Search Results for: gene therapy
Sickle cell gene therapy to boost fetal hemoglobin: A 70-year timeline of discovery
Boston Children’s Hospital is now enrolling patients age 3 to 35 in a clinical trial of gene therapy for sickle cell …Read More
Gene therapy halts progression of cerebral adrenoleukodystrophy in clinical trial
Adrenoleukodystrophy — depicted in the 1992 movie “Lorenzo’s Oil” — is a genetic disease that most severely affects boys. Caused by …Read More
Landmark moment for science as the FDA approves a gene therapy for the first time
Today, the Food and Drug Administration approved a gene therapy known as CAR T-cell therapy that genetically modifies a patient’s own cells …Read More
BCL11A-based gene therapy for sickle cell disease passes key preclinical test
Research going back to the 1980s has shown that sickle cell disease is milder in people whose red blood cells carry a …Read More
Wine used to toast CGD gene therapy trial linked to decades-long scientific journey
When Brenden Whittaker of Columbus, Ohio, the first patient treated with gene therapy for chronic granulomatous disease (CGD), showed successful engraftment …Read More
Forty years waiting for a cure: ALD gene therapy trial shows early promise
A small piece of notepaper, folded twice, sits tucked in a slot of the secretary desk in the living room. …Read More
Gene therapy restores hearing in deaf mice
More than 70 different genes are known to cause deafness when mutated. Jeffrey Holt, PhD, envisions a day when patients with …Read More
Gene therapy gets in the ring with another disease
Seeing that his mother, Kadriye, wasn’t looking, Emir Seyrek got an impish grin on his face, the kind only a …Read More
Gene therapy: Two years and holding
For the Cáceres family of Argentina, it’s a joyous holiday homecoming. Agustín, who received gene therapy at 5½ months of age, journeyed …Read More
A new start for gene therapy for ‘bubble boy’ disease: First U.S.-treated patient doing well
Until this month, Agustín Cáceres’s baptism was the only time his family could come close to him. Everyone had to …Read More