BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pathogenesis.

Yogalingam, Gouri; Luu, Amanda R; Prill, Heather; Lo, Melanie J; Yip, Bryan; Holtzinger, John; Christianson, Terri; Aoyagi-Scharber, Mika; Lawrence, Roger; Crawford, Brett E; LeBowitz, Jonathan H

Yogalingam, Gouri; Luu, Amanda R; Prill, Heather; Lo, Melanie J; Yip, Bryan; Holtzinger, John; Christianson, Terri; Aoyagi-Scharber, Mika; Lawrence, Roger; Crawford, Brett E; LeBowitz, Jonathan H.

Afiliação

Yogalingam G; Research, BioMarin Pharmaceutical, Inc., Novato, CA, United States of America.
Luu AR; Research, BioMarin Pharmaceutical, Inc., Novato, CA, United States of America.
Prill H; Research, BioMarin Pharmaceutical, Inc., Novato, CA, United States of America.
Lo MJ; Research, BioMarin Pharmaceutical, Inc., Novato, CA, United States of America.
Yip B; Research, BioMarin Pharmaceutical, Inc., Novato, CA, United States of America.
Holtzinger J; Research, BioMarin Pharmaceutical, Inc., Novato, CA, United States of America.
Christianson T; Research, BioMarin Pharmaceutical, Inc., Novato, CA, United States of America.
Aoyagi-Scharber M; Research, BioMarin Pharmaceutical, Inc., Novato, CA, United States of America.
Lawrence R; Research, BioMarin Pharmaceutical, Inc., Novato, CA, United States of America.
Crawford BE; Research, BioMarin Pharmaceutical, Inc., Novato, CA, United States of America.
LeBowitz JH; Research, BioMarin Pharmaceutical, Inc., Novato, CA, United States of America.

PLoS One ; 14(1): e0207836, 2019.

Article em En | MEDLINE | ID: mdl-30657762

Assuntos

Acetilglucosaminidase/metabolismo; Endocitose; Fator de Crescimento Insulin-Like II/uso terapêutico; Mucopolissacaridose III/tratamento farmacológico; Mucopolissacaridose III/patologia; Proteínas Recombinantes de Fusão/uso terapêutico; Acetilglucosaminidase/farmacocinética; Acetilglucosaminidase/uso terapêutico; Animais; Astrócitos/metabolismo; Axônios/metabolismo; Cátions; Fibroblastos/metabolismo; Heparitina Sulfato/metabolismo; Hipocampo/patologia; Humanos; Fator de Crescimento Insulin-Like II/farmacocinética; Lisossomos/enzimologia; Microglia/metabolismo; Ratos; Receptor IGF Tipo 2/metabolismo; Proteínas Recombinantes de Fusão/farmacocinética

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Acetilglucosaminidase / Proteínas Recombinantes de Fusão / Fator de Crescimento Insulin-Like II / Mucopolissacaridose III / Endocitose Tipo de estudo: Etiology_studies Limite: Animals / Humans Idioma: En Ano de publicação: 2019 Tipo de documento: Article

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