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Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9.
Chandler, Randy J; Di Pasquale, Giovanni; Sloan, Jennifer L; McCoy, Samantha; Hubbard, Brandon T; Kilts, Tina M; Manoli, Irini; Chiorini, John A; Venditti, Charles P.
Afiliação
  • Chandler RJ; Organic Acid Research Section, Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health, Bldg 10, Room 7N248A, Bethesda, MD 20892, USA.
  • Di Pasquale G; National Institute of Dental and Craniofacial Research, National Institutes of Health, Bethesda, MD 20892, USA.
  • Sloan JL; Organic Acid Research Section, Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health, Bldg 10, Room 7N248A, Bethesda, MD 20892, USA.
  • McCoy S; Organic Acid Research Section, Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health, Bldg 10, Room 7N248A, Bethesda, MD 20892, USA.
  • Hubbard BT; Organic Acid Research Section, Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health, Bldg 10, Room 7N248A, Bethesda, MD 20892, USA.
  • Kilts TM; National Institute of Dental and Craniofacial Research, National Institutes of Health, Bethesda, MD 20892, USA.
  • Manoli I; Organic Acid Research Section, Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health, Bldg 10, Room 7N248A, Bethesda, MD 20892, USA.
  • Chiorini JA; National Institute of Dental and Craniofacial Research, National Institutes of Health, Bethesda, MD 20892, USA.
  • Venditti CP; Organic Acid Research Section, Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health, Bldg 10, Room 7N248A, Bethesda, MD 20892, USA.
Mol Ther Methods Clin Dev ; 27: 61-72, 2022 Dec 08.
Article em En | MEDLINE | ID: mdl-36186952
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Risk_factors_studies Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Risk_factors_studies Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Estados Unidos