Oral Presentation International Congress on Neuronal Ceroid Lipofuscinoses 2025

Adenine Base Editors: A Novel Approach to Treat CLN2 Disease (127780)

Jill M Weimer 1 2 , Mitchell J Rechtzigel 1 , Peyton E Randolph 3 , Gregory Newby 3 , Clarissa D Booth 1 , Melissa A Pratt 1 , Hannah G Leppert 1 , Joelle T Anderson 1 , Kaylie J Timm 1 , David R Liu 3 4
  1. Sanford Health Research, Sioux Falls, South Dakota, United States
  2. Sanford School of Medicine at University of South Dakota, Vermillion, South Dakota, United States
  3. Broad Institure of MIT and Harvard, Cambridge, MA, United States
  4. Harvard Medical School, Boston, MA, United States
Publish consent withheld