A guide RNA [5'- GCAGATCACGAGGGAAGAGG-3'] is selected to introduce a full-length luciferase gene, containing the R387X (c.A1159T) nonsense mutation and T381T silent mutation, into the Gt(ROSA)26Sor locus. The silent T381T mutation creates a new PAM site required for the binding of the base editor. (J:334636)
Legend:
cx: complex: > 1 genome feature ot: other: hemizygous, indeterminate,... (F): Female
(M): Male
N: normal phenotype
(#): related diseases count