Publication | Open Access

Functional analysis of splicing mutations in the IDS gene and the use of antisense oligonucleotides to exploit an alternative therapy for MPS II

GeneticsOligonucleotideMolecular BiologyMps IiMolecular GeneticsAntisense TherapyDisease Gene IdentificationIds GeneFunctional AnalysisMedicineSplicing VariantGenome Editing