세미나/행사

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세미나 담당교수 : 2023-2학기 김진홍 (금요세미나, 콜로퀴움, jinhkim@snu.ac.kr), 강찬희 (신진과학자세미나, chanhee.kang@snu.ac.kr), 윤태영 (10-10 project, tyyoon@snu.ac.kr)
조 교 : 장사라 (02-880-4431, jsarah@snu.ac.kr)
호암교수회관 : 5572, 교수회관: 5241, 두레미담: 9358, 라쿠치나: 1631.

[초청강연] Antisense oligonucleotide therapy for rare genetic diseases

2021-12-13l 조회수 1287

일시: 2021-12-17 11:00 ~ 13:00
발표자: Jinkuk Kim (KAIST Graduate School of Medical Science and Engineering)
담당교수: 생명과학부
장소: https://snu-ac-kr.zoom.us/j/89587444260
Genome sequencing has been revolutionizing the diagnosis of genetic conditions, but many of these
conditions lack specific treatments. We recently demonstrated how molecular diagnosis of a child
with an ultra-rare, fatal neurodegenerative condition can lead to rational design, testing, and
manufacture of milasen, a splice-modulating antisense oligonucleotide (ASO) drug, tailored to the
patient - all within one year. An n-of-1 trial of milasen showed no serious adverse events and
treatment was associated with seizure improvement. As the study offered a proof-of-concept for
rapid development of treatments for neurological disorders in a timely fashion, we are further
expanding and refining this framework to swiftly develop treatments for various other neurological
conditions, such as frontotemporal dementia and Parkinson’s disease.