Antisense therapy restores fragile X protein production in human cells

Joel D. Richter, PhD, and Sneha Shah, PhD

image: Joel D. Richter, PhD, and Sneha Shah, PhD visualization Moreover Credit: UMass Chan Medical School An antisense therapy developed by Joel D. Richter, PhD, Sneha Shah, PhD, and Jonathan K. Watts, PhD, at UMass Chan Medical School and Elizabeth Berry-Kravis, MD, PhD, at RUSH University Medical Center, restores production of the FMRP protein in … Read more

A new gene-editing method tags fragile X mutation for repair

Minimization of mutations: Promoting the formation of specialized loops in the genome may help remove the genetic repeats associated with fragile X syndrome. THOM LEACH / Scientific Source Hun-Goo Lee didn’t set out to discover a new way to potentially treat fragile X syndrome. He just wanted to solve a mystery: why do some … Read more