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Exon-Skipping Antisense Oligonucleotides to Correct Missplicing in Neurogenetic Diseases

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...

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Autors principals: Siva, Kavitha, Covello, Giuseppina, Denti, Michela A.
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2014
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3922311/
https://ncbi.nlm.nih.gov/pubmed/24506781
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/nat.2013.0461
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