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Intracerebroventricular enzyme replacement therapy with ß-galactosidase reverses brain pathologies due to GM1 gangliosidosis in mice.
Chen, Joseph C; Luu, Amanda R; Wise, Nathan; Angelis, Rolando De; Agrawal, Vishal; Mangini, Linley; Vincelette, Jon; Handyside, Britta; Sterling, Harry; Lo, Melanie J; Wong, Hio; Galicia, Nicole; Pacheco, Glenn; Van Vleet, Jeremy; Giaramita, Alexander; Fong, Sylvia; Roy, Sushmita M; Hague, Chuck; Lawrence, Roger; Bullens, Sherry; Christianson, Terri M; d'Azzo, Alessandra; Crawford, Brett E; Bunting, Stuart; LeBowitz, Jonathan H; Yogalingam, Gouri.
Afiliação
  • Chen JC; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Luu AR; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Wise N; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Angelis R; Process Sciences, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Agrawal V; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Mangini L; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Vincelette J; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Handyside B; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Sterling H; Process Sciences, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Lo MJ; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Wong H; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Galicia N; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Pacheco G; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Van Vleet J; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Giaramita A; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Fong S; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Roy SM; Process Sciences, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Hague C; Process Sciences, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Lawrence R; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Bullens S; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Christianson TM; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • d'Azzo A; Department of Genetics, St. Jude Children's Research Hospital, Memphis, Tennessee 38105.
  • Crawford BE; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Bunting S; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • LeBowitz JH; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949.
  • Yogalingam G; Research, BioMarin Pharmaceutical, Inc., Novato, California 94949. Electronic address: gyogalingam@bmrn.com.
J Biol Chem ; 295(39): 13532-13555, 2020 09 25.
Article em En | MEDLINE | ID: mdl-31481471

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Gangliosidose GM1 / Beta-Galactosidase / Terapia de Reposição de Enzimas Tipo de estudo: Prognostic_studies Limite: Animals Idioma: En Ano de publicação: 2020 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Gangliosidose GM1 / Beta-Galactosidase / Terapia de Reposição de Enzimas Tipo de estudo: Prognostic_studies Limite: Animals Idioma: En Ano de publicação: 2020 Tipo de documento: Article