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Research Investigator Profile
Michele Maxwell, PhD is examining the possibilities of using a cutting-edge technique called RNA interference as a way to understand ALS and mimic the effects of potential therapeutics. In ALS, there is a gene called SOD1 which, when mutated,causes inherited forms of the disease. RNA is the material that translates the gene and encodes SOD1 protein. By turning offor interfering with SOD1 RNA, scientists can prevent the production of defective proteins that lead to disease.
Dr.Maxwell’s goal is to use mouse models that have been bred to express mutant SOD1 and then to turn it off in adulthoodby activating RNAi. While her methods would not currently work in humans, these animals can be used to study whetherusing therapeutics that interfere with SOD1 RNA in adulthood would be an effective therapeutic approach. She will also beable to see, in real time, how shutting off SOD1 changes the biological functioning of the mouse’s brain, thereby providingimportant clues about how to change to course of the disease.
Evaluation of histone deacetylases as drug targets in Huntington's disease models. Study of HDACs in brain tissues from R6/2 and CAG140 knock-in HD mouse models and human patients and in a neuronal HD cell model.
Quinti L, Chopra V, Rotili D, Valente S, Amore A, Franci G, Meade S, Valenza M, Altucci L, Maxwell MM, Cattaneo E, Hersch S, Mai A, Kazantsev A.
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