JP7176756B2 - パーキンソン病を処置するためのウイルスベクター - Google Patents

パーキンソン病を処置するためのウイルスベクター Download PDF

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JP7176756B2
JP7176756B2 JP2019502170A JP2019502170A JP7176756B2 JP 7176756 B2 JP7176756 B2 JP 7176756B2 JP 2019502170 A JP2019502170 A JP 2019502170A JP 2019502170 A JP2019502170 A JP 2019502170A JP 7176756 B2 JP7176756 B2 JP 7176756B2
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JP2019520841A5 (enExample
JP2019520841A (ja
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テイラー ラフーゼン，
チャールズ デイビッド パウザ，
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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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
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    • 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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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1048—Glycosyltransferases (2.4)
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    • C12Y204/02—Pentosyltransferases (2.4.2)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/50—Physical structure
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
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JP2019502170A 2016-07-21 2017-07-20 パーキンソン病を処置するためのウイルスベクター Active JP7176756B2 (ja)

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US201662365316P 2016-07-21 2016-07-21
US62/365,316 2016-07-21
PCT/US2017/043157 WO2018017882A1 (en) 2016-07-21 2017-07-20 Viral vectors for treating parkinson's disease

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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022132605A (ja) * 2016-07-21 2022-09-08 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 パーキンソン病を処置するためのウイルスベクター

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100183558A1 (en) 2008-10-17 2010-07-22 Zhennan Lai Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
WO2017007994A1 (en) 2015-07-08 2017-01-12 American Gene Technologies International Inc. Hiv pre-immunization and immunotherapy
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
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
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
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
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
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
WO2021087445A1 (en) * 2019-11-01 2021-05-06 Rutgers, The State University Of New Jersey Targeting deltafosb (δfosb) for treatment of dyskinesia
CN114836472A (zh) * 2021-02-01 2022-08-02 中国人民解放军军事科学院军事医学研究院 PARP-1shRNA重组慢病毒载体、该载体稳定转染HaCaT的细胞系及其应用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100069372A1 (en) 2005-11-07 2010-03-18 The General Hospital Corporation Compositions and methods for modulating poly(adp-ribose) polymerase activity
US20120114607A1 (en) 2008-10-17 2012-05-10 Zhennan Lai Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules

Family Cites Families (137)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5668255A (en) 1984-06-07 1997-09-16 Seragen, Inc. Hybrid molecules having translocation region and cell-binding region
WO1993024632A1 (en) 1992-05-22 1993-12-09 Dana Farber Cancer Institute Hybrid siv/hiv-1 viral vectors and monkey model for aids
AU6014094A (en) 1992-12-02 1994-06-22 Baylor College Of Medicine Episomal vectors for gene therapy
CN1115414C (zh) 1993-07-13 2003-07-23 罗纳-布朗克罗莱尔股份有限公司 用于基因治疗的病毒载体
