Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases.
Nucleic Acid Ther
; 24(1): 69-86, 2014 Feb.
Article
en En
| MEDLINE
| ID: mdl-24506781
ABSTRACT
Alternative splicing is an important regulator of the transcriptome. However, mutations may cause alteration of splicing patterns, which in turn leads to disease. During the past 10 years, exon skipping has been looked upon as a powerful tool for correction of missplicing in disease and progress has been made towards clinical trials. In this review, we discuss the use of antisense oligonucleotides to correct splicing defects through exon skipping, with a special focus on diseases affecting the nervous system, and the latest stage achieved in its progress.
Texto completo:
1
Colección:
01-internacional
Asunto principal:
Oligonucleótidos Antisentido
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Reparación del Gen Blanco
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Enfermedades Genéticas Congénitas
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Enfermedades del Sistema Nervioso
Límite:
Animals
/
Humans
Idioma:
En
Revista:
Nucleic Acid Ther
Año:
2014
Tipo del documento:
Article
País de afiliación:
Italia