Splice modulating therapies for human disease.
review · Level V
Where this comes from
- Record sourced from PubMed, PMID 22424220.
- Also identified by DOI 10.1016/j.cell.2012.02.014.
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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.
Medical subject headings
- Disease
- Genetic Therapy
- RNA Splicing