AU8828598A (en) 1997-08-15 1999-03-08 Rubicon Laboratory, Inc. Retrovirus and viral vectors
AU1124499A (en) 1997-10-28 1999-05-17 Maxygen, Inc. Human papillomavirus vectors
CA2324225A1 (en) 1998-03-20 1999-09-23 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
DE69940521D1 (de) 1998-10-01 2009-04-16 Univ Southern California Retrovirales genübertragungssystem und entsprechendes verwendungverfahren
US6156514A (en) 1998-12-03 2000-12-05 Sunol Molecular Corporation Methods for making recombinant cells
US6410013B1 (en) 1999-01-25 2002-06-25 Musc Foundation For Research Development Viral vectors for use in monitoring HIV drug resistance
WO2000072886A1 (en) 1999-05-26 2000-12-07 Dana-Farber Cancer Institute, Inc. Episomally replicating lentiviral vectors
AU2001257611A1 (en) 2000-04-28 2001-11-12 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
JP2004505892A (ja) 2000-05-12 2004-02-26 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 ヒトパピローマウイルス（ｈｐｖ）−感染細胞の治療
AU2001265187A1 (en) 2000-05-30 2001-12-11 Baylor College Of Medicine Chimeric viral vectors for gene therapy
NO314588B1 (no) 2000-09-04 2003-04-14 Bionor Immuno As HIV-peptider, antigener, vaksinesammensetning, immunoassay- testsett og en fremgangsmåte for å påvise antistoffer indusert av HIV
US7122181B2 (en) 2000-12-19 2006-10-17 Research Development Foundation Lentiviral vector-mediated gene transfer and uses thereof
US20030119770A1 (en) 2001-08-02 2003-06-26 Zhennan Lai Intercellular delivery of a herpes simplex virus VP22 fusion protein from cells infected with lentiviral vectors
WO2003015708A2 (en) 2001-08-18 2003-02-27 Myriad Genetics, Inc Composition and method for treating hiv infection
US7737124B2 (en) 2001-09-13 2010-06-15 California Institute Of Technology Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
US20070203333A1 (en) 2001-11-30 2007-08-30 Mcswiggen James RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
JP2005521393A (ja) 2002-03-20 2005-07-21 マサチューセッツ インスティテュート オブ テクノロジー Ｈｉｖ治療
US20040142416A1 (en) 2002-04-30 2004-07-22 Laipis Philip J. Treatment for phenylketonuria
US20040161412A1 (en) 2002-08-22 2004-08-19 The Cleveland Clinic Foundation Cell-based VEGF delivery
US8034334B2 (en) 2002-09-06 2011-10-11 The United States Of America As Represented By The Secretary, Department Of Health And Human Services Immunotherapy with in vitro-selected antigen-specific lymphocytes after non-myeloablative lymphodepleting chemotherapy
ATE443764T1 (de) 2002-11-04 2009-10-15 Univ Massachusetts Allelspezifische rna-interferenz
WO2004053137A2 (en) 2002-12-11 2004-06-24 Cytos Biotechnology Ag Method for protein production
US20060246520A1 (en) 2003-05-23 2006-11-02 Institut National De La Sante Et De La Recherche Medicale Gamma delta t cell-mediated therapy
WO2005005644A1 (en) 2003-07-11 2005-01-20 Cytos Biotechnology Ag Gene expression system
US20050019927A1 (en) 2003-07-13 2005-01-27 Markus Hildinger DECREASING GENE EXPRESSION IN A MAMMALIAN SUBJECT IN VIVO VIA AAV-MEDIATED RNAi EXPRESSION CASSETTE TRANSFER
US20050138677A1 (en) 2003-09-16 2005-06-23 Pfister Herbert J. Transgenic animal model for the treatment of skin tumors
WO2005028634A2 (en) 2003-09-18 2005-03-31 Emory University Improved mva vaccines
WO2005033282A2 (en) 2003-10-01 2005-04-14 Pharmacia & Upjohn Company Llc Polyamide compositions and therapeutic methods for treatment of human papilloma virus
US20080039413A1 (en) 2003-10-21 2008-02-14 Morris David W Novel compositions and methods in cancer
