Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would advance one day.

A 21-year-old man has become the first person in New York—and one of the first in the world—to be cured of sickle cell anemia. A novel form of gene therapy enabled the man to receive a life ...

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

Victoria Gray is the first person in the world to receive CRISPR,  a gene-editing therapy for sickle cell disease created by ...

BEAM-101, a genetically modified cell therapy for sickle cell, is designed to turn on HbF to counteract mutated adult ...

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

Orphan Drug Designation is awarded to therapies intended to treat rare diseases that affect fewer than 200,000 people in the ...

Gene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments has ...

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

Greater numbers of circulating stem cells increase the efficacy of in vivo gene therapy techniques, potentially reducing the ...

Sickle Cell disease disproportionately affects India’s tribal populations, with complications impacting both children and ...

Though Beauzile is the first in New York to successfully undergo sickle cell anemia-curing gene therapy, he's preceded by Kendric Cromer, a 12-year-old boy from Washington, DC who received ...