Splicing modulation therapy in the treatment of genetic diseases
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چکیده
منابع مشابه
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 versatility of the approach to correct cryptic splicing, modulate alternative splicing, restore the ...
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Spliceosome-mediated RNA trans-splicing, or SMaRT, is a promising strategy to design innovative gene therapy solutions for currently intractable genetic diseases. SMaRT relies on the correction of mutations at the post-transcriptional level by modifying the mRNA sequence. To achieve this, an exogenous RNA is introduced into the target cell, usually by means of gene transfer, to induce a splice ...
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ژورنال
عنوان ژورنال: The Application of Clinical Genetics
سال: 2014
ISSN: 1178-704X
DOI: 10.2147/tacg.s71506