基盤医学特論（分子遺伝学）

基盤医学特論（分子遺伝学）

2026年度 基盤医学特論 開講通知Information on SpecialLecture Tokuron & Tokupro AY2026 Title：The promise of RNA (ASO) therapy for neurodevelopmental disorders.From Angelman Syndrome to personalized therapies.   Teaching Staff：Ype Elgersma, PhD(Dept.of Clinical Genetics, Erasmus University Medical Center,Rotterdam, the Netherlands.)日時：2026年 9月24日（木）17：00～18：30 (第１講義室　基礎研究棟3階) Time and Date : 24th September,2026 17:00-18:30 (Lecture Room1, 3th Floor of the Basic Medical Research Building)使用言語：英語　English Abstract：Our research focuses on understanding the molecular and cellularmechanisms underlying neurodevelopmental disorders, and translating these findings intotargeted therapies.We have studied a broad range of neurodevelopmental disorders, includingAngelman Syndrome, DUP15Q syndrome, CAMK2 syndrome, Tuberous Sclerosis Complex,mTORopathies, and RASopathies.To gain deeper insight into pathophysiology ofneurodevelopmental disorders and to identify targeted treatments, we use mousemodels and induced pluripotent stem cells (iPSC), with a particular emphasis onidentifying robust disease-relevant phenotypes suitable for therapeutic testing. In recent years, we haveparticularly focused on developing RNA therapies the focus of the lab hasshifted towards developing RNA therapies using antisense oligonucleotides(ASOs). Studies in mouse models have demonstrated the therapeutic potential ofASOs for neurodevelopmental disorders, including Angelman syndrome and TuberousSclerosis Complex. In this talk I will discuss thelessons learned from ASO studies in mouse models of Angelman syndrome. In particular, I will also highlight thedevelopment of ASO therapies for neurodevelopmental disorders caused by ultra-rarevariants throughn-of-1 personalized treatment approaches. 関係講座:分子遺伝学部門等の連絡担当者: 分子遺伝学中沢由華（内線2447）Contact: Yuka Nakazawa,Department of Molecular Genetics (ext. 2447)※事前のお申し込みは不要です。  No Registration required.※講義開始後の30分までにご入室下さい。  Please take a seat before 17:30.※途中退室不可  Please stay until the end ofthe lecture.