Correcting malfunctioning genes could perhaps cure diseases linked to a single gene, such as sickle-cell anemia.

FORBES: Magazine Article

More than 6, 000 of these disorders are rare diseases, most of them caused by a single gene, and fewer than 200 have a drug treatment available.

FORBES: Rare Disease Drugs: A $1 Trillion Market?

Gene therapy's tantalizing attraction is that a single treatment has the potential to cure lethal diseases by enabling normal genes to take over for defective ones.

WSJ: The Future of Medicine Is Now: Medical Innovations