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

Splicing is an essential cellular process to generate mature transcripts from pre-mRNA. One of the most important factors for mRNA transcription is the U1snRNA, a spliceosomal component that recognizes 5’ splicing donor sites (SDS) at specific regions in pre-mRNA. Splicing mutations represent one of...

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Bibliographic Details
Main Author: Santos, Juliana Inês (author)
Other Authors: Matos, Liliana (author), Oliveira, Paula Alexandra (author), Gonçalves, Mariana (author), Pires, Maria João (author), Coutinho, Maria Francisca (author), Prata, Maria João (author), Alves, Sandra (author)
Format: conferenceObject
Language:eng
Published: 2021
Subjects:
Lysosomal Storage Diseases
RNA Therapies
Doenças Lisossomais de sobrecarga
Doenças Genéticas
Terapias de RNA
Online Access:http://hdl.handle.net/10400.18/7462
Country:Portugal
Oai:oai:repositorio.insa.pt:10400.18/7462

Internet

http://hdl.handle.net/10400.18/7462

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