Journal
FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY
Volume 10, Issue -, Pages -Publisher
FRONTIERS MEDIA SA
DOI: 10.3389/fbioe.2022.833833
Keywords
Duchenne muscular dystrophy; DMD; gene therapy; CRISPR/Cas9; exon skipping
Funding
- Universiti Kebangsaan Malaysia [GUP-2019-030]
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Duchenne muscular dystrophy (DMD) is a severe form of muscle dystrophy that currently lacks effective treatment options. Gene therapy has been proposed as a potential solution, but it also faces limitations and challenges.
As one of the most severe forms of muscle dystrophy, Duchenne muscular dystrophy (DMD) results in progressive muscle wasting, ultimately resulting in premature death due to cardiomyopathy. In the many years of research, the solution to DMD remains palliative. Although numerous studies including clinical trials have provided promising results, approved drugs, even, the therapeutic window is still minimal with many shortcomings to be addressed. Logically, to combat DMD that arose from a single genetic mutation with gene therapy made sense. However, gene-based strategies as a treatment option are no stranger to drawbacks and limitations such as the size of the dystrophin gene and possibilities of vectors to elicit immune responses. In this systematic review, we aim to provide a comprehensive compilation on gene-based therapeutic strategies and critically evaluate the approaches relative to its efficacy and feasibility while addressing their current limitations. With the keywords DMD AND Gene OR Genetic AND Therapy OR Treatment, we reviewed papers published in Science Direct, PubMed, and ProQuest over the past decade (2012-2021).
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