The TRIKAFTA to treat the Cystic fibrosis
DOI:
https://doi.org/10.61173/8y42kv18Keywords:
Cystic fibrosis, TRIKAFTA, CFTR, F508del mutation, gene therapyAbstract
Cystic fibrosis currently has limited treatment options. This review focuses on the therapeutic potential of TRIKAFTA for treating this disease. We have compiled data from the National Institutes of Health (NIH) and analyzed the composition and mechanism of action of TRIKAFTA. Specifically, we examine how TRIKAFTA targets the F508del mutation, a common genetic variant in cystic fibrosis that results in the deletion of three nucleotides, leading to the loss of phenylalanine at position 508 of the CFTR protein. Our findings indicate that the components of TRIKAFTA effectively address this mutation. While this review provides a general research direction, further studies are necessary to fully understand and treat cystic fibrosis.
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