Anna Victor

Gene editing in Neurodevelopmental Disorders: Applications & Ethics

Started Mar. 14, 2024

Abstract or project description

Genetic disorders, specifically genetic neurodegenerative disorders, affect the quality of life for many individuals. One example of such diseases is Rett Syndrome, a rare neurodegenerative disorder primarily affecting females and caused by a mutation in the MECP2 gene, which is essential for proper neuronal function. Affected individuals appear normal at birth but soon after start to regress rapidly. Previously, studies have attempted to treat this disease with drugs to subside symptoms, but there is no actual cure. Recent advancements in gene editing technology show great promise for the future of Rett Syndrome and other genetic diseases. This paper will review the potential of new gene editing technologies in the context of Rett Syndrome, discussing the scientific progress made in research and the ethics of its use in humans for disease treatment.