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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JP2013554679A
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JP2014511180A (ja
JP6091435B2 (ja
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Priority claimed from PCT/US2012/025970 external-priority patent/WO2012115980A1/en
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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 (enExample) 2015-04-09
JP6091435B2 JP6091435B2 (ja) 2017-03-08

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US (2) US8865881B2 (enExample)
EP (1) EP2678433B1 (enExample)
JP (1) JP6091435B2 (enExample)
CN (1) CN103492574B (enExample)
BR (1) BR112013021494B1 (enExample)
WO (1) WO2012115980A1 (enExample)

Families Citing this family (156)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2328446T5 (es) 2000-02-04 2014-02-27 Children's Hospital Research Foundation Uso de lipasa ácida lisosomal para tratar la aterosclerosis y enfermedades asociadas
US20100183558A1 (en) 2008-10-17 2010-07-22 Zhennan Lai Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
MX338426B (es) 2010-04-23 2016-04-15 Synageva Biopharma Corp Enzima de enfermedad del almacenamiento lisosomico.
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
DK2613798T4 (en) 2010-09-09 2018-04-16 Synageva Biopharma Corp USING LYSOSOMAL SUR LIPASE TO TREAT PATIENTS WITHOUT LYSOSOMAL SUR LIPASE
ES3005233T3 (en) 2010-10-01 2025-03-14 Modernatx Inc Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
EP2675472A4 (en) 2011-02-15 2014-09-17 Synageva Biopharma Corp METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE
US8865881B2 (en) 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
JP2014511687A (ja) 2011-03-31 2014-05-19 モデルナ セラピューティクス インコーポレイテッド 工学操作された核酸の送達および製剤
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2763701B1 (en) 2011-10-03 2018-12-19 Moderna Therapeutics, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
JP2015501844A (ja) 2011-12-16 2015-01-19 モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. 修飾ヌクレオシド、ヌクレオチドおよび核酸組成物
US10093947B2 (en) * 2012-02-28 2018-10-09 Cornell University AAV-directed persistent expression of an anti-nicotine antibody gene for smoking cessation
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
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
WO2013151663A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
US10004811B2 (en) * 2012-04-13 2018-06-26 Cornell University Development of a highly efficient second generation nicotine-conjugate vaccine to treat nicotine addiction
EP2900275A4 (en) 2012-09-29 2016-05-25 Univ Pennsylvania VETERINARY COMPOSITION AND METHOD FOR NONOPERATIVE CASTRATION
EP2914731B1 (en) * 2012-11-01 2019-09-11 California Institute of Technology Reversible gene expression
ES2921623T3 (es) 2012-11-26 2022-08-30 Modernatx Inc ARN modificado terminalmente
KR20230156146A (ko) * 2012-12-13 2023-11-13 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Dna 항체 작제물 및 그 이용 방법
WO2015089492A2 (en) 2013-12-13 2015-06-18 The Trustees Of The University Of Pennsylvania Dna antibody constructs and method of using same
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
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
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
US9884075B2 (en) 2014-01-16 2018-02-06 California Institute Of Technology Domain-swap T cell receptors
MX392639B (es) 2014-03-21 2025-03-24 Univ Leland Stanford Junior Vectores virales recombinantes para la integración de transgenes
MY186389A (en) * 2014-05-13 2021-07-22 Univ Pennsylvania Compositions comprising aav expressing dual antibody constructs and uses thereof
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
CN107106689A (zh) * 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
CN112410338A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
BR112017011556A2 (en) 2014-12-01 2018-03-06 The Trustees Of The University Of Pennsylvania methods for generating a synthetic antibody, preventing or treating a disease, treating a subject of pathogen infection and a subject with cancer, product, nucleic acid molecule, and composition.
