Targeted Activation of Cystic Fibrosis Transmembrane Conductance Regulator.

Villamizar, Olga; Waters, Shafagh A; Scott, Tristan; Saayman, Sheena; Grepo, Nicole; Urak, Ryan; Davis, Alicia; Jaffe, Adam; Morris, Kevin V

Villamizar, Olga; Waters, Shafagh A; Scott, Tristan; Saayman, Sheena; Grepo, Nicole; Urak, Ryan; Davis, Alicia; Jaffe, Adam; Morris, Kevin V.

Afiliação

Villamizar O; Center for Gene Therapy, City of Hope-Beckman Research Institute at the City of Hope, Duarte, CA 91010, USA.
Waters SA; Faculty of Medicine, School of Women's & Children's Health, University of New South Wales (UNSW), Sydney, NSW, Australia; Molecular and Integrative Cystic Fibrosis Research Centre (miCF_RC), School of Women's & Children's Health, Faculty of Medicine, University of New South Wales, Sydney, NS
Scott T; Center for Gene Therapy, City of Hope-Beckman Research Institute at the City of Hope, Duarte, CA 91010, USA.
Saayman S; Department of Molecular and Experimental Medicine, The Scripps Research Institute, La Jolla, CA, USA.
Grepo N; Center for Gene Therapy, City of Hope-Beckman Research Institute at the City of Hope, Duarte, CA 91010, USA.
Urak R; Center for Gene Therapy, City of Hope-Beckman Research Institute at the City of Hope, Duarte, CA 91010, USA.
Davis A; Center for Gene Therapy, City of Hope-Beckman Research Institute at the City of Hope, Duarte, CA 91010, USA.
Jaffe A; Faculty of Medicine, School of Women's & Children's Health, University of New South Wales (UNSW), Sydney, NSW, Australia; Molecular and Integrative Cystic Fibrosis Research Centre (miCF_RC), School of Women's & Children's Health, Faculty of Medicine, University of New South Wales, Sydney, NS
Morris KV; Center for Gene Therapy, City of Hope-Beckman Research Institute at the City of Hope, Duarte, CA 91010, USA. Electronic address: kmorris@coh.org.

Mol Ther ; 27(10): 1737-1748, 2019 10 02.

Article em En | MEDLINE | ID: mdl-31383454

Assuntos

Regulador de Condutância Transmembrana em Fibrose Cística/genética; Regulador de Condutância Transmembrana em Fibrose Cística/metabolismo; Fibrose Cística/genética; Terapia de Alvo Molecular/métodos; Mucosa Nasal/metabolismo; Proteína 9 Associada à CRISPR/metabolismo; Linhagem Celular; Membrana Celular/metabolismo; Peptídeos Penetradores de Células/genética; Fibrose Cística/metabolismo; Fibrose Cística/terapia; Células Epiteliais/citologia; Células Epiteliais/metabolismo; Humanos; Nanopartículas/química; Mucosa Nasal/citologia; RNA Guia de Cinetoplastídeos/farmacologia; RNA Longo não Codificante/genética; Ativação Transcricional; Produtos do Gene tat do Vírus da Imunodeficiência Humana/genética

Palavras-chave

ASO delivery; CFTR; Tat-CPP; cystic fibrosis; exosome; lncRNA BGas; transcriptional regulation

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Regulador de Condutância Transmembrana em Fibrose Cística / Fibrose Cística / Terapia de Alvo Molecular / Mucosa Nasal Limite: Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Estados Unidos

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