Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome.

Kan, Shih-Hsin; Elsharkawi, Ibrahim; Le, Steven Q; Prill, Heather; Mangini, Linley; Cooper, Jonathan D; Lawrence, Roger; Sands, Mark S; Crawford, Brett E; Dickson, Patricia I

Kan, Shih-Hsin; Elsharkawi, Ibrahim; Le, Steven Q; Prill, Heather; Mangini, Linley; Cooper, Jonathan D; Lawrence, Roger; Sands, Mark S; Crawford, Brett E; Dickson, Patricia I.

Afiliación

Kan SH; Department of Pediatrics, The Lundquist Institute (formally Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, CA 90502, United States of America; CHOC Research Institute, Orange, CA 92868, United States of America. Electronic address: skan@choc.org.
Elsharkawi I; Department of Pediatrics, Washington University School of Medicine in St. Louis, St. Louis, MO 63110, United States of America.
Le SQ; Department of Pediatrics, The Lundquist Institute (formally Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, CA 90502, United States of America; Department of Pediatrics, Washington University School of Medicine in St. Louis, St. Louis, MO 63110, United States of A
Prill H; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, United States of America.
Mangini L; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, United States of America.
Cooper JD; Department of Pediatrics, The Lundquist Institute (formally Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, CA 90502, United States of America; Department of Pediatrics, Washington University School of Medicine in St. Louis, St. Louis, MO 63110, United States of A
Lawrence R; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, United States of America.
Sands MS; Department of Pediatrics, Washington University School of Medicine in St. Louis, St. Louis, MO 63110, United States of America.
Crawford BE; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, United States of America.
Dickson PI; Department of Pediatrics, The Lundquist Institute (formally Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, CA 90502, United States of America; Department of Pediatrics, Washington University School of Medicine in St. Louis, St. Louis, MO 63110, United States of A

Mol Genet Metab ; 133(2): 185-192, 2021 06.

Article en En | MEDLINE | ID: mdl-33839004

Asunto(s)

Acetilglucosaminidasa/genética; Terapia de Reemplazo Enzimático; Factor II del Crecimiento Similar a la Insulina/genética; Mucopolisacaridosis III/terapia; Animales; Animales Recién Nacidos; Modelos Animales de Enfermedad; Perros; Heparitina Sulfato/metabolismo; Humanos; Infusiones Intraventriculares; Ratones; Ratones Noqueados; Mucopolisacaridosis III/enzimología; Mucopolisacaridosis III/genética; Mucopolisacaridosis III/patología; Enfermedades del Sistema Nervioso; Proteínas Recombinantes de Fusión/genética; Proteínas Recombinantes de Fusión/farmacología

Palabras clave

Heparan sulfate; Intracerebroventricular enzyme replacement therapy (ICV-ERT); Mucopolysaccharidosis IIIB; Neonatal mice; Sanfilippo syndrome type B

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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Acetilglucosaminidasa / Factor II del Crecimiento Similar a la Insulina / Mucopolisacaridosis III / Terapia de Reemplazo Enzimático Tipo de estudio: Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Mol Genet Metab Asunto de la revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Año: 2021 Tipo del documento: Article

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