Vidcast:  https://youtu.be/BM9C_nTcJR0

 

Cystic Fibrosis is due to a genetic mutation that leads to insufficient moisture in body secretions and thickened respiratory mucus.  Geneticists at New York’s Stony Brook University and the Cold Spring Harbor Laboratory have now demonstrated the possibility of using gene therapy with so-called antisense oligonucleotides to create better functioning versions  of the cell water-controlling protein CFTR, short for cystic fibrosis transmembrane conductance regulator.  This will someday result in a set of more normally-functioning lungs, pancreas, liver, gastrointestinal tract, and reproductive organs for those with cystic fibrosis.

 

https://www.nature.com/articles/s41467-022-30668-y

 

#cysticfibrosis #cf #genetherapy #cftr #aso #antisenseoligotides