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List of works by Liliana Matos

Data in support of a functional analysis of splicing mutations in the IDS gene and the use of antisense oligonucleotides to exploit an alternative therapy for MPS II.

scientific article

From bedside to cell biology: a century of history on lysosomal dysfunction.

scientific article published on 29 September 2014

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

scientific article published on 25 September 2015

Genetic Substrate Reduction Therapy: A Promising Approach for Lysosomal Storage Disorders.

scientific article published on 9 November 2016

Therapeutic strategies based on modified U1 snRNAs and chaperones for Sanfilippo C splicing mutations

scientific article published on 10 December 2014

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