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Purification and Transplantation of Myogenic Progenitor Cell Derived Exosomes to Improve Cardiac Function in Duchenne Muscular Dystrophic Mice
Published on: April 10, 2019
Evolving Therapeutic Options for the Treatment of Duchenne Muscular Dystrophy
Eleonora S D'Ambrosio1, Jerry R Mendell2
1Center for Gene Therapy, Department of Pediatrics, Abigail Wexner Research Institute, Nationwide Children's Hospital, Ohio State University, Columbus, OH, 43205, USA. eleonora.d'ambrosio@nationwidechildrens.org.
Abstract:
Duchenne muscular dystrophy (DMD) is the most common childhood form of muscular dystrophy. It is caused by mutations in the DMD gene, leading to reduced or absent expression of the dystrophin protein. Clinically, this results in loss of ambulation, cardiomyopathy, respiratory failure, and eventually death. In the past decades, the use of corticosteroids has slowed down the disease progression. More recently, the development of genetically mediated therapies has emerged as the most promising treatment for DMD. These strategies include exon skipping with antisense oligonucleotides, gene replacement therapy with adeno-associated virus, and gene editing with CRISPR (clustered regularly interspaced short palindromic repeats) technology. In this review, we highlight the most up-to-date therapeutic progresses in the field, with emphasis on past and recent experiences, as well as the latest clinical results of DMD micro-dystrophin gene therapy. Additionally, we discuss the lessons learned along the way and the challenges encountered, all of which have helped advance the field, with the potential to finally alleviate such a devastating disease.
Insights
Duchenne muscular dystrophy (DMD) treatments are advancing with genetic therapies like exon skipping, gene replacement, and CRISPR gene editing. These innovative approaches offer new hope for managing this severe childhood muscle-wasting disease.
Area of Science:
- Neurology
- Genetics
- Biotechnology
Background:
- Duchenne muscular dystrophy (DMD) is a severe childhood genetic disorder caused by DMD gene mutations, leading to dystrophin deficiency.
- Clinical manifestations include progressive muscle degeneration, cardiomyopathy, respiratory failure, and premature death.
- Corticosteroids have historically managed symptoms, but novel genetic therapies represent a paradigm shift.
Approach:
- This review synthesizes recent advancements in genetic therapies for DMD.
- Key strategies discussed include antisense oligonucleotide-mediated exon skipping, adeno-associated virus (AAV)-based gene replacement therapy, and CRISPR gene editing.
- Emphasis is placed on micro-dystrophin gene therapy, including clinical trial outcomes.
Key Points:
- Genetic therapies offer targeted approaches to address the root cause of DMD.
- Exon skipping, gene replacement, and gene editing are rapidly evolving treatment modalities.
- Micro-dystrophin gene therapy shows promise in early clinical trials for restoring dystrophin function.
Conclusions:
- Recent therapeutic progress in DMD, particularly genetic strategies, holds significant potential for improving patient outcomes.
- Overcoming challenges and learning from past experiences are crucial for advancing DMD treatment.
- Innovative genetic therapies offer hope for alleviating the burden of this devastating neuromuscular disease.
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