EP1753777B1 (en) 2004-02-25 2014-05-07 Dana-Farber Cancer Institute, Inc. METHODS AND COMPOSITIONS FOR THE TREATMENT AND PREVENTION OF HIV INFECTION USING TRIM5a
US20060058255A1 (en) 2004-03-01 2006-03-16 Jianzhu Chen RNAi-based therapeutics for allergic rhinitis and asthma
TWI439284B (zh) 2004-04-09 2014-06-01 艾伯維生物技術有限責任公司 用於治療ＴＮＦα相關失調症之多重可變劑量療法
US20080227736A1 (en) 2004-06-03 2008-09-18 Regents Of The University Of California, Targeting Pseudotyped Retroviral Vectors
WO2006012221A2 (en) 2004-06-25 2006-02-02 The Regents Of The University Of California Target cell-specific short interfering rna and methods of use thereof
WO2006023491A2 (en) 2004-08-16 2006-03-02 The Cbr Institute For Biomedical Research, Inc. Method of delivering rna interference and uses thereof
EP1802641B8 (en) 2004-10-08 2012-03-07 The Board Of Trustees Of The University Of Illinois Bisphosphonate compounds and methods for bone resorption diseases, cancer, bone pain, immune disorders, and infectious diseases
EP1647595A1 (en) 2004-10-15 2006-04-19 Academisch Medisch Centrum bij de Universiteit van Amsterdam Nucleic acids against viruses, in particular HIV
WO2006048215A1 (en) 2004-11-02 2006-05-11 Istituto Di Ricerche Di Biologia Molecolare P Angeletti Spa Adenoviral amplicon and producer cells for the production of replication-defective adenoviral vectors, methods of preparation and use thereof
US7790446B2 (en) 2005-02-11 2010-09-07 Kosagen Cell Factory Oü Vectors, cell lines and their use in obtaining extended episomal maintenance replication of hybrid plasmids and expression of gene products
MX2007010008A (es) 2005-02-16 2008-01-18 Lentigen Corp Vectores lentivirales y su uso.
ES2564823T3 (es) 2005-05-27 2016-03-29 Ospedale San Raffaele S.R.L. Vector génico que comprende miARN
US20070032443A1 (en) 2005-08-02 2007-02-08 Jaeseob Kim Therapy for Alzheimer's disease
WO2007015122A1 (en) 2005-08-02 2007-02-08 Genexel, Inc. Therapy for alzheimer’s disease
WO2007133674A2 (en) 2006-05-12 2007-11-22 Lentigen Corporation Lentiviral vector compositions, methods and applications
WO2007149476A2 (en) 2006-06-19 2007-12-27 Trustees Of Columbia University In The City Of New York Assays for non-apoptotic cell death and uses thereof
US20080003225A1 (en) 2006-06-29 2008-01-03 Henri Vie Method for enhancing the antibody-dependent cellular cytotoxicity (ADCC) and uses of T cells expressing CD16 receptors
WO2008008719A2 (en) 2006-07-10 2008-01-17 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the myc gene
EP1878440A1 (en) 2006-07-13 2008-01-16 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and compositions for increasing the efficiency of therapeutic antibodies using gamma delta cell activator compounds
EP2049103A2 (en) 2006-07-26 2009-04-22 Novartis AG Inhibitors of undecaprenyl pyrophosphate synthase
US20080199961A1 (en) 2006-08-25 2008-08-21 Avi Biopharma, Inc. ANTISENSE COMPOSITION AND METHOD FOR INHIBITION OF miRNA BIOGENESIS
WO2008100292A2 (en) 2006-10-16 2008-08-21 Genelux Corporation Modified vaccinia virus strains for use in diagnostic and therapeutic methods
ES2639568T3 (es) 2007-01-23 2017-10-27 Janssen Pharmaceutica Nv Método para diseñar un régimen farmacológico para pacientes infectados con el VIH
CA2682694A1 (en) 2007-04-12 2008-10-23 The Board Of Trustees Of The University Of Illinois Bisphosphonate compounds and methods with enhanced potency for multiple targets including fpps, ggpps, and dpps
