Gene Therapy for Congenital Disorders: A Critical Review of Preclinical and Clinical Studies

Congenital genetic disorders, otherwise known as birth defects, stem from a myriad of probable causes including, but not limited to, genetic mutations, environmental factors, and changes in epigenetics. Throughout this paper, we explore a multitude of studies surrounding congenital anomalies like heart defects, Phenylketonuria, Sickle Cell Disease, Beta-thalassemia, Multiple Sulfatase Deficiency, and several eye disorders. While preliminary results from laboratory and clinical studies demonstrate potential, it is important to consider challenges in safety, delivery, and cost-accessibility. Gene therapy offers hope for effective and lasting treatments for these lifelong-lasting conditions.