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발표분야
의료용 고분자 부문위원회
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포스터발표
제목
Efficient Genome Editing via Lipid Nanoparticle Delivery of mRNA-Encoded Adenine Base Editors
발표자

황유나 (서울대학교)

연구책임자

이혁진 (서울대학교)

공동저자
황유나 (서울대학교), 이예지 (서울대학교), 구다영 (서울대학교), 이혁진 (서울대학교)

초록

내용
CRISPR-based adenine base editors (ABEs) enable precise A•T-to-G•C conversion without double-strand breaks but are constrained by the packaging limits of adeno-associated viruses (AAVs), often requiring split systems with reduced efficiency. Here, we demonstrate lipid nanoparticles (LNPs) as a non-viral platform for delivering full-length ABE mRNA. LNP-delivered full-length ABE mRNA outperformed split formats in human DMD iPSCs and enabled efficient in vivo editing in a DMD-associated teratoma model. UTR optimization further enhanced ABE8e expression, achieving over 90% correction of a pathogenic splice-site mutation in spinal muscular atrophy models. In addition, subretinal delivery of UTR-optimized full-length ABE mRNA/sgRNA LNPs enabled efficient editing of the Vegfa exon 1 splice site in the mouse retina. These results establish LNP-mediated delivery of full-length ABE mRNA as a robust strategy to overcome viral packaging constraints and enable efficient genome editing.
발표코드
2PS-066
발표일정
2026-04-09  17:00 - 18:30