Author's posts
A Gene Therapy Appears ToReplaceMissing ProteinIn Muscular Dystrophy Patients
An experimental gene therapy appeared to dramatically increase the production of a muscle-making protein in three young boys with Duchenne muscular dystrophy, a deadly and irreversible disease, according to results being presented by the drugmaker Sare…
Bluebird Bio Gene Therapies Show Promise Against Deadly Anemias
Bluebird Bio’s experimental gene therapy LentiGlobin is helping some patients with two life-threatening forms of inherited anemia avoid blood transfusions.
