Juvenile Huntington's Disease Research

Talented researchers in the group are developing gene editing strategies using TALENs and CRISPR technologies. These are molecules that go into the neural progenitor cells and actually cut out the extra CAG repeats, reducing them to the normal range of 15, or modifying the mutant allele so that it cannot be expressed into problematic RNA and protein. Those cells should be "fixed" and go on to make neurons in the brain that are not affected by HD. This is what we need for children with the juvenile form of HD (JHD) - repaired neurons to be made for the rest of their- hopefully long- lives. This work specifically focuses on JHD but the exciting delivery platforms on which the JHD team is working could be applicable to adult-onset HD or any other monogenetic neural disorder.

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