A TRANSGENIC MONKEY: INHERITED NEURODEGENERATIVE DISEASES

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Nonhuman primates (NHPs; monkeys) have contributed significantly in many areas of human medicine research as they are invaluable models for studying human diseases. Recent advancements in monkey genomic information and transgenic technology have resulted in the creation of the first transgenic monkey model of Huntington's disease (HD). Transgenic HD monkeys develop cellular changes and neuropathologies similar to that of human patients, which are rarely observed in rodent models. Besides the neurotoxicity of mutant huntingtin (htt) in monkeys, HD monkeys also develop involuntary movement and difficulties in coordinating body movement similar to that of HD patients. We have successfully accomplished our goal of the original proposal in developing a transgenic HD monkey that expresses mutant htt and develops symptoms comparable to human patients.