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AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease.
Johnson, Tyler B; White, Katherine A; Brudvig, Jon J; Cain, Jacob T; Langin, Logan; Pratt, Melissa A; Booth, Clarissa D; Timm, Derek J; Davis, Samantha S; Meyerink, Brandon; Likhite, Shibi; Meyer, Kathrin; Weimer, Jill M.
Afiliação
  • Johnson TB; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, USA; Amicus Therapeutics, Philadelphia, PA, USA.
  • White KA; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, USA.
  • Brudvig JJ; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, USA.
  • Cain JT; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, USA; Amicus Therapeutics, Philadelphia, PA, USA.
  • Langin L; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, USA.
  • Pratt MA; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, USA.
  • Booth CD; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, USA.
  • Timm DJ; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, USA.
  • Davis SS; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, USA.
  • Meyerink B; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, USA.
  • Likhite S; The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
  • Meyer K; The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics, The Ohio State University, Columbus, OH, USA.
  • Weimer JM; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, USA; Amicus Therapeutics, Philadelphia, PA, USA. Electronic address: jill.weimer@sanfordhealth.org.
Mol Ther ; 29(1): 162-175, 2021 01 06.
Article em En | MEDLINE | ID: mdl-33010819
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Dependovirus / Vetores Genéticos / Proteínas de Membrana / Lipofuscinoses Ceroides Neuronais Limite: Animals / Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Dependovirus / Vetores Genéticos / Proteínas de Membrana / Lipofuscinoses Ceroides Neuronais Limite: Animals / Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article