Open AccessSplice Modulating Therapies for Human Disease
Author(s) -
Pietro Spitali,
Annemieke AartsmaRus
Publication year - 2012
Publication title -
cell
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 26.304
H-Index - 776
eISSN - 1097-4172
pISSN - 0092-8674
DOI - 10.1016/j.cell.2012.02.014
Subject(s) - biology , splice , disease , alternative splicing , computational biology , genetics , gene , exon , medicine
Dysregulation of splicing and alternative splicing underlies many genetic and acquired diseases. We present an overview of recent strategies and successes in modulating splicing therapeutically in clinical and preclinical contexts. Effective approaches include restoring open reading frames, influencing alternative splicing, and inducing exon inclusion to generate beneficial proteins and remove deleterious ones.
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