Gene therapy offers a promising approach to cancer treatment, but current strategies often lack specificity, leading to off-target effects. This study focuses on developing marker-dependent antisense DNA agents targeting housekeeping genes selectively based on the ERBB2 oncomarker. Using a structured design approach, we optimized the classical Antisense Releasing Cassette (ARC) to the advanced 3T-ARC, culminating in a highly selective final construct targeting DYNC mRNA. The optimized design achieved 73% target mRNA cleavage in ERBB2-positive cells, demonstrating enhanced selectivity and therapeutic potential for ASO-based cancer therapy.

Тафран Л. (науч. рук. Эльдиб А.А.) Antisense-based marker-dependent agents for targeting housekeeping genes // Сборник тезисов докладов конгресса молодых ученых. Электронное издание. – СПб: Университет ИТМО, [2025]. URL: https://kmu.itmo.ru/digests/article/15330