Ap4m1^em1Lutzy

CRISPR/cas9 genome editing uses guide RNAs (CCCTCTGTGCAGTGGGACCA, GTGCAGTGGGACCAAGGCTC, and AGTCTCACCGGCCTGAGCCT) were selected to target exon 11. Donor DNAs were created encoding a Q306X mutation (CAA to TGA, glutamine to stop). The orthologous clinical mutation R306X is associated with hereditary spastic paraplegia. (J:94077)