Affiliations: Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, USA
Note: [] Correspondence to: David E. Housman, Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, 500 Main Street, Room 76-553, Cambridge, MA 02139, USA. Tel.: +1 617 253 3013; E-mail: dhousman@mit.edu
Abstract: Rapidly identifying targets for Huntington's Disease (HD) therapeutics in relevant mouse models could hasten the development of patient interventions. We have recently described a method for rapidly and quantitatively measuring the progression of HD-like symptoms in mouse models. Because this method uses flow cytometry to measure GFP levels in affected neurons, it is amenable to pooled approaches. Here we describe a continuation of this work, using pools of shRNA-delivering AAV vectors and high throughput sequencing to determine which hairpins in a mixed population are most effective at preventing the transcriptional dysregulation phenotype of R6/2 mice.
