Genetic Therapies for Neurodevelopmental Disorders

Most of the known gene variants that are associated with neurodevelopmental disorders consist of dominant “haploinsufficiencies”, in which expression of both the maternal and paternal copies of a gene is required for neurodevelopment. As a therapeutic strategy for neurodevelopmental disorders, the Sebat lab is developing Antisense Olignucleotide (ASO) therapies designed to increase the expression of genes by blocking negative regulators of mRNA or protein expression.