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Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.
Kan, Shih-Hsin; Aoyagi-Scharber, Mika; Le, Steven Q; Vincelette, Jon; Ohmi, Kazuhiro; Bullens, Sherry; Wendt, Daniel J; Christianson, Terri M; Tiger, Pascale M N; Brown, Jillian R; Lawrence, Roger; Yip, Bryan K; Holtzinger, John; Bagri, Anil; Crippen-Harmon, Danielle; Vondrak, Kristen N; Chen, Zhi; Hague, Chuck M; Woloszynek, Josh C; Cheung, Diana S; Webster, Katherine A; Adintori, Evan G; Lo, Melanie J; Wong, Wesley; Fitzpatrick, Paul A; LeBowitz, Jonathan H; Crawford, Brett E; Bunting, Stuart; Dickson, Patricia I; Neufeld, Elizabeth F.
Afiliação
  • Kan SH; Department of Pediatrics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA 90502;
  • Aoyagi-Scharber M; Research and Drug Discovery and eneufeld@mednet.ucla.edu MAoyagi-scharber@bmrn.com pdickson@labiomed.org.
  • Le SQ; Department of Pediatrics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA 90502;
  • Vincelette J; Research and Drug Discovery and.
  • Ohmi K; Department of Biological Chemistry, David Geffen School of Medicine, University of California, Los Angeles, CA 90095.
  • Bullens S; Research and Drug Discovery and.
  • Wendt DJ; Analytical Chemistry, BioMarin Pharmaceutical, Inc., Novato, CA 94949; and.
  • Christianson TM; Research and Drug Discovery and.
  • Tiger PM; Research and Drug Discovery and.
  • Brown JR; Research and Drug Discovery and.
  • Lawrence R; Research and Drug Discovery and.
  • Yip BK; Research and Drug Discovery and.
  • Holtzinger J; Research and Drug Discovery and.
  • Bagri A; Research and Drug Discovery and.
  • Crippen-Harmon D; Research and Drug Discovery and.
  • Vondrak KN; Department of Pediatrics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA 90502;
  • Chen Z; Analytical Chemistry, BioMarin Pharmaceutical, Inc., Novato, CA 94949; and.
  • Hague CM; Analytical Chemistry, BioMarin Pharmaceutical, Inc., Novato, CA 94949; and.
  • Woloszynek JC; Research and Drug Discovery and.
  • Cheung DS; Research and Drug Discovery and.
  • Webster KA; Research and Drug Discovery and.
  • Adintori EG; Research and Drug Discovery and.
  • Lo MJ; Research and Drug Discovery and.
  • Wong W; Research and Drug Discovery and.
  • Fitzpatrick PA; Research and Drug Discovery and.
  • LeBowitz JH; Research and Drug Discovery and.
  • Crawford BE; Research and Drug Discovery and.
  • Bunting S; Research and Drug Discovery and.
  • Dickson PI; Department of Pediatrics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA 90502; eneufeld@mednet.ucla.edu MAoyagi-scharber@bmrn.com pdickson@labiomed.org.
  • Neufeld EF; Department of Biological Chemistry, David Geffen School of Medicine, University of California, Los Angeles, CA 90095 eneufeld@mednet.ucla.edu MAoyagi-scharber@bmrn.com pdickson@labiomed.org.
Proc Natl Acad Sci U S A ; 111(41): 14870-5, 2014 Oct 14.
Article em En | MEDLINE | ID: mdl-25267636

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Acetilglucosaminidase / Proteínas Recombinantes de Fusão / Encéfalo / Fator de Crescimento Insulin-Like II / Sistemas de Liberação de Medicamentos / Mucopolissacaridose III Limite: Animals / Humans Idioma: En Revista: Proc Natl Acad Sci U S A Ano de publicação: 2014 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Acetilglucosaminidase / Proteínas Recombinantes de Fusão / Encéfalo / Fator de Crescimento Insulin-Like II / Sistemas de Liberação de Medicamentos / Mucopolissacaridose III Limite: Animals / Humans Idioma: En Revista: Proc Natl Acad Sci U S A Ano de publicação: 2014 Tipo de documento: Article