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Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease.
Keeler, Allison M; Zieger, Marina; Todeasa, Sophia H; McCall, Angela L; Gifford, Jennifer C; Birsak, Samantha; Choudhury, Sourav R; Byrne, Barry J; Sena-Esteves, Miguel; ElMallah, Mai K.
Affiliation
  • Keeler AM; 1 Division of Pulmonary Medicine, Department of Pediatrics, University of Massachusetts Medical School, Worcester Massachusetts.
  • Zieger M; 2 Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester Massachusetts.
  • Todeasa SH; 1 Division of Pulmonary Medicine, Department of Pediatrics, University of Massachusetts Medical School, Worcester Massachusetts.
  • McCall AL; 2 Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester Massachusetts.
  • Gifford JC; 2 Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester Massachusetts.
  • Birsak S; 3 Department of Neurology, University of Massachusetts Medical School, Worcester Massachusetts.
  • Choudhury SR; 4 Department of Pediatrics, Duke University, Durham, North Carolina.
  • Byrne BJ; 2 Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester Massachusetts.
  • Sena-Esteves M; 3 Department of Neurology, University of Massachusetts Medical School, Worcester Massachusetts.
  • ElMallah MK; 1 Division of Pulmonary Medicine, Department of Pediatrics, University of Massachusetts Medical School, Worcester Massachusetts.
Hum Gene Ther ; 30(1): 57-68, 2019 01.
Article in En | MEDLINE | ID: mdl-29901418
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Glycogen Storage Disease Type II / Gene Transfer Techniques / Dependovirus / Alpha-Glucosidases / Genetic Vectors Type of study: Prognostic_studies Limits: Animals / Humans Language: En Journal: Hum Gene Ther Journal subject: GENETICA MEDICA / TERAPEUTICA Year: 2019 Document type: Article Country of publication: United States
Fulltext
Add to My VHL
Print
XML
PubMed Links
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Glycogen Storage Disease Type II / Gene Transfer Techniques / Dependovirus / Alpha-Glucosidases / Genetic Vectors Type of study: Prognostic_studies Limits: Animals / Humans Language: En Journal: Hum Gene Ther Journal subject: GENETICA MEDICA / TERAPEUTICA Year: 2019 Document type: Article Country of publication: United States