Split genes and muscular dystrophy.

Abstract

Recent developments in molecular biology have shown that some genes are split into segments, each coding for part of a protein. The intervening segments are transcribed but later excised, and the messenger RNA is spliced back together again before translation into protein begins. Abnormalities in the nontranslated segments would not be reflected in abnormal protein structure but, presumably, in the control of syntehsis of a particular protein. The muscular dystrophies may be an example of this class of disease. They are particularly amenable to investigation of this idea because muscle produces large amounts of relatively few proteins, facilitating isolation of the appropriate messenger RNA.