Splicing Modulation as a Promising Therapeutic Strategy for Lysosomal Storage Disorders: The Mucopolysaccharidoses Example

Over recent decades, the many functions of RNA have become more evident. This molecule has been recognized not only as a carrier of genetic information, but also as a specific and essential regulator of gene expression. Different RNA species have been identified and novel and exciting roles have bee...

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Bibliographic Details
Main Author: Santos, Juliana Inês (author)
Other Authors: Gonçalves, Mariana (author), Matos, Liliana (author), Moreira, Luciana (author), Carvalho, Sofia (author), Prata, Maria João (author), Coutinho, Maria Francisca (author), Alves, Sandra (author)
Format: article
Language:eng
Published: 2022
Subjects:
lysosomal storage diseases (LSDs)
mucopolysaccharidoses (MPSs)
RNA-based therapies
antisense oligonucleotides (ASOs)
splice-switching oligonucleotides (SSOs)
U1 snRNA (small nuclear RNA)
Online Access:http://hdl.handle.net/10316/103355
Country:Portugal
Oai:oai:estudogeral.sib.uc.pt:10316/103355

Internet

http://hdl.handle.net/10316/103355

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