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 ...

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 ...

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.

CRISPR Therapeutics has the potential to generate significant breakthroughs in gene editing. Regeneron's biggest growth ...

Discover how gene editing and lab-grown meat are revolutionising healthcare and food in 2025, offering personalised ...

bluebird bio has its first ever product approval, getting a green light from the EMA for Zynteglo, its ex vivo gene therapy ... beta-globin, a haemoglobin component that is deficient in ...

Vertex Pharmaceuticals Incorporated today announced data across multiple studies demonstrating positive clinical and quality of life benefits of treatment with CFTR modulators and, in particular, ...

Objectives This study aimed to assess the knowledge gaps regarding the benefits and risks of chelation therapy on thalassemia ... caregivers of beta thalassemia major patients. In India, the mean ...

Researchers have identified an early postnatal window that allows gene transfer to circulating blood stem cells, advancing ...