Human adipose-derived stem cell transplantation as a potential therapy for collagen VI-related congenital muscular dystrophy.

ABSTRACT:

Introduction

Congenital muscular dystrophies (CMD) are a clinically and genetically heterogeneous group of neuromuscular disorders characterized by muscle weakness within the first two years of life. Collagen VI-related muscle disorders have recently emerged as one of the most common types of CMD. COL6 CMD is caused by deficiency and/or dysfunction of extracellular matrix (ECM) protein collagen VI. Currently, there is no specific treatment for this disabling and life-threatening disease. The primary cellular targets for collagen VI CMD therapy are fibroblasts in muscle, tendon and skin, as opposed to muscle cells for other types of muscular dystrophies. However, recent advances in stem cell research have raised the possibility that use of adult stem cells may provide dramatic new t

SUBMITTER: Alexeev V 

PROVIDER: S-EPMC4054951 | biostudies-literature | 2014 Feb

REPOSITORIES: biostudies-literature