University Compounds and methods of use thereof for treating neurodegenerative disorders
IN2015KN00074A (en) 2012-07-06 2015-07-31 Univ Iowa Res Found
CN104603272B (en) 2012-07-06 2017-11-07 宝生物工程株式会社 The cell of adeno-associated virus vector can be produced
US9556433B2 (en) 2012-07-17 2017-01-31 Université De Genève Nucleic acids for down-regulation of gene expression
WO2014022582A1 (en) 2012-08-01 2014-02-06 Nationwide Children's Hospital, Inc. Intrathecal delivery of recombinant adeno-associated virus 9
JP2015529685A (en) 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 Compositions and methods for the treatment of amyotrophic lateral sclerosis
CN104797593B (en) 2012-09-28 2020-05-08 北卡罗来纳-查佩尔山大学 AAV vectors targeting oligodendrocytes
US20140243783A1 (en) 2012-10-06 2014-08-28 Raghu Raghavan Method of backflow reduction during material delivery through a needle into tissue
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
EP2914728B1 (en) 2012-11-01 2020-07-08 Factor Bioscience Inc. Methods and products for expressing proteins in cells
US20140179770A1 (en) 2012-12-12 2014-06-26 Massachusetts Institute Of Technology Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
WO2014103957A1 (en) 2012-12-25 2014-07-03 タカラバイオ株式会社 Aav variant
RU2015133252A (en) 2013-01-08 2017-02-10 Бенитек Байофарма Лимитед TREATMENT OF AGE-RELATED MACULAR DEGENERATION
ES2741502T3 (en) 2013-02-08 2020-02-11 Univ Pennsylvania AAV8 capsid modified for gene transfer for retinal therapies
JP2016514152A (en) 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 Adeno-associated virus vector and method 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
EP3517612A1 (en) 2013-03-15 2019-07-31 The University of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
EP2970943A4 (en) 2013-03-15 2017-04-19 The Children's Hospital of Philadelphia Vectors comprising stuffer/filler polynucleotide sequences and methods of use
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
US9827295B2 (en) 2013-05-15 2017-11-28 Regents Of The University Of Minnesota Methods to treat mucopolysaccharide type I or deficiency in alpha-L-iduronidase using a recombinant adeno-associated virus encoding alpha-L-iduronidase
US10006049B2 (en) 2013-05-16 2018-06-26 University Of Florida Research Foundation, Incorporated Hairpin mRNA elements and methods for the regulation of protein translation
CN105247044B (en) 2013-05-31 2021-05-07 加利福尼亚大学董事会 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
KR20220041963A (en) 2013-07-22 2022-04-01 더 칠드런스 호스피탈 오브 필라델피아 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 (en) 2013-08-05 2015-02-06 Consiglio Nazionale Ricerche ADENO-ASSOCIATED MOMCULAR-SPECIFIC VECTOR AND ITS EMPLOYMENT IN THE TREATMENT OF MUSCLE PATHOLOGIES
KR20160106040A (en) 2013-08-14 2016-09-09 퀴아젠 맨스필드, 인코퍼레이티드 Compositions and methods for multimodal analysis of cmet nucleic acids
PL3702466T3 (en) 2013-08-27 2023-04-24 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
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
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
EP3049527A4 (en) 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
EP3744730A1 (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
JP6649265B2 (en) 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 Adeno-associated virus vector for treatment of glycogen disease
WO2015080223A1 (en) 2013-11-29 2015-06-04 タカラバイオ株式会社 Method for quantifying adeno-associated virus
WO2015084254A1 (en) 2013-12-03 2015-06-11 Agency For Science, Technology And Research Tailed Mirtron Effectors For RNAi-Mediated Gene Silencing
DK3080269T3 (en) 2013-12-09 2019-07-08 Baylor College Medicine HIPPO AND DYSTROPHIN COMPLEX SIGNALING BY CARDIOMYOCYTY RENEW
JP6712948B2 (en) 2013-12-12 2020-06-24 ザ・ブロード・インスティテュート・インコーポレイテッド Compositions and methods of using the CRISPR-cas system in nucleotide repeat disorders
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
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
WO2015134643A1 (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
CA2942289A1 (en) 2014-03-10 2015-09-17 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
AU2015231294B2 (en) 2014-03-18 2020-10-29 University Of Massachusetts rAAV-based compositions and methods for treating amyotrophic lateral sclerosis
KR20220077933A (en) * 2014-04-01 2022-06-09 바이오젠 엠에이 인코포레이티드 Compositions for modulating sod-1 expression
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
JP6741590B2 (en) 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 LDLR variants for lowering cholesterol levels and their use in compositions
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
US9194250B1 (en) 2014-05-07 2015-11-24 General Electric Company Embedded wireless sensors for turbomachine component defect monitoring
CN106413760B (en) 2014-05-08 2020-01-14 桑格摩生物科学股份有限公司 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 (en) 2014-05-20 2017-08-18 衣阿华大学研究基金会 The therapeutic compounds of Huntington's disease
EP3160980B1 (en) 2014-05-28 2020-05-20 The Regents of the University of California HYBRID tRNA/pre-miRNA MOLECULES AND METHODS OF USE
EP3151866B1 (en) 2014-06-09 2023-03-08 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
WO2016004319A1 (en) 2014-07-02 2016-01-07 University Of Florida Research Foundation, Inc. Compositions and methods for purifying recombinant adeno-associated virus
EP3168298B1 (en) 2014-07-10 2019-04-17 Takara Bio Inc. Production method for non-enveloped virus particles
ES2806087T3 (en) 2014-07-31 2021-02-16 Association Inst De Myologie Treatment of amyotrophic lateral sclerosis
US10392622B2 (en) 2014-08-01 2019-08-27 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
JP6842410B2 (en) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ AAV vector identified by a novel high efficiency library
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
US10842886B2 (en) 2014-10-10 2020-11-24 Research Institute At Nationwide Children's Hospital Guided injections for AAV gene transfer to muscle
RU2020140209A (en) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс RECOMBINANT AAV OPTIONS AND THEIR APPLICATIONS
WO2016077607A1 (en) 2014-11-13 2016-05-19 The Regents Of The University Of California Adjustable stepped cannula
CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2016077689A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2016081811A1 (en) 2014-11-21 2016-05-26 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
US10900053B2 (en) 2014-11-21 2021-01-26 University Of Florida Research Foundation, Incorporated Genome-modified recombinant adeno-associated virus vectors
ES2751919T5 (en) 2014-11-28 2023-10-04 Uniqure Ip Bv DNA impurities in a composition comprising a parvoviral virion
JP6938377B2 (en) 2015-01-14 2021-09-22 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル Methods and compositions for targeting gene transfer
CA2970730A1 (en) 2015-01-16 2016-07-21 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
JP2018506530A (en) 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 Spinal subpuffy 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
JP6928558B2 (en) 2015-02-10 2021-09-01 ジェンザイム・コーポレーション Enhanced delivery of viral particles to the striatum and cortex
WO2016137949A1 (en) 2015-02-23 2016-09-01 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
WO2016154055A1 (en) 2015-03-20 2016-09-29 Bluebird Bio, Inc. Vector formulations
CN107532177A (en) 2015-03-24 2018-01-02 加利福尼亚大学董事会 Adeno-associated virus variant and its application method
US9809817B2 (en) 2015-04-03 2017-11-07 University Of Massachusetts Oligonucleotide compounds for targeting huntingtin mRNA
JP6892433B2 (en) 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 Well-stabilized asymmetric 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 (en) 2015-04-08 2020-10-21 美商健臻公司 Production of oversized adeno-associated vectors
CN115074366A (en) 2015-04-16 2022-09-20 埃默里大学 Recombinant promoter and vector for protein expression in liver and application thereof
CA3019315A1 (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
US20160346359A1 (en) 2015-05-01 2016-12-01 Spark Therapeutics, Inc. Adeno-associated Virus-Mediated CRISPR-Cas9 Treatment of Ocular Disease
CA2985223A1 (en) 2015-05-07 2016-11-10 Massachusetts Eye And Ear Infirmary Methods of delivering an agent to the eye
JP6993234B2 (en) 2015-05-11 2022-01-13 アルキオーネ・ライフサイエンシズ・インコーポレイテッド Drug delivery system and method
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
US10729790B2 (en) 2015-05-26 2020-08-04 Salk Institute For Biological Studies Motor neuron-specific expression vectors
