Scientists have developed the first genetically altered monkey model that replicates some symptoms observed in patients with Huntington's disease, according to a new study funded by the National Institutes of Health. Researchers are now able to better understand this complex, devastating and incurable genetic disorder affecting the brain. This advance, reported in the May 18 advance of online publication edition of Nature, could lead to major breakthroughs in the effort to develop new treatments for a range of neurological diseases.
Huntingtons is an inherited disease caused by a defective gene that triggers certain nerve cells in the brain to die. Symptoms may include uncontrolled movements, mood swings, cognitive decline, balance problems, and eventually losing the ability to walk, talk or swallow. It affects five to 10 people in every 100,000. There is no known treatment to halt progression of the disease, only medications to relieve symptoms. Death typically occurs 15 to 20 years after onset.
This study marks the first time that researchers have developed a rhesus macaque model of a specific human disease using transgenic technologies, a marked improvement over mouse models. Transgenic animals are created using a recombinant DNA method to modify a genome.
This research allows scientists to advance beyond mouse models which do not replicate all of the changes in the brain and behavior that humans with Huntingtons disease experience, said John D. Harding, Ph.D., director of primate resources at the NIHs National Center for Research Resources (NCRR), which funded the study. Primate models better mirror human diseases and are a critical link between research with small laboratory animals and studies involving humans.
To unravel the genetic components of this disease, NIH-supported researchers Anthony W.S. Chan, D.V.M., Ph.D.; Xiao-Jiang Li, M.D., Ph.D.; and Shi-Hua Li, M.D., Ph.D., collaborated with their colleagues at
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| Contact: Joyce McDonald info@ncrr.nih.gov 301-435-0888 NIH/National Center for Research Resources Source:Eurekalert |