JP2014511180A5 - - Google Patents

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JP2014511180A5
JP2014511180A5 JP2013554679A JP2013554679A JP2014511180A5 JP 2014511180 A5 JP2014511180 A5 JP 2014511180A5 JP 2013554679 A JP2013554679 A JP 2013554679A JP 2013554679 A JP2013554679 A JP 2013554679A JP 2014511180 A5 JP2014511180 A5 JP 2014511180A5
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sequence
virus
antibody
optionally
viral vector
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JP6091435B2 (ja
JP2014511180A (ja
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JP2013554679A 2011-02-22 2012-02-21 アデノ随伴ウイルス(aav)ベクターを用いたタンパク質の送達 Active JP6091435B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201161445449P 2011-02-22 2011-02-22
US61/445,449 2011-02-22
US201161550123P 2011-10-21 2011-10-21
US61/550,123 2011-10-21
US201261598728P 2012-02-14 2012-02-14
US61/598,728 2012-02-14
PCT/US2012/025970 WO2012115980A1 (en) 2011-02-22 2012-02-21 Delivery of proteins using adeno-associated virus (aav) vectors

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JP2014511180A JP2014511180A (ja) 2014-05-15
JP2014511180A5 true JP2014511180A5 (cg-RX-API-DMAC10.html) 2015-04-09
JP6091435B2 JP6091435B2 (ja) 2017-03-08

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US (2) US8865881B2 (cg-RX-API-DMAC10.html)
EP (1) EP2678433B1 (cg-RX-API-DMAC10.html)
JP (1) JP6091435B2 (cg-RX-API-DMAC10.html)
CN (1) CN103492574B (cg-RX-API-DMAC10.html)
BR (1) BR112013021494B1 (cg-RX-API-DMAC10.html)
WO (1) WO2012115980A1 (cg-RX-API-DMAC10.html)

Families Citing this family (160)

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Publication number Priority date Publication date Assignee Title
DE60138535D1 (de) 2000-02-04 2009-06-10 Children S Hospital Res Founda Verwendung von lysosomal acid lipase zur behandlung von atherosklerose und ähnlichen krankheiten
WO2010045659A1 (en) 2008-10-17 2010-04-22 American Gene Technologies International Inc. Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
KR101742346B1 (ko) 2010-04-23 2017-06-15 시나게바 바이오파르마, 코포레이션 리소솜 축적병 효소
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
BR122019021757B1 (pt) 2010-09-09 2020-12-08 Alexion Pharmaceuticals, Inc composição farmacêutica para tratamento de um paciente humano sofrendo de uma deficiência de lipase ácida lisossomal (lal)
EP2857499A1 (en) 2010-10-01 2015-04-08 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2675472A4 (en) 2011-02-15 2014-09-17 Synageva Biopharma Corp METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE
JP6091435B2 (ja) 2011-02-22 2017-03-08 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス(aav)ベクターを用いたタンパク質の送達
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
PT3682905T (pt) 2011-10-03 2022-04-07 Modernatx Inc Nucleósidos, nucleótidos e ácidos nucleicos modificados e respetivas utilizações
ES2923757T3 (es) 2011-12-16 2022-09-30 Modernatx Inc Composiciones de ARNm modificado
WO2013130393A1 (en) * 2012-02-28 2013-09-06 Cornell University Aav-directed persistent expression of an anti-nicotine antibody gene for smoking cessation
WO2013151664A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
US10004811B2 (en) * 2012-04-13 2018-06-26 Cornell University Development of a highly efficient second generation nicotine-conjugate vaccine to treat nicotine addiction
WO2014052693A2 (en) 2012-09-29 2014-04-03 The Trustees Of The University Of Pennsylvania Veterinary composition and methods for non-surgical neutering and castration
EP2914731B1 (en) * 2012-11-01 2019-09-11 California Institute of Technology Reversible gene expression
PT2922554T (pt) 2012-11-26 2022-06-28 Modernatx Inc Arn modificado nas porções terminais
EP2931318A4 (en) * 2012-12-13 2016-07-20 Univ Pennsylvania DNA ANTIBODY CONSTRUCTS AND METHODS FOR USE THEREOF
PL2931304T3 (pl) 2012-12-13 2021-11-08 The Trustees Of The University Of Pennsylvania Szczepionka wt1
WO2014120975A1 (en) 2013-02-01 2014-08-07 California Institute Of Technology Antibody-mediated immunocontraception
