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Prenatal Interventions for the Treatment of Congenital Disorders

Book Contribution - Chapter

Congenital genetic defects are the leading cause of morbidity and mortality. With progress in diagnostic methods, many of these diseases can be diagnosed prenatally. Prenatal therapeutic interventions can help in improving pathological outcomes and therefore, they are evolving as central strategies to ameliorate congenital disorders. Small size, less developed biological barriers, accessible progenitor and stem cells, and immature immune response are inherent characteristics of the fetus. These characteristics make fetus an ideal candidate for early intervention and modern therapeutic strategies. Advancements in new diagnostic methodologies (e.g., next-generation sequencing), surgical modalities, and novel preclinical research (e.g., gene editing, cell therapy, and DNA barcoding) are paving way for treating congenital disorders even before pathological consequences start to set in. In this chapter, modern approaches and advances in prenatal pharmacotherapy, surgery, cell therapy, gene therapy, and gene editing are discussed.

Book: Prenatal Interventions for the Treatment of Congenital Disorders
Pages: 259–268
Number of pages: 10
Accessibility:Closed