With gene therapy offering new hope for thalassemia, experts urge Indian govt to invest in early diagnosis, genetic testing, ...

Thalassemia, a group of inherited blood disorders characterized by reduced or abnormal hemoglobin production, continues to ...

Geneticists have used a cutting-edge DNA base editing treatment on several patients to cure thalassemia, a prevalent and severe genetic blood disease.

A four-year-old Pakistani girl diagnosed with severe thalassemia has been successfully treated with a Chinese-developed ...

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

Editas Medicine reports successful gene editing levels in stem cells, supporting potential treatment for sickle cell disease and beta thalassemia. Editas Medicine announced promising new data on ...

G ene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments has been challenging.

In medical breakthrough, a baby diagnosed with rare genetic disorder is thriving after customized CRISPR gene editing therapy ...

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

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

Beam Therapeutics rises after the FDA grants orphan drug status to its gene-editing therapy BEAM-101 for sickle cell disease.