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AAV gene therapy for Tay-Sachs disease.
Flotte, Terence R; Cataltepe, Oguz; Puri, Ajit; Batista, Ana Rita; Moser, Richard; McKenna-Yasek, Diane; Douthwright, Catherine; Gernoux, Gwladys; Blackwood, Meghan; Mueller, Christian; Tai, Phillip W L; Jiang, Xuntian; Bateman, Scot; Spanakis, Spiro G; Parzych, Julia; Keeler, Allison M; Abayazeed, Aly; Rohatgi, Saurabh; Gibson, Laura; Finberg, Robert; Barton, Bruce A; Vardar, Zeynep; Shazeeb, Mohammed Salman; Gounis, Matthew; Tifft, Cynthia J; Eichler, Florian S; Brown, Robert H; Martin, Douglas R; Gray-Edwards, Heather L; Sena-Esteves, Miguel.
Affiliation
  • Flotte TR; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA. Terry.Flotte@umassmed.edu.
  • Cataltepe O; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA. Terry.Flotte@umassmed.edu.
  • Puri A; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA.
  • Batista AR; Department of Neurosurgery, UMass Chan Medical School, Worcester, MA, USA.
  • Moser R; Department of Radiology, UMass Chan Medical School, Worcester, MA, USA.
  • McKenna-Yasek D; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA.
  • Douthwright C; Department of Neurology, UMass Chan Medical School, Worcester, MA, USA.
  • Gernoux G; Department of Neurosurgery, UMass Chan Medical School, Worcester, MA, USA.
  • Blackwood M; Department of Neurology, UMass Chan Medical School, Worcester, MA, USA.
  • Mueller C; Department of Neurology, UMass Chan Medical School, Worcester, MA, USA.
  • Tai PWL; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA.
  • Jiang X; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA.
  • Bateman S; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA.
  • Spanakis SG; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA.
  • Parzych J; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA.
  • Keeler AM; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA.
  • Abayazeed A; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA.
  • Rohatgi S; Department of Medicine and Cardiovascular Division, Washington University School of Medicine, St. Louis, MO, USA.
  • Gibson L; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA.
  • Finberg R; Departments of Anesthesiology and Perioperative Medicine, UMass Chan Medical School, Worcester, MA, USA.
  • Barton BA; Departments of Anesthesiology and Perioperative Medicine, UMass Chan Medical School, Worcester, MA, USA.
  • Vardar Z; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA.
  • Shazeeb MS; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA.
  • Gounis M; Department of Radiology, UMass Chan Medical School, Worcester, MA, USA.
  • Tifft CJ; Department of Radiology, UMass Chan Medical School, Worcester, MA, USA.
  • Eichler FS; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA.
  • Brown RH; Department of Internal Medicine, UMass Chan Medical School, Worcester, MA, USA.
  • Martin DR; Department of Internal Medicine, UMass Chan Medical School, Worcester, MA, USA.
  • Gray-Edwards HL; Population and Quantitative Health Sciences, UMass Chan Medical School, Worcester, MA, USA.
  • Sena-Esteves M; Department of Radiology, UMass Chan Medical School, Worcester, MA, USA.
Nat Med ; 28(2): 251-259, 2022 02.
Article in En | MEDLINE | ID: mdl-35145305
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Tay-Sachs Disease Limits: Humans Language: En Journal: Nat Med Journal subject: BIOLOGIA MOLECULAR / MEDICINA Year: 2022 Document type: Article Affiliation country: United States 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: Tay-Sachs Disease Limits: Humans Language: En Journal: Nat Med Journal subject: BIOLOGIA MOLECULAR / MEDICINA Year: 2022 Document type: Article Affiliation country: United States Country of publication: United States