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[초청강연] Antisense oligonucleotide therapy for rare genetic diseases

2021-12-13l Hit 338

Date: 2021-12-17 11:00 ~ 13:00
Speaker: Jinkuk Kim (KAIST Graduate School of Medical Science and Engineering)
Professor: 생명과학부
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.