CRISPR-targeting introduced at the beginning of exon 2 a strong polyadenylation signal from beta-globin and two MAZ sites to promote efficient cleavage and transcriptional termination. Western blot analysis confirmed the absence of p and m isoforms. (J:266332)
Legend:
cx: complex: > 1 genome feature ot: other: hemizygous, indeterminate,... (F): Female
(M): Male
N: normal phenotype
(#): related diseases count