Allele Specific Gene Silencing in Autosomal-Dominant Skeletal Myopathies
نویسندگان
چکیده
منابع مشابه
Allele-Specific Gene Silencing in Two Mouse Models of Autosomal Dominant Skeletal Myopathy
We explored the potential of mutant allele-specific gene silencing (ASGS) in providing therapeutic benefit in two established mouse models of the autosomal dominantly-inherited muscle disorders, Malignant Hyperthermia (MH) and Central Core Disease (CCD). Candidate ASGS siRNAs were designed and validated for efficacy and specificity on ryanodine receptor (RyR1) cDNA mini-constructs expressed in ...
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ژورنال
عنوان ژورنال: Biophysical Journal
سال: 2010
ISSN: 0006-3495
DOI: 10.1016/j.bpj.2009.12.3908