US11332719B2 (en) * 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US9597658B2 (en) 2013-04-01 2017-03-21 Savannah River Nuclear Solutions, Llc Metal-organic framework templated synthesis of porous inorganic materials as novel sorbents
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
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
SG11201604719WA (en) 2013-12-13 2016-07-28 Univ Pennsylvania Dna antibody constructs and method of using same
US9884075B2 (en) 2014-01-16 2018-02-06 California Institute Of Technology Domain-swap T cell receptors
US10612041B2 (en) 2014-03-21 2020-04-07 The Board Of Trustees Of The Leland Stanford Junior University Genome editing without nucleases
CR20160519A (es) 2014-05-13 2017-01-27 The Trustees Of The Univ Of Pennysylvania Composiciones que comprenden avv que expresa constructos de anticuerpos duales y sus usos
CN107043774B (zh) * 2014-09-25 2021-05-04 中国人民解放军第二军医大学东方肝胆外科医院 一种嵌合强启动子及其用途
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2016089862A1 (en) 2014-12-01 2016-06-09 The Trustees Of The University Of Pennsylvania Dna antibody constructs and method of using same
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
US11000597B2 (en) * 2015-01-21 2021-05-11 University Of Florida Research Foundation, Incorporated Engineered receptor/ligand system for delivery of therapeutic agents
CN107427666B (zh) 2015-01-30 2022-11-04 加利福尼亚大学校董 脊髓软膜下基因递送系统
CN115094062A (zh) 2015-04-16 2022-09-23 埃默里大学 用于肝脏中蛋白质表达的重组启动子和载体及其用途
SI3288379T1 (sl) 2015-05-01 2022-06-30 Onl Therapeutics, Inc. Peptidne sestave in načini uporabe
CA2984629C (en) 2015-05-07 2024-06-18 University Of South Florida Modified ube3a gene for a gene therapy approach for angelman syndrome
WO2016200543A2 (en) 2015-05-13 2016-12-15 The Trustees Of The University Of Pennsylvania Aav-mediated expression of anti-inluenza antibodies and methods of use thereof
US10016514B2 (en) 2015-05-15 2018-07-10 New Hope Research Foundation Polynucleotides, vectors and methods for insertion and expression of transgenes
US10966414B2 (en) 2015-05-26 2021-04-06 California Institute Of Technology Population control using engineered translocations
US11980663B2 (en) 2015-07-08 2024-05-14 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
WO2017023708A1 (en) 2015-07-31 2017-02-09 California Institute Of Technology Activity-dependent expression of nucleic acids
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
IL297238B2 (en) 2016-01-15 2024-08-01 American Gene Tech Int Inc Methods and preparations for gamma-delta T-cell activation
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
JP7153332B2 (ja) 2016-02-08 2022-10-14 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド Hivワクチン接種および免疫療法
ES2911448T3 (es) 2016-03-09 2022-05-19 American Gene Tech Int Inc Vectores combinados y métodos para el tratamiento del cáncer
US20230279434A1 (en) * 2016-03-31 2023-09-07 University Of Cincinnati Methods and compositions for the treatment of als
IL262207B2 (en) * 2016-04-15 2026-01-01 Univ Pennsylvania Preparations for the treatment of wet age-related macular degeneration
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2017189963A1 (en) * 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017213697A1 (en) 2016-06-08 2017-12-14 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
CA3028982A1 (en) 2016-07-08 2018-01-11 American Gene Technologies International Inc. Hiv pre-immunization and immunotherapy
WO2018017882A1 (en) 2016-07-21 2018-01-25 American Gene Technologies International Inc. Viral vectors for treating parkinson's disease
US20190160187A1 (en) * 2016-07-26 2019-05-30 Cornell University Gene therapy for the treatment of aldehyde dehydrogenase deficiency
CN106434575A (zh) * 2016-09-26 2017-02-22 中国动物卫生与流行病学中心 流感病毒作为表达载体的构建方法
EP3519431A1 (en) 2016-09-28 2019-08-07 Cohbar Inc. Therapeutic mots-c related peptides
