Restoration of the CFTR function by splicing modulation
Abstract:
Oligonucleotides capable of binding to and modulating the splicing of the pre-mRNA of the CFTR gene, compositions including the oligonucleotides, kits including the compositions, and uses thereof. Compositions of oligonucleotides useful in methods for suppressing exon 10 skipping optionally in combination with additional CFTR therapeutics.
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