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Cell. 2012 Mar 16;148(6):1085-8. doi: 10.1016/j.cell.2012.02.014.

Splice modulating therapies for human disease.

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  • 1Department of Human Genetics, Leiden University Medical Center, 2300 RC Leiden, The Netherlands.


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.

Copyright © 2012 Elsevier Inc. All rights reserved.

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