IL255912B2 (en) 2015-05-29 2023-10-01 Univ Pennsylvania Compositions and methods for degradation of misfolded 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
US20170009304A1 (en) 2015-07-07 2017-01-12 Splicingcodes.Com Method and kit for detecting fusion transcripts
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
US20180216133A1 (en) 2015-07-17 2018-08-02 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
WO2017019876A1 (en) 2015-07-28 2017-02-02 University Of Massachusetts Transgenic expression of dnase i in vivo delivered by an adeno-associated virus vector
US10738087B2 (en) 2015-07-30 2020-08-11 Massachusetts Eye And Ear Infirmary Ancestral virus sequences and uses thereof
WO2017024111A1 (en) 2015-08-04 2017-02-09 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
IL299901A (en) 2015-09-28 2023-03-01 Univ Florida Methods and compositions for antibody-evading virus vectors
MX2018004366A (en) 2015-10-09 2018-08-01 Genzyme Corp 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
WO2017070476A2 (en) 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
EP3364996B1 (en) 2015-10-22 2021-08-25 University of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
WO2017070525A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
JP7190352B2 (en) 2015-10-23 2022-12-15 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 Methods of treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
EP3371217A1 (en) 2015-11-08 2018-09-12 H. Hoffnabb-La Roche Ag Methods of screening for multispecific antibodies
WO2017083423A1 (en) 2015-11-10 2017-05-18 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for modulating aav infection
SG10202106287YA (en) 2015-12-01 2021-07-29 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
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
WO2017093330A1 (en) 2015-12-03 2017-06-08 Genethon Compositions and methods for improving viral vector efficiency
US11499165B2 (en) 2015-12-11 2022-11-15 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
EP3387118B1 (en) 2015-12-11 2022-04-06 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
US11015174B2 (en) 2015-12-11 2021-05-25 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
EP3403675B1 (en) 2016-01-15 2021-04-21 Jichi Medical University Adeno-associated virus virion for use in treatment of epilepsy
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
DK3411484T3 (en) 2016-02-05 2023-11-20 Univ Emory INJECTION OF SINGLE-STRANDED OR SELF-COMPLEMENTARY ADENO-ASSOCIATED VIRUS 9 INTO THE CEREBROSPINAL FLUID
EP3426787A1 (en) 2016-03-07 2019-01-16 University of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
EP3429605A4 (en) 2016-03-18 2019-09-18 The Children's Hospital of Philadelphia Therapeutic for treatment of diseases including the central nervous system
JP7366388B2 (en) 2016-03-28 2023-10-23 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 Anti-Ryk antibody and its use
US11091776B2 (en) 2016-04-15 2021-08-17 The Trustees Of The University Of Pennsylvania AAV8 mutant capsids and compositions containing same
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 the treatment of disease
EP3452495B1 (en) 2016-05-03 2021-06-23 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
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
MX2018014154A (en) 2016-05-18 2019-05-06 Voyager Therapeutics Inc Modulatory polynucleotides.
AU2018260998A1 (en) 2017-05-05 2019-11-28 Voyager Therapeutics, Inc. Modulatory polynucleotides
JP2020518258A (en) 2017-05-05 2020-06-25 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． Amyotrophic lateral sclerosis (ALS) treatment composition and method
US20220090127A1 (en) 2017-06-02 2022-03-24 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
CA3071978A1 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
JP2021502060A (en) 2017-10-16 2021-01-28 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． Treatment of amyotrophic lateral sclerosis (ALS)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CA3103963A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
WO2020010035A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Cannula system
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2020223296A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord

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