Novel Gene Therapies

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Executive Summary  The accumulation of the neurotoxin quinolinic acid has been linked to Parkinson’s disease, Alzheimer’s disease, ALS, Huntington’s disease, brain ischemia, CNS infections, autoimmune disorders, mood disorders, and schizophrenia. If the pathway which quinolinic acid is synthesized in, the kynurenine pathway, could be manipulated at the last step, an earlier product could be diverted to form the benign compound picolinic acid. MSU has developed a novel technology which targets specific cells in the brain to divert away from quinolinic acid to picolinic acid.   Description of Technology MSU researchers have developed a variety of gene therapy vectors to alleviate quinolinate toxicity. This novel technology utilizes adeno-associated viral vectors to overexpress an quinolinate-modulating enzyme in a cell-specific manner in various cell populations in the brains. This involves using novel viral vectors that are designed to divert the kynurenine pathway away from producing the neurotoxin quinolinic acid and instead producing a neuroprotective compound.  This approach thus provides a potential neurorestorative and/or neuroprotective environment.   Key Benefits Neuroprotectant for a plethora of neurological disorders Cell Specific Targeting: Unique library of viral vectors aims at precisely targeting specific cells Modulating a normal cellular pathway, thus, no ectopic genes will be expressed.   Applications Treatment of multiple neurodegenerative diseases Personalized medicine: Ability to treat patients based on their individual disease    Patent Status:   Under review