COMPOSITIONS AND METHODS FOR SELECTIVE GENE REGULATION
Application
US20260078404A1
Kind: A1
Mar 19, 2026
Inventors
Stephanie TAGLIATELA, Anne TANENHAUS, Kartik RAMAMOORTHI, Andrew YOUNG, David OBERKOFLER
Abstract
Provided herein are engineered transcription factors for selective upregulation of SCN1a and uses thereof for treating diseases and disorders, such as, Dravet syndrome. Also provided are microRNA binding sites and uses thereof for selective expression in parvalbumin neurons.
CPC Classifications
C12N 15/86
A61P 25/08
C07K 14/4705
C07K 14/705
C12N 15/1138
C12N 15/62
C07K 2319/71
C07K 2319/81
C12N 2320/32
C12N 2710/10343
C12N 2710/10344
C12N 2750/14143
C12N 2750/14152
C12N 2750/14171
C12N 2830/008
C12N 2840/007
Filing Date
2025-07-11
Application No.
19267193