Rice bioengineer wins NIH support for a new strategy to fight cystic fibrosis


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….

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