FRIDAY, July 28, 2006 (HealthDay News) -- An enzyme critical to the repair of damaged cells and maintenance of cellular energy may be a potential drug target for treatment of people with Huntington's disease, U.S. researchers say.
Researchers at MassGeneral Institute for Neurodegenerative Disease in Boston found that blocking the action of Poly (ADP-ribose) polymerase (PARP1), which is known to play an important role in cell repair and maintenance, may be an effective therapy in cases of Huntington's and other disorders characterized by low cellular energy levels.
"While PARP1 is essential for the repair of damaged DNA, we also know that, if over activated, it can cause cell death by excessive energy depletion," study leader Aleksey Kazantsev, director of the High Throughput Drug Screening Laboratory at MassGeneral, said in a prepared statement.
"It has recently been shown that neurons from patients with Huntington's appear to be energy deficient, so, we hypothesized that modest stresses that would be tolerated by healthy cells could send HD (Huntington's disease) cells below a viable energy threshold and that blocking PARP1 activation could be protective," Kazantsev said.
In laboratory research, the MassGeneral team found that an enzyme called K245-14 could block PARP1 activity and reduce energy depletion in cells with the HD genetic mutation.
The study was published in the August issue of the journal Chemistry & Biology.
More information
The group WeMove has more about Huntington's disease.
