Effects of Mini-Dystrophin on Dystrophin-Deficient, Human Skeletal Muscle-Derived Cells.

ABSTRACT:

Background

We are developing a novel therapy for Duchenne muscular dystrophy (DMD), involving the transplantation of autologous, skeletal muscle-derived stem cells that have been genetically corrected to express dystrophin. Dystrophin is normally expressed in activated satellite cells and in differentiated muscle fibres. However, in past preclinical validation studies, dystrophin transgenes have generally been driven by constitutive promoters that would be active at every stage of the myogenic differentiation process, including in proliferating muscle stem cells. It is not known whether artificial dystrophin expression would affect the properties of these cells.

Aims

Our aims are to determine if mini-dystrophin expression affects the proliferation or myogenic differentiation

SUBMITTER: Meng J 

PROVIDER: S-EPMC7582244 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature