Inhaled drug could treat rare cystic fibrosis mutations
SpliSense has demonstrated in human-derived cells that it can generate a functioning protein to replace one that is defective in 20% of CF cases.
SpliSense has demonstrated in human-derived cells that it can generate a functioning protein to replace one that is defective in 20% of CF cases.
Israeli parents and scientists are actively working on new treatments for some of the 7,000 known inherited diseases.