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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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Päätekijät: | , , , , , |
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Aineistotyyppi: | Artigo |
Kieli: | Inglês |
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Oxford University Press
2010
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Linkit: | 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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