[HTML][HTML] Splice modulating therapies for human disease
P Spitali, A Aartsma-Rus - Cell, 2012 - cell.com
Cell, 2012•cell.com
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.
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.
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.
cell.com
