UCLA researchers have made strides in developing a stem cell gene therapy for alpha thalassemia major, a severe, inherited blood disorder in which patients lack the gene needed to produce hemoglobin.

The therapy involves collecting a patient’s own blood stem cells, adding the missing alpha-globin gene using a viral vector, and then returning the corrected cells to the patient.

In patient cells in culture, the gene therapy successfully restored normal alpha-globin protein production to healthy levels, enabling the modified cells to produce functional hemoglobin.

Researchers have developed an AI model that estimates long-term disease risk across more than 1,000 medical conditions

The model, trained and tested on anonymised medical data from the UK and Denmark, can forecast health outcomes over a decade in advance

While not ready for direct clinical use, the AI model offers new ways to study disease and inform healthcare strategies