Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy.

Vink, Conrad A; Counsell, John R; Perocheau, Dany P; Karda, Rajvinder; Buckley, Suzanne M K; Brugman, Martijn H; Galla, Melanie; Schambach, Axel; McKay, Tristan R; Waddington, Simon N; Howe, Steven J

Vink, Conrad A; Counsell, John R; Perocheau, Dany P; Karda, Rajvinder; Buckley, Suzanne M K; Brugman, Martijn H; Galla, Melanie; Schambach, Axel; McKay, Tristan R; Waddington, Simon N; Howe, Steven J.

Afiliação

Vink CA; Molecular and Cellular Immunology, UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK.
Counsell JR; Molecular and Cellular Immunology, UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK; Gene Transfer Technology Group, Institute for Women's Health, University College London, 86-96 Chenies Mews, London WC1E 6HX, UK. Electronic address: j.counsell@ucl.ac.uk.
Perocheau DP; Gene Transfer Technology Group, Institute for Women's Health, University College London, 86-96 Chenies Mews, London WC1E 6HX, UK.
Karda R; Gene Transfer Technology Group, Institute for Women's Health, University College London, 86-96 Chenies Mews, London WC1E 6HX, UK.
Buckley SMK; Gene Transfer Technology Group, Institute for Women's Health, University College London, 86-96 Chenies Mews, London WC1E 6HX, UK.
Brugman MH; Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, 2300 RC Leiden, the Netherlands.
Galla M; Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.
Schambach A; Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.
McKay TR; School of Healthcare Science, John Dalton Building, Manchester Metropolitan University, Chester Street, Manchester M15 6BH, UK.
Waddington SN; Gene Transfer Technology Group, Institute for Women's Health, University College London, 86-96 Chenies Mews, London WC1E 6HX, UK; MRC Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witswatersrand, Johannesburg 2000, South Africa.
Howe SJ; Molecular and Cellular Immunology, UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK; Gene Transfer Technology Group, Institute for Women's Health, University College London, 86-96 Chenies Mews, London WC1E 6HX, UK.

Mol Ther ; 25(8): 1790-1804, 2017 08 02.

Article em En | MEDLINE | ID: mdl-28550974

Assuntos

Técnicas de Transferência de Genes; Vetores Genéticos/genética; HIV-1/genética; RNA Viral; Sequências Reguladoras de Ácido Ribonucleico; Transdução Genética; Animais; Linhagem Celular; Modelos Animais de Doenças; Fator IX/genética; Expressão Gênica; Ordem dos Genes; Genes Reporter; Terapia Genética; Genoma Viral; Repetição Terminal Longa de HIV; Hemofilia B/sangue; Hemofilia B/genética; Hemofilia B/terapia; Humanos; Camundongos; Provírus/genética; Recombinação Genética; Transgenes; Replicação Viral/genética

Palavras-chave

HIV-1 packaging; HIV-1 remobilization; gene therapy; hemophilia; lentivirus; reverse transcription; splicing; viral vector

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Transdução Genética / RNA Viral / HIV-1 / Técnicas de Transferência de Genes / Sequências Reguladoras de Ácido Ribonucleico / Vetores Genéticos Tipo de estudo: Prognostic_studies Limite: Animals / Humans Idioma: En Ano de publicação: 2017 Tipo de documento: Article

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