WO2018060288A1 (en) 2016-09-29 2018-04-05 Glaxosmithkline Biologicals S.A. Compositions and methods of treatment of persistent hpv infection
CN109996809A (zh) 2016-11-14 2019-07-09 诺华股份有限公司 与促融合蛋白minion相关的组合物、方法和治疗用途
US11564944B2 (en) * 2016-11-21 2023-01-31 Nant Holdings Ip, Llc Fractal combination therapy
JP7118062B2 (ja) 2016-12-09 2022-08-15 グラクソスミスクライン バイオロジカルズ ソシエテ アノニム リサウイルス抗原を有するチンパンジーアデノウイルス構築物
GB201620968D0 (en) 2016-12-09 2017-01-25 Glaxosmithkline Biologicals Sa Adenovirus polynucleotides and polypeptides
CA3087881A1 (en) 2017-01-17 2018-07-26 Michael David FORREST Therapeutic inhibitors of the reverse mode of atp synthase
US11117930B2 (en) 2017-02-23 2021-09-14 Adrx, Inc. Peptide inhibitors of transcription factor aggregation
RS65241B1 (sr) 2017-02-28 2024-03-29 Univ Pennsylvania Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
TW201837170A (zh) 2017-02-28 2018-10-16 賓州大學委員會 新穎aav媒介的流感疫苗
MA47820A (fr) * 2017-03-10 2020-01-15 Genethon Traitement de la glycogénose de type iii
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
CA3059891A1 (en) 2017-04-14 2018-10-18 National Taiwan University Gene therapy for aadc deficiency
WO2018226992A1 (en) 2017-06-07 2018-12-13 Adrx, Inc. Tau aggregation inhibitors
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
US12559769B2 (en) 2017-06-16 2026-02-24 American Gene Technologies International Inc. Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells
CA3068010A1 (en) * 2017-06-20 2018-12-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Codon-optimized human npc1 genes for the treatment of niemann-pick type c1 deficiency and related conditions
CA3068304A1 (en) * 2017-06-28 2019-01-03 University Of South Florida Modified ube3a gene for a gene therapy approach for angelman syndrome
CA3099077A1 (en) 2017-07-13 2019-01-17 Michael David FORREST Therapeutic modulators of the reverse mode of atp synthase
EP3655539A1 (en) 2017-07-21 2020-05-27 GlaxoSmithKline Biologicals S.A. Chikungunya virus antigen constructs
BR112020001060A2 (pt) 2017-07-27 2020-07-14 Novartis Ag variantes de trem2 resistentes a shedase
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
MX2020001593A (es) * 2017-08-09 2020-07-13 Bioverativ Therapeutics Inc Moleculas de acido nucleico y usos de las mismas.
SG11202003058UA (en) 2017-10-16 2020-04-29 Glaxosmithkline Biologicals Sa Replication competent adenoviral vectors
US20200299651A1 (en) 2017-10-16 2020-09-24 Glaxosmithkline Biologicals Sa Simian adenoviral vectors with two expression cassettes
US11859199B2 (en) 2017-10-16 2024-01-02 Glaxosmithkline Biologicals Sa Adenoviral vectors with two expression cassettes encoding RSV antigenic proteins or fragments thereof
MX394509B (es) 2017-10-16 2025-03-12 Glaxosmithkline Biologicals Sa Promotor mejorado
AU2018354195A1 (en) * 2017-10-23 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
US20200283800A1 (en) * 2017-10-23 2020-09-10 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative diseases
CA3086045A1 (en) 2017-12-19 2019-06-27 Akouos, Inc. Aav-mediated delivery of therapeutic antibodies to the inner ear
KR20200101394A (ko) 2017-12-20 2020-08-27 글락소스미스클라인 바이오로지칼즈 에스.에이. 엡스타인-바 바이러스 항원 구축물
CN108103104B (zh) * 2017-12-21 2021-07-06 北京锦篮基因科技有限公司 一种预防和治疗脉络膜新生血管相关眼部疾病的基因药物
JP7569216B2 (ja) 2018-01-17 2024-10-17 アドリーナス セラピューティクス, インコーポレイテッド 21-ヒドロキシラーゼ欠損症のためのアデノ随伴ウイルス遺伝子療法
AU2019221642A1 (en) * 2018-02-14 2020-07-09 Generation Bio Co. Non-viral DNA vectors and uses thereof for antibody and fusion protein production
US11738093B2 (en) 2018-04-09 2023-08-29 Hope For Annabel AAV-mediated delivery of ATP1A3 genes to central nervous system
CA3098807A1 (en) * 2018-05-08 2019-11-14 Neuracle Science Co., Ltd. Adeno-associated virus (aav) delivery of anti-fam19a5 antibodies
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2019222444A2 (en) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Directed evolution
MX2020013553A (es) 2018-06-12 2021-02-26 Glaxosmithkline Biologicals Sa Polinucleotidos y polipeptidos de adenovirus.
