RNA editing as a therapeutic approach for retinal gene therapy requiring long coding sequences
RNA editing aims to treat genetic disease through altering gene expression at the transcript level. Pairing site-directed RNA-targeting mechanisms with engineered deaminase enzymes allows for the programmable correction of G>A and T>C mutations in RNA. This offers a promising therapeutic appro...
| Main Authors: | , , , , |
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| Format: | Journal article |
| Language: | English |
| Published: |
MDPI
2020
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