US20080293142A1 (en) 2007-04-19 2008-11-27 The Board Of Regents For Oklahoma State University Multiple shRNA Expression Vectors and Methods of Construction
EP2008656A1 (en) 2007-06-28 2008-12-31 Bergen Teknologioverforing AS Compositions for the treatment of hyperphenylalaninemia
US20110177155A1 (en) 2007-08-21 2011-07-21 Immune Disease Institute, Inc. Methods of delivery of agents to leukocytes and endothelial cells
EP2090659A1 (en) 2008-02-14 2009-08-19 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Infectious particle, process for its preparation and use thereof
GB0810209D0 (en) 2008-06-04 2008-07-09 Cambridge Entpr Ltd Pluripotency associated epigenetic factor
US8629334B2 (en) 2008-07-16 2014-01-14 University Of Florida Research Foundation, Inc. Viral-based transient-expression vector system for trees
US20110207226A1 (en) 2008-08-20 2011-08-25 Virxsys Corporation Non-integrating lenti/adeno-associated virus hybrid vector system
WO2010033722A2 (en) 2008-09-17 2010-03-25 Isogenis, Inc. Construction of fully-deleted adenovirus-based gene delivery vectors and uses thereof
CN102994500B (zh) * 2008-10-22 2015-04-01 夸克制药公司 治疗眼部疾病的方法
WO2010051521A1 (en) 2008-10-31 2010-05-06 Lentigen Corporation Cell therapy product for the treatment of hiv infection
AU2009308707A1 (en) 2008-10-31 2010-05-06 Biogen Idec Ma Inc. LIGHT targeting molecules and uses thereof
CN102369288A (zh) 2008-11-14 2012-03-07 生命技术公司 用于工程化细胞的组合物和方法
EP2191834A1 (en) 2008-11-26 2010-06-02 Centre National De La Recherche Scientifique (Cnrs) Compositions and methods for treating retrovirus infections
US20120034197A1 (en) 2009-04-09 2012-02-09 Stemcyte Inc. Hiv-resistant stem cells and uses thereof
KR20120088542A (ko) 2009-04-13 2012-08-08 아프세스 게엠베하 & 씨오. 카게 종양 치료를 위한 조작된 중간엽 줄기세포 및 이를 사용하는 방법
EP2425001A4 (en) 2009-04-30 2012-11-14 Univ California ANTI-HIV COMBINATION VECTORS, TARGETING VECTORS AND METHOD OF USE
AU2010274031B2 (en) 2009-07-15 2015-07-16 Csl Behring Gene Therapy, Inc. Dual vector for inhibition of human immunodeficiency virus
EP2295543A1 (en) * 2009-09-11 2011-03-16 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Method for the preparation of an influenza virus
WO2011066578A1 (en) 2009-11-30 2011-06-03 American Gene Technologies International Inc. Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules to treat liver cancer
CN101805750B (zh) 2009-12-29 2011-11-30 浙江大学 法呢基焦磷酸合成酶rna干扰重组慢病毒载体构建及用途
JP5300152B2 (ja) 2010-02-02 2013-09-25 株式会社Ｎｔｔファシリティーズ 防火扉
WO2011103417A2 (en) 2010-02-18 2011-08-25 Emory University Of Technology Transfer Vectors expressing hiv antigens and gm-csf and related methods for generating an immune response
WO2011119942A1 (en) 2010-03-25 2011-09-29 Vistagen Therapeutics, Inc. Induction of ips cells using transient episomal vectors
US20130090371A1 (en) 2010-04-20 2013-04-11 President And Fellows Of Harvard College Methods and compositions for inhibition of beta2-adrenergic receptor degradation
DK2561078T3 (en) 2010-04-23 2019-01-14 Cold Spring Harbor Laboratory NEW STRUCTURALLY DESIGNED SHRNAs
JP5805089B2 (ja) 2010-08-10 2015-11-04 タカラバイオ株式会社 細胞集団の製造方法
WO2012048303A2 (en) 2010-10-07 2012-04-12 Columbia University METHOD FOR TREATING CANCER HARBORING A p53 MUTATION
WO2012061075A2 (en) 2010-10-25 2012-05-10 The Regents Of The University Of California Hiv resistant and functional hematopoietic stem/progenitor cells and macrophages from induced pluripotent stem cells
US8865881B2 (en) 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
