Article
Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases.
Nucleic acid therapeutics - 1 Feb 2014
Siva Kavitha, Covello Giuseppina, Denti Michela A
Abstract excerpt
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...
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