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Treatment of GM2 Gangliosidosis in Adult Sandhoff Mice Using an Intravenous Self-Complementary Hexosaminidase Vector.
Osmon, Karlaina Jl; Thompson, Patrick; Woodley, Evan; Karumuthil-Melethil, Subha; Heindel, Cliff; Keimel, John G; Kaemmerer, William F; Gray, Steven J; Walia, Jagdeep S.
Affiliation
  • Osmon KJ; Centre for Neuroscience Studies, Queen\'s University, Kingston, ON K7L 3N6, Canada.
  • Thompson P; Department of Biomedical and Molecular Sciences, Queen\'s University, Kingston, ON K7L 3N6, Canada.
  • Woodley E; Medical Genetics/Departments of Pediatrics, Queen\'s University, Kingston, ON K7L 2V7, Canada.
  • Karumuthil-Melethil S; Department of Biomedical and Molecular Sciences, Queen\'s University, Kingston, ON K7L 3N6, Canada.
  • Heindel C; Gene Therapy Center, University of North Carolina, Chapel Hill, NC, USA.
  • Keimel JG; Gene Therapy Center, University of North Carolina, Chapel Hill, NC, USA.
  • Kaemmerer WF; New Hope Research Foundation, North Oaks, MN, USA.
  • Gray SJ; New Hope Research Foundation, North Oaks, MN, USA.
  • Walia JS; Gene Therapy Center, University of North Carolina, Chapel Hill, NC, USA.
Curr Gene Ther ; 22(3): 262-276, 2022.
Article in En | MEDLINE | ID: mdl-34530708

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Sandhoff Disease / Gangliosidoses, GM2 Limits: Animals / Humans Language: En Journal: Curr Gene Ther Journal subject: GENETICA MEDICA / TERAPEUTICA Year: 2022 Document type: Article Affiliation country: Canada Country of publication: United Arab Emirates

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Sandhoff Disease / Gangliosidoses, GM2 Limits: Animals / Humans Language: En Journal: Curr Gene Ther Journal subject: GENETICA MEDICA / TERAPEUTICA Year: 2022 Document type: Article Affiliation country: Canada Country of publication: United Arab Emirates