Sentences with phrase «hereditary gene defect»

People with haemophilia A, the commonest form of the disease, need infusions of factor 8 because a hereditary gene defect means they can not make their own.

Not exact matches

Philadelphia — Ten years ago this month the promise of using normal genes to cure hereditary defects crashed and burned, as Jesse Gelsinger, an 18 - year - old from Tucson, Ariz., succumbed to multiorgan failure during a gene therapy trial at the University of Pennsylvania.

One possible alternative for treating such hereditary inner ear defects is gene therapy.

The babies are all suffering from a rare hereditary disease called Hurler's syndrome, caused by a defect in the gene that produces the enzyme.

Howard Hughes Medical Institute (HHMI) researchers have identified subtle defects in a single gene that underlie a hereditary form of age - related macular degeneration, the leading cause of...