Generation of an isogenic iPSC-based model of Alexander’s Disease using genome editing
Alexander’s Disease is a progressive neurological disorder caused by a wide array of dominant, gain of function mutations in the GFAP gene. The disorder is characterized by the accumulation of GFAP in astrocytes, leading to the formation of protein inclusion bodies known as Rosenthal fibers. This results in a disruption of astrocytic function and the subsequent degeneration of white matter. Howeve
