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Liver-Targeted AAV8 Gene Therapy Ameliorates Skeletal and Cardiovascular Pathology in a Mucopolysaccharidosis IVA Murine Model.
Sawamoto, Kazuki; Karumuthil-Melethil, Subha; Khan, Shaukat; Stapleton, Molly; Bruder, Joseph T; Danos, Olivier; Tomatsu, Shunji.
Affiliation
  • Sawamoto K; Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE 19899-0269, USA.
  • Karumuthil-Melethil S; REGENXBIO, Rockville, MD 20850, USA.
  • Khan S; Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE 19899-0269, USA.
  • Stapleton M; Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE 19899-0269, USA.
  • Bruder JT; REGENXBIO, Rockville, MD 20850, USA.
  • Danos O; REGENXBIO, Rockville, MD 20850, USA.
  • Tomatsu S; Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE 19899-0269, USA.
Mol Ther Methods Clin Dev ; 18: 50-61, 2020 Sep 11.
Article in En | MEDLINE | ID: mdl-32577432
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Ther Methods Clin Dev Year: 2020 Document type: Article Affiliation country: Estados Unidos Country of publication: Estados Unidos
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: 2020 Document type: Article Affiliation country: Estados Unidos Country of publication: Estados Unidos