This program is exploring ways to correct common and rare mutations in the CFTR gene using CRISPR/Cas approaches. These technologies aim to edit DNA so that the genetic defect that leads to a faulty CFTR protein is fixed.
Status
Laboratory studies to develop and test these technologies are underway.
Sponsor
This program is sponsored by Editas Medicine and partially funded by Cystic Fibrosis Foundation Therapeutics (CFFT).
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