Two pairs of sgRNAs were cleaved together to generate a large approximately 6300 bp deletion between the two target sites (up- and downstream of the Galns gene). GALNS enzyme activity is not detectable in tissues and blood of homozygotes. (J:342524)
Legend:
cx: complex: > 1 genome feature ot: other: hemizygous, indeterminate,... (F): Female
(M): Male
N: normal phenotype
(#): related diseases count