Sumary of Rice bioengineer wins NIH support for a new strategy to fight cystic fibrosis:
- Chemical and biomolecular engineer Xue Sherry Gao of Rice University’s Brown School of Engineering has won National Institutes of Health support for a new strategy to fight cystic fibrosis..
- The agency awarded Gao a four-year R01 grant of more than $2 million to adapt tools developed by her lab that increase the accuracy of CRISPR/Cas9-based gene editing to repair mutations responsible for the disease that affects 70,000 people worldwide..
- Cystic fibrosis is a hereditary disease that could be caused by single-point mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that normally regulates chloride and bicarbonate transportation..
- Law Assistant Professor of Chemical and Biomolecular Engineering and an assistant professor of bioengineering and of chemistry..
- “Researchers have checked genetic mutations in this specific gene, and they found there are more than 2,000 variations..
- Not all of them lead to the disease, but it’s believed more than 350 of the mutations cause cystic fibrosis.”.
- While gene therapy often aims to replace an entire faulty gene with a good one, Gao’s tools are intended to correct single-point mutations..
- Gao has experience with the concept, having developed gene editing to treat hearing loss from genetic mutations, as described in a 2018 Nature paper….