Yissum Research Development Company of the Hebrew University of Jerusalem Ltd.

Batsheva Kerem

Stephen Donald Wilton

Sue Fletcher

Loren Price

Michal Tur Sinai

Provided are oligonucleotides capable of binding to and modulating the splicing of the pre-mRNA of the CFTR gene, compositions including said oligonucleotides, kits including the compositions, and uses thereof. In particular, the subject matter provides compositions of oligonucleotides useful in methods for suppressing exon skipping optionally in combination with additional CFTR therapeutics.

Restoration of the CFTR function by splicing modulation

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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Mevaseret Zion, Israel

Batsheva Kerem