WO2020019002A1 (en) * 2018-07-20 2020-01-23 University Of Utah Research Foundation Gene therapy for macular degeneration
JP7602454B2 (ja) 2018-08-09 2024-12-18 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
US11858969B2 (en) 2018-09-18 2024-01-02 California Institute Of Technology Engineered light-sensitive proteins
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3876952A4 (en) 2018-11-05 2022-08-24 American Gene Technologies International Inc. VECTOR SYSTEM FOR EXPRESSING REGULATORY RNA
US11965172B2 (en) 2018-11-05 2024-04-23 California Institute Of Technology DNA sequence modification-based gene drive
US20200148762A1 (en) * 2018-11-09 2020-05-14 Avamab Pharma Inc. Methods for regulating endogenous production of antibodies against infectious diseases
EP3886898A4 (en) 2018-12-01 2022-08-24 International AIDS Vaccine Initiative RECOMBINANT HIV ENV POLYPEPTIDES AND THEIR USE
CN113423419A (zh) * 2019-01-04 2021-09-21 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物
WO2020150065A1 (en) 2019-01-14 2020-07-23 California Institute Of Technology Methods and compositions for modulating appetite and intake of sodium
CA3125770A1 (en) 2019-01-18 2020-07-23 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
SG11202107761SA (en) 2019-01-28 2021-08-30 Cohbar Inc Therapeutic peptides
US12582724B2 (en) 2019-02-20 2026-03-24 Duke University Compositions comprising microbial polypeptides for degrading glycogen and methods for the treatment of glycogen storage diseases
MA55321A (fr) 2019-03-15 2022-01-19 Modernatx Inc Vaccins à base d'arn contre le vih
WO2020243659A1 (en) * 2019-05-31 2020-12-03 University Of Florida Research Foundation, Incorporated Vector-based therapy for thyroid disease
CN113966228A (zh) 2019-06-11 2022-01-21 葛兰素史密斯克莱生物公司 黏膜疫苗配制剂
US20230038502A1 (en) 2019-06-11 2023-02-09 Shire Human Genetic Therapies, Inc. Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein
CN110423778B (zh) * 2019-07-26 2023-04-21 上海科技大学 一种逆行性重组2型腺相关病毒在感染螺旋神经元中的应用
WO2021030745A1 (en) * 2019-08-15 2021-02-18 The Children's Hospital Of Philadelphia Combined transgene and intron-derived mirna therapy for treatment of sca1
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
WO2021081280A1 (en) 2019-10-23 2021-04-29 Shire Human Genetic Therapies, Inc. Adeno-associated virus vectors based gene therapy for hereditary angioedema
CN116096904A (zh) * 2019-12-31 2023-05-09 天鹅生物疗法有限公司 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途
WO2021169167A1 (en) * 2020-02-29 2021-09-02 Nanjing GenScript Biotech Co., Ltd. Method for treating coronavirus infections
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
CN111705069A (zh) * 2020-06-19 2020-09-25 武汉纽福斯生物科技有限公司 一种多神经营养因子联合表达载体及其应用
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
EP4214242A1 (en) * 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized antibodies for anti-viral therapy
WO2022098906A1 (en) * 2020-11-04 2022-05-12 The Trustees Of The University Of Pennsylvania Viral vectors encoding canine insulin for treatment of metabolic diseases in dogs
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
EP4263614A1 (en) 2020-12-16 2023-10-25 Takeda Pharmaceutical Company Limited Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein
US20250325469A1 (en) 2021-01-24 2025-10-23 Michael David FORREST Inhibitors of atp synthase - cosmetic and therapeutic uses
AU2022214529A1 (en) * 2021-02-01 2023-08-03 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of niemann pick type a disease
EP4306131A4 (en) 2021-03-08 2025-06-18 Nanjing GenScript Biotech Co., Ltd. ANTIBODY DELIVERY USING A DUAL VIRAL VECTOR SYSTEM
CA3212876A1 (en) 2021-03-19 2022-09-22 Adrenas Therapeutics, Inc. Gene therapies for 21-hydroxylase deficiency
IL310597A (en) * 2021-08-06 2024-04-01 Univ Pennsylvania Preparations and methods for the treatment and prevention of misfolded proteins
PE20241891A1 (es) * 2021-09-01 2024-09-19 Jaguar Gene Therapy Llc Vectores y viriones adenoasociados para tratar la galactosemia y metodos de uso y fabricacion
CN117535291A (zh) * 2021-09-08 2024-02-09 北京锦篮基因科技有限公司 一种高表达活性的组成型启动子及其应用
