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논문 기본 정보
- 자료유형
- 학술대회자료
- 저자정보
- 발행연도
- 2021.7
- 수록면
- 208 - 208 (1page)
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Genetic disorders with loss of function could overcome AAV-mediated gene therapy, but AAV has a limitation of capsid-mediated immunogenicity and the possibility of random integration. In contrast, genome editing offers the potential to get permanent treatment without repeated transduction. However, in vivo knock-in efficiency is extremely low; thus, a new innovative strategy to overcome this is needed. We tried to evaluate the knockin platform against human genetic disorder by inserting the protein-coding gene. In this experiment, hemophilia B was selected as a candidate genetic disorder, and the KI efficiency and therapeutic effect were verified by dual transduction of AAV-packed Campylobacter jejuni Cas9 and donor template. HR strategy did not develop any evidence of in vivo therapeutic KI, and it seemed to be reasoned by the non-dividing characteristics of ... 전체 초록 보기
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