Skipping the Pathology in Rare Diseases: Antisense exon-skipping therapy for Mucolipidosis type II

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About Skipping the Pathology in Rare Diseases: Antisense exon-skipping therapy for Mucolipidosis type II

Doenças Genéticas Genética Humana Mucolipidosis type II RNA Therapeutics Lysosomal Storage Disorders Exon Skipping Oligonucleotide-based Therapies