Key result
Gene therapy offers a potential cure for genetic muscle diseases, with ongoing clinical trials currently assessing its safety and efficacy in human patients.
Why the study?
Current treatments for genetic muscle diseases manage symptoms rather than the root cause, but advancements in gene therapy offer a potential cure.
Gene therapy represents a promising potential cure for genetic muscle diseases, transitioning from preclinical success to ongoing clinical trials.
Supports advancing gene therapy for genetic muscle diseases; leaves open confirmation of clinical efficacy and safety.
Genetic muscle diseases encompass a group of conditions where genetic alterations affect skeletal muscles, leading to muscle weakness and hypotonia. Presently, the focus of treatment is on managing symptoms rather than addressing the root cause of the disease. However, recent advancements in gene therapy offer potential cure for these diseases, with over 180 genes identified as contributing factors. Encouraging results have emerged from preclinical trials conducted using animal models, prompting the initiation of several clinical trials aimed at assessing the safety and efficacy of gene therapy in human patients. In this review, we aim to provide an overview of the fundamental concept of gene therapy and discuss ongoing clinical trials that hold promise for curing genetic muscle diseases.
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J. W. Lee (2023) conducted a review in Genetic muscle diseases. Gene therapy was evaluated. Gene therapy offers a potential cure for genetic muscle diseases, with ongoing clinical trials currently assessing its safety and efficacy in human patients.
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