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Liliana Matos

University of Porto
Instituto de Biologia Molecular e Celular

45

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From bench to bioterium and back again: development of a U1snRNA-based therapeutic strategy for mucopolysaccharidosis IIIC

2020
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Development of an antisense oligonucleotide-mediated exon skipping therapeutic strategy for Mucolipidosis II: validation at RNA level.

2020 Human Gene Therapy
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Age-related oxidative modifications to uterine albumin impair extravillous trophoblast cells function.

2020 Free Radical Biology and Medicine
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Molecular Characterization of a Novel Splicing Mutation underlying Mucopolysaccharidosis (MPS) type VI—Indirect Proof of Principle on Its Pathogenicity

2020
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From bench to bioterium and back again: development of a U1 SNRNA-based therapeutic strategy for mucopolysaccharidosis III

2020
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The use of a modified U1 snRNA as a therapeutic strategy to correct a 5’ splice-site mutation in Mucopolysaccharidosis IIIC: in vitro steps towards an in vivo approach

2019
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Development of RNA based approaches to exploit alternative therapies for Lysosomal Storage Diseases

2019
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RNA Therapeutics: How Far Have We Gone?

2019 Advances in Experimental Medicine and Biology
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Next Generation Sequencing and Lysosomal Dysfunction: Novel mutations associated with Neurodegenerative disorders.

2018
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Development of an antisense-mediated exon skipping therapeutic strategy to correct a frequent causing mutation in Mucolipidosis II

2017
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