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SUMMARY:基盤医学特論（分子遺伝学）
DTSTART;VALUE=DATE-TIME:20260924T080000Z
DTEND;VALUE=DATE-TIME:20260924T093000Z
UID:159024861777
DESCRIPTION:2026年度 基盤医学特論 開講通知Information on Speci
 alLecture Tokuron &amp\; Tokupro AY2026 Title：The promise of RNA (ASO) 
 therapy for neurodevelopmental disorders.From Angelman Syndrome to persona
 lized therapies.                             Teaching Staff：Ype Elgers
 ma\, PhD(Dept.of Clinical Genetics\, Erasmus University Medical Center\,Ro
 tterdam\, 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 Septemb
 er\,2026 17:00-18:30 (Lecture Room1\, 3th Floor of the Basic Medical Resea
 rch Building)使用言語：英語　English Abstract：Our research focu
 ses on understanding the molecular and cellularmechanisms underlying neuro
 developmental disorders\, and translating these findings intotargeted ther
 apies.We have studied a broad range of neurodevelopmental disorders\, incl
 udingAngelman Syndrome\, DUP15Q syndrome\, CAMK2 syndrome\, Tuberous Scler
 osis Complex\,mTORopathies\, and RASopathies.To gain deeper insight into p
 athophysiology ofneurodevelopmental disorders and to identify targeted tre
 atments\, we use mousemodels and induced pluripotent stem cells (iPSC)\, w
 ith a particular emphasis onidentifying robust disease-relevant phenotypes
  suitable for therapeutic testing. In recent years\, we haveparticularly f
 ocused 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 neu
 rodevelopmental disorders\, including Angelman syndrome and TuberousSclero
 sis Complex. In this talk I will discuss thelessons learned from ASO studi
 es in mouse models of Angelman syndrome. In particular\, I will also highl
 ight thedevelopment of ASO therapies for neurodevelopmental disorders caus
 ed 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 Molecula
 r Genetics (ext. 2447)※事前のお申し込みは不要です。  No R
 egistration 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. 
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