Repairing RNA
Splicing-Directed Repair™
One design, many mutations
One construct is designed to cover many different mutations in the same gene.
Works at the RNA level
Nothing permanent is written to the genome.
Uses the cell's own spliceosome
No non-human protein for the immune system to see.
Follows the gene's own expression
The design restricts protein to the cells that already express the gene.
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