Two patients who received Sangamo’s zinc finger–based treatment for Hunter syndrome have lower biomarkers of the condition, but no signs of new enzyme production.

A mouse study adds to the growing body of work disputing the ability of progenitor cells to regenerate muscle tissue in adult mammals’ hearts.

The Germany-based study will test an ex vivo genome-editing therapy for the inherited blood disorder β-thalassemia.

Without knowing the cells’ mechanism of action, researchers question the best way to administer them to patients.

Scientists race to develop CRISPR therapies that could save the lives of kids with muscle-wasting conditions.

Investigators have launched numerous clinical trials that test the efficacy of the immunotherapy. Here is a global accounting of these experiments.

Epidiolex, a cannabis-derived medicine for epilepsy, will prompt federal regulatory changes that could crack open access to study the plant.

The agency proposes ceding its scrutiny of these studies to the FDA.

The medicine increases the activity of a deficient enzyme in certain patients with the condition.