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IDS Crossing of the Blood-Brain Barrier Corrects CNS Defects in MPSII Mice

Mucopolysaccharidosis type II (MPSII), or Hunter syndrome, arises from a deficiency in iduronate 2-sulfatase (IDS), and it is characterized by progressive somatic and neurological involvement. The MPSII mouse model reproduces the features of MPSII patients. Systemic administration of the AAV2/5CMV-h...

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Sonraí Bibleagrafaíochta
Main Authors: Polito, Vinicia Assunta, Cosma, Maria Pia
Formáid: Artigo
Teanga:Inglês
Foilsithe: Elsevier 2009
Ábhair:
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC2725243/
https://ncbi.nlm.nih.gov/pubmed/19679226
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ajhg.2009.07.011
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