But the Cystic Fibrosis Trust said the medicine was "transformational", had a "dramatic effect" and that experts say "no other medicine addresses the basic geneticdefect in cystic fibrosis".
The vast majority of colorblind people have trouble seeing red or green, due to a geneticdefect in the color-sensing cells, called cones, at the back of the eye.
Isis needs one in order to start a large and expensive clinical trial testing its drug in patients who have high cholesterol that is not caused by a geneticdefect.
Nowell's research at Penn in Philadelphia was the first to show that a geneticdefect could be responsible for cancer, His work has led to numerous discoveries into the growth of cells related to cancers and other disorders.
In recent experiments carried out by Chiara Conti and other members of Dr Bensimon's team, molecular combing was used to identify a geneticdefect responsible for cancer of the kidney, which is notoriously difficult to diagnose in its early (and therefore treatable) stages.