Gene Therapy for Congenital Disorders: A Critical Review of Preclinical and Clinical Studies
Year: 2025
Type: article
Abstract: 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.
Authors: Matos, Eliana X, Britton, Patricia, Chan, Alyssa, Ip, Gabriella, Koo, Eric, Liu, Alyssa, Luna, Leslie, Nguyen, Ivan, Nguyen, Sophia Nguyen, Sekhon, Ishaan, Sobhani, Sanoja, Zhu, Malika
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: green
OpenAlex ID: W7047558888
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