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Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia.
Chandler, Randy J; Di Pasquale, Giovanni; Choi, Eun-Young; Chang, David; Smith, Stephanie N; Sloan, Jennifer L; Hoffmann, Victoria; Li, Lina; Chiorini, John A; Venditti, Charles P.
Affiliation
  • Chandler RJ; National Human Genome Research Institute, Bethesda, MD 20892, USA.
  • Di Pasquale G; National Institute of Dental and Craniofacial Research, Bethesda, MD 20892, USA.
  • Choi EY; National Human Genome Research Institute, Bethesda, MD 20892, USA.
  • Chang D; National Human Genome Research Institute, Bethesda, MD 20892, USA.
  • Smith SN; National Human Genome Research Institute, Bethesda, MD 20892, USA.
  • Sloan JL; National Human Genome Research Institute, Bethesda, MD 20892, USA.
  • Hoffmann V; Office of Research Services, National Institutes of Health, Bethesda, MD 20892, USA.
  • Li L; National Human Genome Research Institute, Bethesda, MD 20892, USA.
  • Chiorini JA; National Institute of Dental and Craniofacial Research, Bethesda, MD 20892, USA.
  • Venditti CP; National Human Genome Research Institute, Bethesda, MD 20892, USA.
Mol Ther Methods Clin Dev ; 30: 181-190, 2023 Sep 14.
Article in En | MEDLINE | ID: mdl-37746248
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Ther Methods Clin Dev Year: 2023 Document type: Article Affiliation country: United States
Fulltext
Add to My VHL
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Ther Methods Clin Dev Year: 2023 Document type: Article Affiliation country: United States