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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...
Autores principales: | , , , , , |
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Formato: | Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2010
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2989894/ https://www.ncbi.nlm.nih.gov/pubmed/20876612 http://dx.doi.org/10.1093/hmg/ddq420 |