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

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

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

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

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.

Cell and gene therapy (C&GT) is set to revolutionise treatment of complex diseases, offering new possibilities for patients.

So, when Quartey learned of a newly FDA-approved gene therapy to treat Sickle Cell ... affecting about 65% of people with the disease — or beta thalassemia, a blood disorder that reduces the ...

Gene therapy treatments ... sickle cell disease and beta thalassemia, which affect tens of thousands of patients in the U.S. Those treatments are sold by major pharmaceutical and biotechnology ...

Editas Medicine reports successful gene editing levels in stem cells, supporting potential treatment for sickle cell disease and beta thalassemia ... of Gene and Cell Therapy in New Orleans.