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학술발표회초록보기

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  • 05월 20일 18시 이후 : 초록수정 불가능, 일정확인 및 검색만 가능

제125회 대한화학회 학술발표회 및 총회 Therapeutic development using chemically modified asymmetric small interfering RNAs

등록일
2020년 2월 16일 20시 20분 06초
접수번호
1735
발표코드
LIFE1-1 이곳을 클릭하시면 발표코드에 대한 설명을 보실 수 있습니다.
발표시간
화 11시 : 00분
발표형식
심포지엄
발표분야
Life Chemistry - Nucleic Acid and Its Derivative Chemistry
저자 및
공동저자
Dong-ki Lee
Department of Chemistry, Sungkyunkwan University, Korea
Small interfering RNAs (siRNAs) are rapidly emerging as the third-generation therapeutic development platform with the capability to target virtually any disease-causing genes present in the human genome. We have developed asymmetric siRNA (asiRNA) structures, wherein the sense strands are shorter than conventional siRNAs and demonstrated that the asiRNAs show comparable target gene silencing activity with reduced non-specific effects. To make asiRNA-based therapeutics, we have introduced various chemical modifications in sugar and backbone structure of RNA, along with conjugation of delivery moieties such as lipids and N-acetylglactosamine (GalNAc) to generate cp-asiRNA and GalNAc-asiRNA, respectively. In this presentation, I will summarize our asiRNA platform for therapeutics development and the pre-clinical and clinical data based on this highly efficient and versatile drug development platform.

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