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
WO2016122791A1 (en) 2015-01-30 2016-08-04 The Regents Of The University Of California Spinal subpial gene delivery system
EP3283126B1 (en) 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
CN107708416B (zh) 2015-05-01 2021-06-29 Onl医疗股份有限公司 肽组合物和使用方法
EP4154914B1 (en) * 2015-05-07 2024-08-28 University of South Florida Modified ube3a gene for a gene therapy approach for angelman syndrome
EA201792500A1 (ru) 2015-05-13 2018-04-30 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Aav-опосредованная экспрессия антител против гриппа и способы их использования
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
WO2017007994A1 (en) 2015-07-08 2017-01-12 American Gene Technologies International Inc. Hiv pre-immunization and immunotherapy
US10266844B2 (en) 2015-07-31 2019-04-23 California Institute Of Technology Activity-dependent expression of nucleic acids
EP3875593A1 (en) 2015-12-11 2021-09-08 California Institute of Technology Targeting peptides for directing adeno-associated viruses (aavs)
CN111569090A (zh) 2016-01-15 2020-08-25 美国基因技术国际有限公司 用于活化γ-δ T细胞的方法和组合物
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
WO2017180936A1 (en) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US20190224339A1 (en) * 2016-04-29 2019-07-25 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11976292B2 (en) 2016-06-08 2024-05-07 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
EP3481418A4 (en) 2016-07-08 2020-03-11 American Gene Technologies International Inc. PRE-IMMUNIZATION AND IMMUNOTHERAPY OF HIV
US11583562B2 (en) 2016-07-21 2023-02-21 American Gene Technologies International Inc. Viral vectors for treating Parkinson's disease
KR102621134B1 (ko) * 2016-07-26 2024-01-04 코넬 유니버시티 알데히드 데히드로게나제 결핍의 치료를 위한 유전자 요법
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
AU2017336440B2 (en) 2016-09-28 2022-06-02 Cohbar, Inc. Therapeutic MOTS-c related peptides
EP3518966A1 (en) 2016-09-29 2019-08-07 GlaxoSmithKline Biologicals S.A. Compositions and methods of treatment of persistent hpv infection
EP3538546B1 (en) 2016-11-14 2025-01-08 Novartis AG Compositions, methods, and therapeutic uses related to fusogenic protein minion
GB201620968D0 (en) 2016-12-09 2017-01-25 Glaxosmithkline Biologicals Sa Adenovirus polynucleotides and polypeptides
CN110036020A (zh) 2016-12-09 2019-07-19 葛兰素史密丝克莱恩生物有限公司 具有狂犬病病毒抗原的黑猩猩腺病毒构建体
WO2018134265A1 (en) 2017-01-17 2018-07-26 Forrest Michael David 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
MX2019010275A (es) 2017-02-28 2019-10-15 Univ Pennsylvania Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este.
BR112019017697A2 (pt) 2017-02-28 2020-04-07 The Trustees Of The University Of Pennsylvania vacinas contra influenza baseadas em vetores de vírus adenoassociado (aav)
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
WO2018162748A1 (en) * 2017-03-10 2018-09-13 Genethon Treatment of glycogen storage disease iii
JP2020512815A (ja) * 2017-04-03 2020-04-30 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 フェニルケトン尿症を処置するための組成物および方法
WO2018191450A2 (en) 2017-04-14 2018-10-18 National Taiwan University Hospital Gene therapy for aadc deficiency
EP3634978A1 (en) 2017-06-07 2020-04-15 Adrx, Inc. Tau aggregation inhibitors
