The TRIKAFTA to treat the Cystic fibrosis

Authors

  • Ziye Zhao Author

DOI:

https://doi.org/10.61173/8y42kv18

Keywords:

Cystic fibrosis, TRIKAFTA, CFTR, F508del mutation, gene therapy

Abstract

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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Published

2024-10-29

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Section

Articles