Minor genetic anomalies can have catastrophic consequences. Some of the “simplest” of genetic diseases—with known defects in a single gene—are still incurable. And fatal.
Chih-Hao “Lucas” Chang's immune system and metabolism research have led him to a prospective treatment option for the autoimmune disease systemic lupus erythematosus (SLE or lupus).
For the first time, there's an FDA approved treatment for Friedreich’s ataxia (FA). This is a major step forward for the FA community, but is something even better on the horizon?
Supported by an NIH grant, Cat Lutz will lead a multi-institutional team to develop and validate new gene editing-based therapeutic approaches for four neurological conditions lacking effective treatments.