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

Genetic therapy directed towards the correction of RNA missplicing is being investigated not only at basic research level but even in late-stage clinical trials. Many mutations that change the normal splicing pattern and lead to aberrant mRNA production have been identified in Lysosomal Storage Diso...

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Bibliographic Details
Main Author: Matos, L. (author)
Other Authors: Santos, J.I. (author), Rocha, M. (author), Coutinho, M.F. (author), Prata, M.J. (author), Alves, S. (author)
Format: conferenceObject
Language:eng
Published: 2020
Subjects:
Lysosomal Storage Disorders
RNA Therapies
Terapias de RNA
Terapias de Splicing
U1 snRNA
MPS IIIC
Doenças Lisossomais de Sobrecarga
Doenças Genéticas
Online Access:http://hdl.handle.net/10400.18/6792
Country:Portugal
Oai:oai:repositorio.insa.pt:10400.18/6792

Internet

http://hdl.handle.net/10400.18/6792

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