CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)-Reference-Cited by-同舟云学术

CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)

Published:2016-06-16 Issue:9 Volume:1 Page:
ISSN:2379-3708
Container-title:JCI Insight
language:en
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Author:

Motas Sandra,Haurigot Virginia,Garcia Miguel,Marcó Sara,Ribera Albert,Roca Carles,Sánchez Xavier,Sánchez Víctor,Molas Maria,Bertolin Joan,Maggioni Luca,León Xavier,Ruberte Jesús,Bosch Fatima

Publisher

American Society for Clinical Investigation

Subject

General Medicine

Reference86 articles.

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2. Overview of the mucopolysaccharidoses

3. Japan Elaprase® Treatment (JET) study: Idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS II)

4. Mucopolysaccharidosis type II (Hunter disease): 13 gene mutations in 52 Japanese patients and carrier detection in four families;Yamada;Hum Genet,1993

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