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

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

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

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

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

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 ...

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

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

A renowned Harvard hematologist, Maureen Okam Achebe, has called for the institutionalisation of continuous newborn screening ...

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

MiNA Therapeutics Limited, the pioneer in small activating RNA (RNAa) therapeutics, announced today that it will present new pre-clinical data on its RNA activation program for the treatment of sickle ...