The Role of Stem Cells in Muscular Dystrophies

Abstract

Muscular dystrophies are heterogeneous neuromuscular disorders of inherited origin, including Duchenne muscular dystrophy (DMD). Cell-based therapies were used to promote muscle regeneration with the hope that the host cells repopulated the muscle and improved muscle function and pathology. Stem cells were preferable for therapeutic applications, due to their capacity of self-renewal and differentiative potential. In the last years, encouraging results were obtained with adult stem cells to treat muscular dystrophies. Adult stem cells were found into various tissues of the body and they were able to maintain, generate, and replace terminally differentiated cells within their own specific tissue because of cell turnover or tissue injury. Moreover, it became clear that these cells could participate into regeneration of more than just their resident organ. Here, we described multiple types of muscle and non muscle-derived myogenic stem cells, their characterization and their possible use to treat muscular dystrophies. We also underlined that most promising possibility for the management and therapy of DMD is a combination of different approaches, such as gene and stem cell therapy.

CD117+amniotic fluid stem cells

“…62 The identification of a stem cell population providing efficient muscle regeneration is critical for the progression of cell therapy for muscle diseases. 63 Despite early data reporting AFS cell capacity of expressing molecular markers of muscle differentiation under specific culture conditions, 35 high impact papers have just been very recently published. First, Gekas et al 48 demonstrated that human, 2nd trimester, CD117 + -selected AFS cells can acquire a myogenic-like phenotype (i.e., expression of desmin and MyoD) after culture in myogenic-specific induction conditions (i.e., culture on plastic plates coated with Matrigel and 3 mM 5-aza-2'-deoxycytidine).…”

Stem Cell Therapy in Pediatric Neurological Disabilities

“…Continuous cycles of degeneration and regeneration of muscle fibers exhausts the muscle stem cell pool, leading to muscle being replaced by adipose and fibrotic tissue. Stem cell therapy holds great promise as a treatment for Duchenne muscular dystrophy by providing cells that can both deliver functional muscle proteins and replenish the stem cell pool [72].…”

Recent advances in Duchenne muscular dystrophy

“…69 In recent years, a number of tissue-specific adult stem cells, which maintain, generate, and replace terminally differentiated cells within their resident organ, have demonstrated myogenic potential. 53 Among the most promising are adult MSCs, which can differentiate to form myogenic cells in situ. 20 In contrast to other DMD cell-based therapies, MSCs also possess distinct anti-inflammatory activities and represent an ethical alternative to ES cells.…”