In Silico Design and Evaluation of CRISPR Cytosine Base Editing Strategy for Potential Correction of the ASPA Variant Associated with Canavan Disease
A candidate sgRNA with favourable predicted editing characteristics and genome-wide specificity for potential CBE-mediated correction of the ASPA c.691T>C (p.Tyr231His) variant is identified and a rational framework for prioritizing this guide for experimental evaluation is provided.
Aditi Verma, Elamathi Natarajan
· bioRxiv · 0 citations