Amyotrophic Lateral Sclerosis
RNA interference represses mutated SOD1 genes in mammalian cells
Published in Gene Therapy Weekly, March 11th, 2004
"In many of autosomal dominant diseases such as familial amyotrophic lateral sclerosis (ALS) with SOD1 mutation, a missense point mutation may induce the disease by its gain of adverse property. Reduction of such a mutant protein expression is expected to improve the disease phenotype. Duplex of 21-nt RNA, known as siRNA, has recently emerged as a powerful tool to silence gene, but the sequence specificity and efficacies have not been fully studied in comparison with ribozyme and DNA enzyme," scientists writing in the journal Biochemical and Biophysical Research Communications report.
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Source: Gene Therapy Weekly (2004-03-11)
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