In Silico Design and Evaluation of CRISPR Cytosine Base Editing Strategy for Potential Correction of the ASPA Variant Associated with Canavan Disease
Aditi Verma, Elamanti Natrajan
The paper designs and computationally evaluates a CRISPR base editing strategy to correct the ASPA variant associated with Canavan disease, identifying a candidate sgRNA with favorable editing characteristics and genome-wide specificity.
bioRxiv · Oct 27, 2026 · PreprintBioinformatics