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

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 Authors: Juliana Inês Santos, Mariana Gonçalves, Liliana Matos, Luciana Moreira, Sofia Carvalho, Maria João Prata, Maria Francisca Coutinho, Sandra Alves
Format: Article
Language:English
Published: MDPI AG 2022-04-01
Series:Life
Subjects:
lysosomal storage diseases (LSDs)
mucopolysaccharidoses (MPSs)
RNA-based therapies
antisense oligonucleotides (ASOs)
splice-switching oligonucleotides (SSOs)
U1 snRNA (small nuclear RNA)
Online Access:https://www.mdpi.com/2075-1729/12/5/608

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https://www.mdpi.com/2075-1729/12/5/608

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