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Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy

Mucopolysaccharidosis type II (MPSII), or Hunter syndrome, is a devastating disorder associated with a shortened life expectancy. Patients affected by MPSII have a variety of symptoms that affect all organs of the body and may include progressive cognitive impairment. MPSII is due to inactivity of t...

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Main Authors: Polito, Vinicia Assunta, Abbondante, Serena, Polishchuk, Roman S., Nusco, Edoardo, Salvia, Rosaria, Cosma, Maria Pia
Format: Artigo
Jezik:Inglês
Izdano: Oxford University Press 2010
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC2989894/
https://ncbi.nlm.nih.gov/pubmed/20876612
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddq420
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