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Long-term functional correction of cystathionine ß-synthase deficiency in mice by adeno-associated viral gene therapy.
Lee, Hyung-Ok; Salami, Christiana O; Sondhi, Dolan; Kaminsky, Stephen M; Crystal, Ronald G; Kruger, Warren D.
Afiliação
  • Lee HO; Cancer Biology Program, Fox Chase Cancer Center, Philadelphia, Pennsylvania, USA.
  • Salami CO; Department of Genetic Medicine, Weill Cornell Medical College, New York, New York, USA.
  • Sondhi D; Department of Genetic Medicine, Weill Cornell Medical College, New York, New York, USA.
  • Kaminsky SM; Department of Genetic Medicine, Weill Cornell Medical College, New York, New York, USA.
  • Crystal RG; Department of Genetic Medicine, Weill Cornell Medical College, New York, New York, USA.
  • Kruger WD; Cancer Biology Program, Fox Chase Cancer Center, Philadelphia, Pennsylvania, USA.
J Inherit Metab Dis ; 44(6): 1382-1392, 2021 11.
Article em En | MEDLINE | ID: mdl-34528713

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Dependovirus / Cistationina beta-Sintase / Vetores Genéticos / Homocistinúria Tipo de estudo: Prognostic_studies / Risk_factors_studies Limite: Animals Idioma: En Ano de publicação: 2021 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Dependovirus / Cistationina beta-Sintase / Vetores Genéticos / Homocistinúria Tipo de estudo: Prognostic_studies / Risk_factors_studies Limite: Animals Idioma: En Ano de publicação: 2021 Tipo de documento: Article