The entire coding region of human DMD plus 190 bp of the 3' untranslated region was used as the transgene. The rat Acta1 promoter, exon 1, intron 1, and part of exon 2, was used to drive transgene expression in skeletal muscle. Expression of the transgene was confirmed by Western blot analysis of skeletal muscle tissue. (J:22142)
Legend:
cx: complex: > 1 genome feature ot: other: hemizygous, indeterminate,... (F): Female
(M): Male
N: normal phenotype
(#): related diseases count