Stem Cell Therapy for Hereditary Neuromuscular Diseases

Hereditary neuromuscular diseases encompass a diverse group of inherited disorders that affect motor neurons, peripheral nerves, skeletal muscles, or neuromuscular junctions. These diseases are significant contributors to mortality, disability, and mental health challenges worldwide, making them a major public health concern. An example is Duchenne Muscular Dystrophy (DMD), the most common form of muscular dystrophy, which is a severe X-linked condition characterized by mutations in the dystrophin gene. These mutations lead to the formation of defective dystrophin-related glycoprotein complexes (DGCs) in muscle stem cells and skeletal muscle. The absence of functional dystrophin results in improper muscle regeneration and increased susceptibility of myofibers to damage, contributing to the progressive nature of the disease.

Advances in genetic research since the identification of the DMD gene in 1986 have uncovered numerous disease-causing mutations, yet effective treatments remain limited. The development of novel therapies, particularly those involving stem cell-based regenerative medicine, is therefore critical.

This Research Topic aims to elucidate the latest advancements in therapeutic approaches for hereditary neuromuscular diseases, with a particular focus on muscle stem cell therapy and other innovative treatment methods applicable to diseases like DMD. We will explore the biological mechanisms underlying these conditions, the clinical applications of regenerative medicine, and the emerging prospects of cutting-edge therapies. Additionally, we will examine the complex multi-cellular interactions within muscle tissues using advanced techniques such as high-resolution imaging, systems biology, proteomics, and epigenetics.

Areas to be covered may include but are not limited to:

1. Examining the role and potential of muscle stem cells in regenerative medicine for hereditary neuromuscular diseases

2. Innovative genetic therapeutic approaches including exon skipping, gene therapy, and gene editing technologies.

3. Cellular and molecular mechanisms of muscle regeneration

4. Recent developments in the field of multi-cellular crosstalk between muscle stem cells and other cell types via high-resolution imaging techniques, system biology, and proteomic approaches

5. Epigenetic modifications in muscle disease and muscle stem cell therapy

We accept different article types including Mini-Reviews, Brief Research Reports, and Perspectives. A full list of accepted article types, including descriptions, can be found at this link.

Keywords: stem cells, neuromuscular diseases, hereditary diseases, muscle regeneration, stem cell therapy, duchenne muscular dystrophy

Important note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.