Huntington’s Advance: Drug limits disease effects in laboratory mice
By Nathan Seppa
A compound that inhibits enzymes that act as stop signs for genes counteracts the movement disorders brought on by Huntington’s disease, a mouse study suggests.
In this hereditary disease, a genetic mutation results in oversized versions of the so-called huntingtin protein, portions of which stack up in the nuclei of brain cells. Patients develop impairments of thought, movement, and emotions for which there is no treatment or cure.
The mutant version of the huntingtin protein inhibits the action of indispensable enzymes, called acetyltransferases, in brain cells (SN: 4/28/01, p. 271: Huntington’s protein may be kidnapper). Responding to chemical signals, acetyltransferases normally work with other cellular chemicals to switch on genes as needed. Counterpart enzymes, called deacetyltransferases, reverse the process and shut off the genes once they’ve done their job.