Exploring the Possibilities of Gene Therapy for Marfan Syndrome by Analyzing Potential Interventions to Prevent Cardiac Manifestations
DOI:
https://doi.org/10.58445/rars.2099Keywords:
Genetics, Marfan Syndrome, Biology, Gene TherapiesAbstract
ABSTRACT
Marfan Syndrome is an autosomal dominant genetic condition resulting from a mutation in the FBN1 gene of Chromosome 15. Approximately 200,000 patients residing in the United States are affected by this condition, with about 50,000 new diagnoses each year [1]. Extreme cases of Marfan syndrome can lead to deadly outcomes from massive internal bleeding with predominant abnormal manifestations in the skeletal, cardiovascular, and ocular systems. Cardiac involvement is prominent and is the primary cause of sudden death. Aortic aneurysms can advance into aortic dissections or ruptures, which lead to complete tears of the aortic layers. As of now, there is no direct cure for Marfan syndrome but treatment typically involves symptom management, which ranges from medication to preventative surgery. Gene therapy targeting the FBN1 gene can develop new, promising treatment options. In this review, we aim to discuss the usage of gene therapy for the treatment of Marfan Syndrome.
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