Genetically Modulated Substrate Reduction Therapy for Sanfilippo Syndrome: Proof of Principle

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GENETICALLY MODULATED SUBSTRATE REDUCTION THERAPY for SANFILIPPO SYNDROME.pdf		1.97 MB	Adobe PDF	Ver/Abrir

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About proof of principle in genetically modulated substrate reduction therapy for Sanfilippo syndrome.

Lysosomal Storage Diseases Mucopilysaccharidoses Substrate Reduction Therapy RNAi Doenças Genéticas Genética Humana