Systematic empirical evaluation of individual base editing targets: validating therapeutic targets in USH2A and comparison of methods
Yuki Tachida, Kannan Vrindavan Manian, Rossano M. Butcher, Jonathan Ma Levy, Nachiket D. Pendse, Erin Hennessey, David R. Liu, Eric A. Pierce, Qin Liu, Jason Comander
The paper evaluates base editing efficiency for USH2A mutations using in vitro screening and a mouse model, showing high correction rates and comparing methods.
bioRxiv · Oct 2, 2024 · PreprintGenetics