Genetic carrier screening is conducted to determine if an asymptomatic person is a carrier for a potentially inheritable pathogenic gene.

A Falmouth teen, Jayden Wilsey, is the first to receive gene therapy to treat sickle cell at Boston Children's Hospital.

Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using ...

KJ Muldoon's life-threatening disorder, called severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, was treated with ...

But researchers were hopeful a larger, ongoing trial will prove the approach effective. Hemophilia is a bleeding disorder caused by a genetic mutation. In the most common form -- hemophilia A -- ...

Genetic testing for hereditary cancer unexpectedly uncovers a case of myelodysplastic syndrome (MDS), highlighting the need ...

Researchers from the Centenary Institute have uncovered a reason why some people with hemophilia A develop resistance to ...

Jim Wilson‘s pioneering gene therapy explorations have helped launch eight companies focused on translating his 40 years of ...

Genentech’s short film draws on the experiences of multiple families caring for children with hemophilia A, seeking to ...

Cell and gene therapies harbor great hopes for biomedicine. They are becoming increasingly important for diseases that are ...

The death of a patient in a trial of an experimental gene therapy casts new shadows over a technology once seen as the future ...

A man’s acquired hemophilia coincided with a rare liver disease, a connection that had not been reported before, per a case ...