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Splicing modulation therapy in the treatment of genetic diseases

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 ver...

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Библиографические подробности
Опубликовано в: :Appl Clin Genet
Главные авторы: Arechavala-Gomeza, Virginia, Khoo, Bernard, Aartsma-Rus, Annemieke
Формат: Artigo
Язык:Inglês
Опубликовано: Dove Medical Press 2014
Предметы:
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC4259397/
https://ncbi.nlm.nih.gov/pubmed/25506237
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/TACG.S71506
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