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Splicing Modulation Therapy In The Treatment Of...

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Splicing Modulation Therapy In The Treatment Of Genetic Diseases
Author: Virginia Arechavala-gomeza, Bernard Khoo, Annemieke Aartsma-rus
Publisher: Derivative Works
8 pages
One time payment: €0.00
Required subscription: Free
Type of publication: Article
ISBN/ISSN: 1178-704X
DOI: 10.2147/TACG.S71506
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Description:

Abstract: Antisense-mediated splicing modulation is a tool that can be exploited in several ways to provide a potential therapy for rare genetic diseases. This approach is currently being tested in clinical trials for Duchenne muscular dystrophy and spinal muscular atrophy. The present review outlines the versatility of the approach to correct cryptic splicing, modulate alternative splicing, restore the open reading frame, and induce protein knockdown, providing examples of each. Finally, we outline a possible path forward toward the clinical application of this approach for a wide variety of inherited rare diseases.

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