mRNA trans‐splicing in gene therapy for genetic diseases
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 exogeno...
| Main Authors: | , , , , , |
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| Format: | Online |
| Language: | English |
| Published: |
John Wiley & Sons, Inc.
2016
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| Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5071737/ |