Article
Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis.
Aging cell - 1 Aug 2003
Ding Hongliu, Schwarz Dianne S, Keene Alex, Affar El Bachir, Fenton Laura, Xia Xugang, Shi Yang, Zamore Phillip D, Xu Zuoshang
Abstract excerpt
RNA interference (RNAi) can achieve sequence-selective inactivation of gene expression in a wide variety of eukaryotes by introducing double-stranded RNA corresponding to the target gene. Here we explore the potential of RNAi as a therapy for amyotrophic lateral sclerosis (ALS) caused by mutations in the Cu, Zn superoxide dismutase (SOD1) gene. Although the mutant SOD1 is toxic, the wild-type SOD1 performs...
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