CRISPR/cas9 genome editing uses two guide RNAs (uORF1 sgRNA: 5'- CCGGCGGGAATTTTTCAAAA-3' and uORF2 sgRNA: 5'- GCGTAGAGAAGGCGTCCTGA-3') and a single-stranded DNA repair template to insert the desired modification (ATG start codons of uORF1 and uORF2 were changed to ATA). (J:326689)
Legend:
cx: complex: > 1 genome feature ot: other: hemizygous, indeterminate,... (F): Female
(M): Male
N: normal phenotype
(#): related diseases count