“More than a half-dozen biotechnology companies have been launched to develop and commercialize ribozyme-based diagnostics and therapeutics. The pioneer was Ribozyme Pharmaceuticals, Inc. (RPI), of Boulder, Colorado. Founded in 1992 by current HHMI President Thomas R. Cech, the company was organized to pursue the discovery from Cech’s laboratory that some types of RNA, called ribozymes, can cleave other RNA molecules. RPI is developing therapeutic ribozymes to target the messenger RNA (mRNA) of proteins implicated in specific diseases. Like all proteins, disease-related proteins are encoded by genes, whose instructions are carried to the ribosome by mRNA. By synthesizing ribozymes that bind and cleave these mRNAs, RPI scientists are trying to prevent undesirable proteins from being produced.
Illustration: J.W. Stewart
source: the HHMI Bulletin,
June 2002, pages 14-19.
©2002 Howard Hughes Medical Institute