US9226976B2 (en) 2011-04-21 2016-01-05 University Of Massachusetts RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
CA2856137A1 (en) 2011-11-22 2013-05-30 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
US9745631B2 (en) 2011-12-20 2017-08-29 Dana-Farber Cancer Institute, Inc. Methods for diagnosing and treating oncogenic kras-associated cancer
SG11201404741VA (en) 2012-02-07 2014-09-26 Global Bio Therapeutics Usa Inc Compartmentalized method of nucleic acid delivery and compositions and uses thereof
WO2013174404A1 (en) 2012-05-23 2013-11-28 Ganymed Pharmaceuticals Ag Combination therapy involving antibodies against claudin 18.2 for treatment of cancer
CA2875624A1 (en) 2012-06-06 2013-12-12 Bionor Immuno As Hiv vaccine
US9556433B2 (en) 2012-07-17 2017-01-31 Université De Genève Nucleic acids for down-regulation of gene expression
WO2014052855A1 (en) 2012-09-27 2014-04-03 Population Diagnostics, Inc. Methods and compositions for screening and treating developmental disorders
CA2930339A1 (en) 2012-11-13 2014-05-22 Jan Lotvall Delivery of therapeutic agent
US9255250B2 (en) 2012-12-05 2016-02-09 Sangamo Bioscience, Inc. Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene
US9642921B2 (en) 2012-12-20 2017-05-09 Tocagen Inc. Cancer combination therapy and recombinant vectors
WO2014117050A2 (en) 2013-01-26 2014-07-31 Mirimus, Inc. Modified mirna as a scaffold for shrna
CN103184224A (zh) 2013-04-03 2013-07-03 衡阳师范学院 一种抗艾滋病病毒感染的三联miRNA及构建方法
EP3461909A1 (en) 2013-05-21 2019-04-03 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Isoforms of gata6 and nkx2-1 as markers for diagnosis and therapy of cancer and as targets for anti-cancer therapy
CA2954168C (en) 2013-08-02 2023-09-19 The Regents Of The University Of California Engineering antiviral t cell immunity through stem cells and chimeric antigen receptors
WO2015042308A2 (en) 2013-09-18 2015-03-26 City Of Hope Rna-based hiv inhibitors
WO2015042720A1 (en) * 2013-09-27 2015-04-02 St. Michael's Hospital Novel lincrna and interfering nucleic acid molecules, compositions and methods and uses thereof for regulating angiogenesis and related conditions
MX2016005239A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Tratamiento con acido ribonucleico mensajero para la fenilcetonuria.
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
EP2878674A1 (en) 2013-11-28 2015-06-03 Fundación Centro Nacional de Investigaciones Cardiovasculares Carlos III (CNIC) Stable episomes based on non-integrative lentiviral vectors
IL313845A (en) 2014-04-23 2024-08-01 Juno Therapeutics Inc Methods for enriching or producing immune cell populations for adoptive therapy
IL296691B2 (en) 2014-04-25 2023-11-01 2Seventy Bio Inc MND promoter chimeric antigen receptors
CA3205555A1 (en) 2014-04-25 2015-10-29 Genethon Treatment of hyperbilirubinemia
CA2955254A1 (en) 2014-08-29 2016-03-03 Immunomedics, Inc. Identification of cancer genes by in-vivo fusion of human cancer cells and animal cells
ES2896401T3 (es) 2014-09-22 2022-02-24 Tanea Medical Ab Proteínas recombinantes exentas de Phe para su uso en el tratamiento de la fenilcetonuria
WO2016057980A1 (en) 2014-10-10 2016-04-14 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Methods to eliminate cancer stem cells by targeting cd47
EP3206696A4 (en) 2014-10-14 2018-08-08 Texas Tech University System Multiplexed shrnas and uses thereof
WO2016069716A1 (en) 2014-10-30 2016-05-06 The Scripps Research Institute Compositions and methods comprising tyrosyl-trna synthetases and resveratrol compounds
