Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update
| Autor: | Chengmei Sun, Zheng Zhang, Luoan Shen, Xin Xie |
|---|---|
| Rok vydání: | 2020 |
| Předmět: |
Duchenne muscular dystrophy
musculoskeletal diseases 0301 basic medicine congenital hereditary and neonatal diseases and abnormalities lcsh:QH426-470 Cell Transplantation dystrophin restoration Genetic enhancement Review Bioinformatics Pathogenesis 03 medical and health sciences 0302 clinical medicine Fibrosis Genetics medicine Animals Humans Muscular dystrophy Genetics (clinical) biology business.industry pathogenesis Genetic Therapy medicine.disease gene therapy Muscular Dystrophy Duchenne Transplantation lcsh:Genetics 030104 developmental biology biology.protein Stem cell Dystrophin business 030217 neurology & neurosurgery |
| Zdroj: | Genes Genes, Vol 11, Iss 837, p 837 (2020) |
| ISSN: | 2073-4425 |
| DOI: | 10.3390/genes11080837 |
| Popis: | Neuromuscular disorders encompass a heterogeneous group of conditions that impair the function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being the most common and most severe type of muscular dystrophy, Duchenne muscular dystrophy (DMD), is caused by mutations in the X-linked dystrophin gene. Loss of dystrophin protein leads to recurrent myofiber damage, chronic inflammation, progressive fibrosis, and dysfunction of muscle stem cells. Over the last few years, there has been considerable development of diagnosis and therapeutics for DMD, but current treatments do not cure the disease. Here, we review the current status of DMD pathogenesis and therapy, focusing on mutational spectrum, diagnosis tools, clinical trials, and therapeutic approaches including dystrophin restoration, gene therapy, and myogenic cell transplantation. Furthermore, we present the clinical potential of advanced strategies combining gene editing, cell-based therapy with tissue engineering for the treatment of muscular dystrophy. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|