The current emphasis, in both the scientific literature and mass media, on the promise of gene therapy and therapeutic cloning has blinded researchers and the public alike to the best way forward in treating genetic diseases. We could not, and have not, waited for genes and their mutations to be identified and for futuristic therapies to be devised. Quietly and efficiently, conventional treatments and symptomatic management have had a growing impact on quality of life and life expectancy of patients with a range of genetic conditions. These are today's treatments for genetic disease.
Patients, after all, suffer not from their mutations but from the functional consequences of these mutations. Several genetic diseases were treatable long before the age of molecular genetics. We did not have to wait for cloning of the phenylalanine hydroxylase gene to treat phenylketonuria with a low-protein diet. Since the 1970s, more than 20 million French babies ...
