First, the human tropomyosin 3 gene was used to isolate a full-length alphaTmslow cDNA sequence. Next, the sequence was modified by site-directed mutagenesis to have the ATG-to-AGG base pair substitution encoding a methionine-to-arginine substitution at codon 9 (M9R); resulting in the dominant-negative alphaTmslow(Met9Arg) mutation associated with human nemaline myopathy. The mutant cDNA sequence was placed downstream of a 2.2 kbp human alpha-skeletal actin (ACTA1) promoter sequence, and upstream of a 1022 bp cassette containing the SV40 small t antigen intron and 3' untranslated region. Lines 4, 9 and 14 were generated with high, low and moderate expression levels, respectively. (J:67596)
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cx: complex: > 1 genome feature ot: other: hemizygous, indeterminate,... (F): Female
(M): Male
N: normal phenotype
(#): related diseases count