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Gene Therapy Marches Forward

Illustration: Erica P. Johnson After years of methodically lumbering along with antisense and gene knockout technologies, gene therapy has been given fresh legs. Techniques such as RNA interference (RNAi)--small nuclear RNAs to mask aberrant splice sites--and transposon technologies that extend the lives of transgenes are offering more applications than previously thought possible. A trio of recent papers highlights these approaches to gene therapy. RNAi is being used to boost gene therapy ef

Hal Cohen
Illustration: Erica P. Johnson

After years of methodically lumbering along with antisense and gene knockout technologies, gene therapy has been given fresh legs. Techniques such as RNA interference (RNAi)--small nuclear RNAs to mask aberrant splice sites--and transposon technologies that extend the lives of transgenes are offering more applications than previously thought possible. A trio of recent papers highlights these approaches to gene therapy.

RNAi is being used to boost gene therapy efforts in treating Huntington disease (HD). The challenge of fighting autosomal dominant diseases such as HD lies in decreasing aberrant gene expression. Citing Thomas Tuschl's seminal work on RNAi as their inspiration,1 researchers at the University of Iowa, Iowa City, silenced gene expression in a group of dominantly inherited neruodegenerative diseases using RNAi in the animal tissue of a cell model. These diseases, known as polyglutamine expansion diseases,2 cause HD.3

"Each siRNA [small interfering RNA]...

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