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2018232359A1 (en) 2017-06-16 2018-12-20 American Gene Technologies International Inc. Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells
US12116577B2 (en) * 2017-06-20 2024-10-15 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
RU2019143627A (ru) * 2017-06-28 2021-07-28 Юниверсити Оф Соус Флорида Модифицированный ген ube3a для генной терапии синдрома ангельмана
IL271845B2 (en) 2017-07-13 2024-06-01 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
CN110945018A (zh) 2017-07-27 2020-03-31 诺华股份有限公司 抗脱落酶的trem2变体
TWI832036B (zh) 2017-08-03 2024-02-11 美商航海家醫療公司 用於aav之遞送之組合物及方法
BR112020002394A2 (pt) * 2017-08-09 2020-07-28 Bioverativ Therapeutics Inc. moléculas de ácidos nucleicos e usos das mesmas
EP3697902A1 (en) 2017-10-16 2020-08-26 GlaxoSmithKline Biologicals S.A. Enhanced promoter
KR20200066349A (ko) 2017-10-16 2020-06-09 글락소스미스클라인 바이오로지칼즈 에스.에이. 복제 가능 아데노바이러스 벡터
CA3084346A1 (en) 2017-10-16 2019-04-25 Glaxosmithkline Biologicals Sa Simian adenoviral vectors with two expression cassettes
CA3079048A1 (en) 2017-10-16 2019-04-25 Glaxosmithkline Biologicals Sa Adenoviral vectors with two expression cassettes encoding rsv antigenic proteins or fragments thereof
CN111819281B (zh) * 2017-10-23 2024-12-31 普利维尔治疗公司 用于神经变性疾病的基因疗法
US20200283800A1 (en) * 2017-10-23 2020-09-10 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative diseases
BR112020012336A2 (pt) 2017-12-19 2021-03-30 Akouos, Inc. Administração de anticorpos terapêuticos mediada por aav para o ouvido interno
MX2020006225A (es) 2017-12-20 2020-12-07 Glaxosmithkline Biologicals Sa Constructos antigenicos del virus de epstein-barr.
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
BR112020014404A2 (pt) 2018-01-17 2020-12-29 Adrenas Therapeutics, Inc. Terapia gênica de vírus adenoassociado para deficiência de 21-hidroxilase
MA51842A (fr) * 2018-02-14 2020-12-23 Generation Bio Co Vecteurs d'adn non viraux et utilisations associées pour la production d'anticorps et de protéines de fusion
WO2019199841A1 (en) * 2018-04-09 2019-10-17 Cure Ahc, Inc. Aav-mediated delivery of atp1a3 genes to central nervous system
CN119662732A (zh) * 2018-05-08 2025-03-21 纽洛可科学有限公司 抗fam19a5抗体的腺相关病毒(aav)递送
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3793615A2 (en) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Directed evolution of aav to improve tropism for cns
CN112638936A (zh) 2018-06-12 2021-04-09 葛兰素史密丝克莱恩生物有限公司 腺病毒多核苷酸和多肽
KR102925832B1 (ko) 2018-08-09 2026-02-11 바이오버라티브 테라퓨틱스 인크. 핵산 분자 및 비바이러스 유전자 치료를 위한 이의 용도
WO2020061079A1 (en) 2018-09-18 2020-03-26 California Institute Of Technology Engineered light-sensitive proteins
CN113383010B (zh) 2018-09-28 2025-09-09 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US11352646B2 (en) 2018-11-05 2022-06-07 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
WO2020113199A1 (en) 2018-12-01 2020-06-04 International Aids Vaccine Initiative Recombinant hiv env polypeptides and their use
BR112021012249A2 (pt) * 2019-01-04 2021-09-08 Sangamo Therapeutics, Inc. Métodos e composições para o tratamento da doença de fabry
WO2020150065A1 (en) 2019-01-14 2020-07-23 California Institute Of Technology Methods and compositions for modulating appetite and intake of sodium
MX2021008542A (es) 2019-01-18 2021-11-12 Voyager Therapeutics Inc Métodos y sistemas para la producción de partículas de aav.
