Chimeraplasty Diagram

If single-gene disorders are akin to minor misspellings in the human genome, then it stands to reason that the biological equivalent of a word processor's search-and-replace function could correct them. Some researchers are hoping that chimeraplasty can be that tool. The biological software--a chimeric oligonucleotide constructed from both DNA and RNA--was invented and first tested in vitro in 1994. But before it can be "shipped" to the clinic, its developers and others must optimize it and debug it.

Gene therapy researchers, in particular, are enthusiastic about the tool, because correcting defects in existing genes rather than flooding a patient's body with duplicate, correct copies seems safer. The promise--and the elegance--of the chimeraplasty approach lured R. Michael Blaese away from his National Institutes of Health post and into a position with Kimeragen Inc., a Newtown, Pa., firm that plans to develop and market the technology invented and patented by...

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