In Silico Design and Evaluation of CRISPR Cytosine Base Editing Strategy for Potential Correction of the ASPA Variant Associated with Canavan Disease
**Background** Canavan disease is a rare autosomal recessive neurodegenerative leukodystrophy caused by pathogenic variants in the ASPA gene, for which effective molecularly targeted therapies remain limited. The NM_000049.4:c.691T>C (p.Tyr231His) variant introduces a pathogenic C-G base pair that is potentially amenable to correction using cytosine base editing (CBE). This study aimed to design…
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