Exploring the alternatives

Researchers agree that recent findings about alternative splicing point toward the therapeutic applications that lie in its future. But the newest news about this ubiquitous mechanism for making many mRNAs from a single gene—more than 38,000, in one extreme Drosophila example—is that it appears to be crucial not just for generating lots of proteins, but probably for controlling gene expression as well.

"I believe it will be possible to generate therapeutic interventions based on alternate splicing," Donald Rio, of the University of California at Berkeley, told The Scientist. It is now understood that many disease-causing mutations ultimately result in altered RNA splicing. These mutations occur not only in well defined intron–exon junctions, but also in protein-coding exonic splicing control elements, said Rio, the keynote speaker at Splicing 2003, a meeting held Tuesday (September 23) in Rockville, Md.

Practical medical applications are perhaps 20 years away, according to Fyodor Kondrashov, who ...