Genetic correction of muscular stem cells

Research type

Laboratorium onderzoek

Start date

01/09/2016

Introduction

A genetic defect in a gene required for energy production or muscle function can result in progressive myopathy and exercise intolerance. Muscle stem cells called pericytes fulfill all criteria to be used as cell therapy to improve muscle mass and strength.

Description research

In order to use pericytes for autologous cell therapy in persons with an inheritable myopathy, we first need to correct the genetic defect. The CRISPR/Cas9 technology is a method to correct a genetic defect in cells with high specificity. The goal of this project is to apply genetic correction of pericytes using the CRISPR/Cas9 method and correct pericytes of patients with a mutation in TMEM126b, LAMA2 or DMPK1.