Therapeutic strategies targeting muscle stem cells in satellite cell-opathies

Satellite cells, the resident muscle stem cells, are essential for skeletal muscle post-natal growth and regeneration. Dysfunction in these cells contributes to a group of muscle disorders known as satellite cell-opathies, which can be categorized into primary and secondary forms. Primary satellite cell-opathies stem from intrinsic defects within satellite cells, including genetic mutations that impair their survival, self-renewal, proliferation, or differentiation. Alternatively, secondary satellite cell-opathies result from pathological conditions affecting both the satellite cells and the muscle fibers. This review explores the pathophysiology of satellite cell-opathies, highlighting key molecular mechanisms underlying their dysfunction. Additionally, we discuss emerging therapeutic approaches, including gene therapy, pharmacological interventions, and cell-based therapies, which aim to restore satellite cell function and promote muscle regeneration. A deeper comprehension of these mechanisms and satellite cell–targeted strategies is essential to drive the development of innovative therapies for this emerging class of muscle disorders.