Year: 2024
Type: article
Abstract: The Duchenne Muscular Dystrophy (DMD) is a widespread genetic disorder caused by a mutation in the dystrophin gene. Current treatments, such as corticosteroids and genetic therapies like exon skipping, offer some symptom management but fall short of providing a cure. The CRISPR/Cas9 technology, which is related with Clustered Regularly Interspaced Short Palindromic Repeats-associated protein 9, could edit the genome with great precision. This technique holds potential in tackling the underlying issue of DMD by rectifying mutations in the dystrophin gene. Various strategies, including exon skipping, frame remodeling, and expression regulation, have demonstrated the potential to resumption dystrophin expression and improve muscle fitness in preclinical models. Even with these developments, there are still a number of important obstacles to overcome, such as enhancing the effectiveness of delivery, reducing off-target effects, and managing immune reactions. Future research will focus on refining these techniques and ensuring their safe and effective translation to clinical therapies, potentially revolutionizing the treatment of DMD and other genetic disorders.
Source: Theoretical and Natural Science
Authors: Jingxin Sun, Jiaxi Li
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: hybrid
OpenAlex ID: W4404509364