Social•Apr 14, 2026
In‑vivo Base Editing Rescues Zellweger Disorder in Mice
Today in @natBME we report an in vivo base editing strategy that corrects a common disease-causing mutation and rescues pathology in a mouse model of Zellweger spectrum disorder (ZSD) and restore peroxisomal function in patient derived cells. This work highlights the promise of precision genome editing for treating peroxisomal biogenesis disorders. 1/13
By David Liu
Social•Feb 23, 2026
Prime-Edited Therapy Restores Immune Function in Teen
Below is the story of the first patient treated with a prime-edited therapeutic, developed by @PrimeMedicine in a trial led by Dr. Élie Haddad and his team at CHU Sainte-Justine. This teenager suffered from chronic granulomatous disease (CGD), an immunodeficiency,...
By David Liu
