Date
Thursday, July 30, 2026
Time
12:20 PM - 12:35 PM (MDT)
Name
Comparison of Clinical-Stage ASOs for the Treatment of Angelman Syndrome in a Novel Humanized Ube3a-ATS Mouse Model
Description
Lucas James Submitted:
Disrupting UBE3A-ATS to unsilence patUBE3A is a major treatment strategy for AS, but rodent–primate sequence divergence limits therapeutic development. Here, we generated a humanized Ube3a-ATS mouse model and demonstrate its utility by testing therapeutics in development.
Location Name
Colorado B
Speakers
Moderator(s)
Becky Burdine, Dylan Ritter
Session Type
Abstract