Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts.
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Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III.Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923-2016).Emerging drugs for the treatment of mucopolysaccharidoses.Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice.Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy.How close are we to therapies for Sanfilippo disease?Phase I and II clinical trials for the mucopolysaccharidoses.Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy.Brain-targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms.
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Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts.
description
article científic
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article scientifique
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articolo scientifico
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artigo científico
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bilimsel makale
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scientific article published on March 2014
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
Insulin-like growth factor II ...... ridosis type IIIB fibroblasts.
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Insulin-like growth factor II ...... ridosis type IIIB fibroblasts.
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type
label
Insulin-like growth factor II ...... ridosis type IIIB fibroblasts.
@en
Insulin-like growth factor II ...... ridosis type IIIB fibroblasts.
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prefLabel
Insulin-like growth factor II ...... ridosis type IIIB fibroblasts.
@en
Insulin-like growth factor II ...... ridosis type IIIB fibroblasts.
@nl
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Insulin-like growth factor II ...... ridosis type IIIB fibroblasts.
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Amanda K Todd
Ariana Di Stefano
Brigette L Tippin
Carolyn S Sinow
Karyn Haitz
Larisa A Troitskaya
Patricia I Dickson
Shih-hsin Kan
Steven Q Le
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P304
P356
10.1042/BJ20130845
P407
P577
2014-03-01T00:00:00Z