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Life-Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy.
Johnson, Aime K; McCurdy, Victoria J; Gray-Edwards, Heather L; Maguire, Anne S; Cochran, J Nicholas; Gross, Amanda L; Skinner, Haleigh E; Randle, Ashley N; Shirley, Jamie L; Brunson, Brandon L; Bradbury, Allison M; Leroy, Stanley G; Hwang, Misako; Rockwell, Hannah E; Cox, Nancy R; Baker, Henry J; Seyfried, Thomas N; Sena-Esteves, Miguel; Martin, Douglas R.
Afiliação
  • Johnson AK; Department of Clinical Sciences, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • McCurdy VJ; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • Gray-Edwards HL; Department of Anatomy, Physiology & Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • Maguire AS; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • Cochran JN; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • Gross AL; Department of Anatomy, Physiology & Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • Skinner HE; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • Randle AN; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • Shirley JL; Department of Anatomy, Physiology & Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • Brunson BL; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • Bradbury AM; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • Leroy SG; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • Hwang M; Department of Anatomy, Physiology & Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • Rockwell HE; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • Cox NR; Department of Anatomy, Physiology & Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • Baker HJ; Department of Neurology and Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts, USA.
  • Seyfried TN; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
  • Sena-Esteves M; Biology Department, Boston College, Chestnut Hill, Massachusetts, USA.
  • Martin DR; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.
Ann Neurol ; 94(5): 969-986, 2023 11.
Article em En | MEDLINE | ID: mdl-37526361

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Sandhoff / Gangliosidoses GM2 Limite: Animals / Child / Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Sandhoff / Gangliosidoses GM2 Limite: Animals / Child / Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article