For example, non-random inactivation of the X chromosome that carries the defective Wiskott-Aldrich syndrome gene (mutation of which causes a life-threatening immunodeficiency in affected males ...

Italian research charity Telethon Foundation is one step closer to getting a gene therapy to treat rare disease ...

Researchers analyzed 53 patients treated with lentiviral HSC-GT for metachromatic leukodystrophy, Wiskott-Aldrich syndrome, or β-thalassemia. Patients were followed for up to eight years ...

In some cases, people can inherit lymphocytopenia. For example, people with Wiskott-Aldrich syndrome have genetic mutations that reduce the body’s capacity to produce lymphocytes. Doctors will ...