Abstract：
: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.
journal_name
Celljournal_title
Cellauthors
Spitali P,Aartsma-Rus Adoi
10.1016/j.cell.2012.02.014subject
Has Abstractpub_date
2012-03-16 00:00:00pages
1085-8issue
6eissn
0092-8674issn
1097-4172pii
S0092-8674(12)00214-0journal_volume
148pub_type
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