Many scientists think that other effective cancer treatments might be created through the same idea: Target the defectiveprotein that causes the disease.
Kalydeco works by correcting the protein that is defective in cystic fibrosis, called the cystic fibrosis transmembrane conductance regulator, which helps move salt ions across cells.
This suggests that those people with an aberrant form of the protein (the result of the defective gene) might be more resistant to herpes infection than their fellows.
At Bristol Gregg had realized that a protein called the microsomal triglyceride transfer protein (MTP) had gone defective in his old patients, and he set out to come up with a drug that blocked it in heart disease patients.
The reason kids like Arya live at all is that humans have a slightly defective cousin of the SMA gene that produces only enough nourishing protein to keep patients alive for a while.
The theory is that the protein in sperm which triggers chemical changes in the egg, leading to embryo development, is defective or missing from the sperm of some infertile men.