GB201509202D0 (en) 2015-05-28 2015-07-15 Ge Healthcare Bio Sciences Ab Semi-static cell culture
US20180161455A1 (en) 2015-06-10 2018-06-14 American Gene Technologies International, Inc. Non-integrating viral delivery system and methods of use therof
WO2017007994A1 (en) 2015-07-08 2017-01-12 American Gene Technologies International Inc. Hiv pre-immunization and immunotherapy
US11028369B2 (en) * 2015-08-13 2021-06-08 Beihao Stem Cell And Regenerative Medicine Research Institute Co., Ltd. Induced extended pluripotent stem cells, method of making and using
CN105112370B (zh) 2015-08-25 2019-02-05 杭州优善生物科技有限公司 一种体外刺激外周血γδT细胞高效增殖的方法及其应用
WO2017068077A1 (en) 2015-10-20 2017-04-27 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and products for genetic engineering
WO2017100551A1 (en) 2015-12-09 2017-06-15 Alexion Pharmaceuticals, Inc. HETEROLOGOUS UTR SEQUENCES FOR ENHANCED mRNA EXPRESSION
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
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
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
WO2017173453A1 (en) 2016-04-01 2017-10-05 The Brigham And Women's Hospital, Inc. Stimuli-responsive nanoparticles for biomedical applications
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
BR112019013576A2 (pt) 2016-12-30 2020-02-04 The Trustees Of The University Of Pennsylvania terapia genica para o tratamento da fenilcetonuria
KR20240023703A (ko) 2017-01-09 2024-02-22 아메리칸 진 테크놀로지스 인터내셔널 인코포레이티드 예비-면역화 단계가 없는 hiv 면역요법
US12023385B2 (en) 2017-02-08 2024-07-02 Dana-Farber Cancer Institute, Inc. Tunable endogenous protein degradation with heterobifunctional compounds
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
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
CA3077355A1 (en) 2017-10-02 2019-04-11 American Gene Technologies International Inc. Vectors with promoter and enhancer combinations for treating phenylketonuria
US11352646B2 (en) 2018-11-05 2022-06-07 American Gene Technologies International Inc. Vector system for expressing regulatory RNA
KR20220068954A (ko) 2019-05-31 2022-05-26 아메리칸 진 테크놀로지스 인터내셔널 인코포레이티드 최적화된 페닐알라닌 히드록실라아제 발현

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100069372A1 (en) 2005-11-07 2010-03-18 The General Hospital Corporation Compositions and methods for modulating poly(adp-ribose) polymerase activity
US20120114607A1 (en) 2008-10-17 2012-05-10 Zhennan Lai Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
Erdelyi, K. et al.，Dual role of poly(ADP-ribose) glycohydrolase in the regulation of cell death in oxidatively stressed A549 cells，FASEB J.，2009年，Vol. 23(10)，pp. 3553-3563
Lee, Y. et al.，Poly (ADP-ribose) in the pathogenesis of Parkinson's disease，BMB Rep.，2014年，Vol. 47(8)，pp. 424-432
Liu, F. et al.，Effects of poly (ADP-ribose) polymerase-1 (PARP-1) inhibition on sulfur mustard-induced cutaneous injuries in vitro and in vivo，PeerJ.，2016年，Vol. 4:e1890，pp. 1-28
Martin, K. A.，Global Transcriptome Analysis Reveals That Poly(ADP-Ribose) Polymerase 1 Regulates Gene Expression through EZH2，Mol. Cell Biol.，2015年，Vol. 35(23)，pp. 3934-3944
Nie, J.，DNA repair proteins Metnase and PARP1 regulate DNA integration，2000-2019 - CSU Theses and Dissertations[online] ，2015年，CHAPTER 3，pp. 95-141，URL:http://hdl.handle.net/10217/166898

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022132605A (ja) * 2016-07-21 2022-09-08 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 パーキンソン病を処置するためのウイルスベクター

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