Can Gene Therapy Treat Dominantly Inherited Disorders?

Research

November 16, 2018

Big Gains in FSHD Research: A Newly Published Model of FSHD and a Potential Gene Therapy to Improve Functional Outcomes

The model aims to provide the basis for many future studies to bring therapeutic options to patients with FSHD.

Research

July 24, 2018

Preclinical Gene Therapy Study Shows Muscle Restoration in Charcot-Marie-Tooth Disease Model

Tests of NT-3 gene therapy on mouse models suggest potential to treat a host of muscle-wasting conditions.