ArticleHuman gene therapy2017

Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I.

Lalitha R Belur et al.PubMed ↗Full text ↗Publisher ↗

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