CN113518784A (zh) 2019-01-28 2021-10-19 科巴公司 治疗性肽
WO2020172465A1 (en) 2019-02-20 2020-08-27 Duke University Compositions and methods for the treatment of genetic diseases
WO2020190750A1 (en) 2019-03-15 2020-09-24 Modernatx, Inc. Hiv rna vaccines
US20220227875A1 (en) * 2019-05-31 2022-07-21 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
JP2022545378A (ja) * 2019-08-15 2022-10-27 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア SCA1の治療のための、導入遺伝子およびイントロン由来miRNAを組み合わせた治療法
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
EP4048800A1 (en) 2019-10-23 2022-08-31 Shire Human Genetic Therapies, Inc. Adeno-associated virus vectors based gene therapy for hereditary angioedema
IL294278A (en) * 2019-12-31 2022-08-01 Swanbio Therapeutics Ltd Improved aav-abcd1 constructs and use for the treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)
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
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
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
WO2022060916A1 (en) * 2020-09-15 2022-03-24 Regenxbio Inc. Vectorized antibodies for anti-viral therapy
EP4240389A4 (en) * 2020-11-04 2025-05-28 The Trustees of The University of Pennsylvania VIRAL VECTORS ENCODING DOG INSULIN FOR THE 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
US20220186252A1 (en) 2020-12-16 2022-06-16 Takeda Pharmaceutical Company Limited Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein
WO2022157548A1 (en) 2021-01-24 2022-07-28 Forrest Michael David Inhibitors of atp synthase - cosmetic and therapeutic uses
US20240148903A1 (en) 2021-03-08 2024-05-09 Nanjing GenScript Biotech Co., Ltd. Delivery of antibody by using dual viral vector system
AR125143A1 (es) 2021-03-19 2023-06-14 Adrenas Therapeutics Inc Terapias génicas para la deficiencia de la 21-hidroxilasa
CN117940135A (zh) * 2021-08-06 2024-04-26 宾夕法尼亚大学理事会 用于治疗和预防错误折叠的蛋白的组合物和方法
EP4395808A4 (en) * 2021-09-01 2025-11-19 Jaguar Gene Therapy Llc Adenine-associated vectors and virions for the treatment of galactosemia and methods of use and manufacture
CN115772520B (zh) * 2021-09-08 2023-11-21 北京锦篮基因科技有限公司 用于治疗庞贝氏病的基因治疗构建体、药物组合物和方法
EP4415746A1 (en) 2021-10-12 2024-08-21 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
CN119365208A (zh) 2022-04-13 2025-01-24 巴塞罗那自治大学 通过表达klotho蛋白的基因疗法治疗神经肌肉疾病
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
US20250381304A1 (en) 2022-06-21 2025-12-18 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
CN116396983A (zh) * 2022-10-10 2023-07-07 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
AU2023417630A1 (en) 2022-12-29 2025-05-15 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
AU2024276475A1 (en) * 2023-05-22 2025-09-25 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
US6825945B1 (en) 1999-05-25 2004-11-30 Silverbrook Research Pty Ltd Method and system for delivery of a brochure
CA2413156C (en) * 2000-07-03 2009-08-18 Gala Design, Inc. Expression vectors
US7939059B2 (en) 2001-12-10 2011-05-10 California Institute Of Technology Method for the generation of antigen-specific lymphocytes
EP2036985A1 (en) * 2002-04-09 2009-03-18 Nationwide Children's Hospital, Inc. Antibody gene transfer and recombinant AAV therefor
WO2004061113A1 (en) * 2003-01-07 2004-07-22 The University Of Hong Kong Adeno-associated virus mediated b7.1 vaccination synergizes with angiostatin to eradicate disseminated liver metastatic cancers
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
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
CA2526120A1 (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
WO2005056762A2 (en) * 2003-12-05 2005-06-23 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
EP1807157A2 (en) * 2004-10-22 2007-07-18 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
CN101287838B (zh) * 2005-10-14 2016-03-30 美国政府健康及人类服务部,疾病控制和预防中心 狂犬病病毒组合物和方法
US20110110892A1 (en) * 2008-03-24 2011-05-12 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
US9132173B2 (en) * 2010-10-15 2015-09-15 Institut National De La Sante Et De La Recherche Medicale (Inserm) Expression vector for cholesterol 24-hydrolase in therapy of Huntington's disease
US8865881B2 (en) 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors

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