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Lentiviral gene therapy for X-linked chronic granulomatous disease recapitulates endogenous CYBB regulation and expression.
Wong, Ryan L; Sackey, Sarah; Brown, Devin; Senadheera, Shantha; Masiuk, Katelyn; Quintos, Jason P; Colindres, Nicole; Riggan, Luke; Morgan, Richard A; Malech, Harry L; Hollis, Roger P; Kohn, Donald B.
Afiliação
  • Wong RL; Department of Molecular and Medical Pharmacology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA.
  • Sackey S; ImmunoVec, Los Angeles, CA.
  • Brown D; Department of Microbiology, Immunology and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA.
  • Senadheera S; Department of Microbiology, Immunology and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA.
  • Masiuk K; Department of Microbiology, Immunology and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA.
  • Quintos JP; ImmunoVec, Los Angeles, CA.
  • Colindres N; Department of Microbiology, Immunology and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA.
  • Riggan L; ImmunoVec, Los Angeles, CA.
  • Morgan RA; Department of Microbiology, Immunology and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA.
  • Malech HL; ImmunoVec, Los Angeles, CA.
  • Hollis RP; ImmunoVec, Los Angeles, CA.
  • Kohn DB; Department of Molecular and Medical Pharmacology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA.
Blood ; 141(9): 1007-1022, 2023 03 02.
Article em En | MEDLINE | ID: mdl-36332160

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença Granulomatosa Crônica Tipo de estudo: Prognostic_studies Limite: Animals Idioma: En Revista: Blood Ano de publicação: 2023 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença Granulomatosa Crônica Tipo de estudo: Prognostic_studies Limite: Animals Idioma: En Revista: Blood Ano de publicação: 2023 Tipo de documento: Article