Figure 1.

CRISPR-Pass for Restoring Abbreviated Gene Expression

(A) Schematic of ABE-mediated CRISPR-pass. Targetable adenines are located in the coding or noncoding strand depending on the PAM’s orientation. All possible PTCs are shown in the upper boxes (coding strand targeting-TAA, TAG, TGA; noncoding strand targeting-TAA, TAG, TGA). The orange-colored shapes represent adenosine deaminase. (B) The percentages of different types of mutations causing pathological phenotypes in the ClinVar database. (C) The percentages of PTCs that are targetable by CRISPR-pass with various PAMs of variant ABEs and the recoverable rate of intact amino acids and bypassing alternative amino acids are depicted.