AU2022366947A1 (en) 2021-10-12 2024-05-02 Bridgebio Gene Therapy Llc Methods and compositions for treating leukodystrophies
CN114085816B (zh) * 2021-10-26 2023-05-26 四川大学华西医院 一种标记临近星形胶质细胞的方法
CN114107390B (zh) * 2021-11-05 2023-10-24 中国科学院精密测量科学与技术创新研究院 一种用于表达抗体IgG1的rAAV载体及其应用
US20250049955A1 (en) 2021-11-17 2025-02-13 Voyager Therapeutics, Inc. Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
EP4507719A1 (en) 2022-04-13 2025-02-19 Universitat Autònoma de Barcelona Treatment of neuromuscular diseases via gene therapy that expresses klotho protein
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
WO2023248147A1 (en) 2022-06-21 2023-12-28 Crispr Therapeutics Ag Methods and compositions for in vivo editing of stem cells
WO2023248145A1 (en) 2022-06-21 2023-12-28 Crispr Therapeutics Ag Compositions and methods for treating human immunodeficiency virus
CN116396983B (zh) * 2022-10-10 2026-05-26 苏州吉纳星辰生物技术有限公司 一种检测aav抗体滴度的方法
WO2024145474A2 (en) 2022-12-29 2024-07-04 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
AU2023427408A1 (en) 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
US20240398984A1 (en) * 2023-05-22 2024-12-05 Fred Hutchinson Cancer Center Treatment of hsv-2 using a meganuclease
WO2025171001A1 (en) 2024-02-06 2025-08-14 Regeneron Pharmaceuticals, Inc. Compositions and methods for improved production of aav viral vectors
CN118045206B (zh) * 2024-04-12 2024-07-05 四川至善唯新生物科技有限公司 一种治疗脊髓型肌肉萎缩的药物组合物及其用途

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4937190A (en) 1987-10-15 1990-06-26 Wisconsin Alumni Research Foundation Translation enhancer
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
WO2000015260A1 (en) * 1998-09-16 2000-03-23 Tanox, Inc. Anti-ige gene therapy
US7102772B1 (en) 1999-05-25 2006-09-05 Silverbrook Research Pty Ltd Method and system for delivery of a facsimile
AU2001270252B2 (en) * 2000-07-03 2007-02-08 Catalent Pharma Solutions, Llc Expression vectors
US7939059B2 (en) 2001-12-10 2011-05-10 California Institute Of Technology Method for the generation of antigen-specific lymphocytes
JP2006506948A (ja) * 2002-04-09 2006-03-02 チルドレンズ ホスピタル, インコーポレイテッド 抗体遺伝子送達およびそのための組換えaav
CN1761757A (zh) * 2003-01-07 2006-04-19 香港大学 用血管抑制素协同加强腺伴随病毒介导的b7.1免疫接种以根除扩散的肝转移性肿瘤
US7485291B2 (en) * 2003-06-03 2009-02-03 Cell Genesys, Inc. Compositions and methods for generating multiple polypeptides from a single vector using a virus derived peptide cleavage site, and uses thereof
WO2005017149A1 (en) 2003-06-03 2005-02-24 Cell Genesys, Inc. Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site
US7186699B2 (en) * 2003-06-03 2007-03-06 Cell Genesys, Inc. Method for treating cancer by vector-mediated delivery of one or more anti-angiogenic or pro-apoptotic genes
US7407801B2 (en) * 2003-12-05 2008-08-05 University Of Iowa Research Foundation Truncated CMV promoters and vectors containing same
EP1765846A4 (en) * 2004-07-13 2010-02-17 Cell Genesys Inc AAV VECTOR COMPOSITIONS AND ITS USE IN PROCESSES FOR INCREASING IMMUNOGLOBULIN EXPRESSION
CA2584986A1 (en) * 2004-10-22 2006-05-04 Neurologix, Inc. Use of apoptosis inhibiting compounds in degenerative neurological disorders
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
CA2943039C (en) * 2005-10-14 2018-11-06 The Government Of The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services, Centers For Disease Control And Prevention Rabies virus compositions and methods
WO2009120339A2 (en) * 2008-03-24 2009-10-01 President And Fellows Of Harvard College Vectors for delivering disease neutralizing agents
EP2108706A1 (en) * 2008-04-10 2009-10-14 Institut Pasteur New method for selecting compounds useful for treating and/or preventing microbial Shigella infection
US7943379B2 (en) 2008-04-30 2011-05-17 Nationwide Children's Hospital, Inc. Production of rAAV in vero cells using particular adenovirus helpers
ES2566495T3 (es) * 2010-10-15 2016-04-13 Inserm - Institut National De La Santé Et De La Recherche Médicale Vector de expresión para colesterol 24-hidrolasa en terapia de la enfermedad de Huntington
JP6091435B2 (ja) 2011-02-22 2017-03-08 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス(aav)ベクターを用いたタンパク質の送達

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