HD-DittoGraph: a digital human Embryonic Stem Cell platform for Huntington’s repeats
|Title||HD-DittoGraph: a digital human Embryonic Stem Cell platform for Huntington’s repeats|
|Sponsor||European Research Council - Advanced Grant (ERC-AdG)|
|Institution||University of Milan|
Associated cell lines
This project is aimed at identifying the molecular mechanisms that have brought the human Huntington Disease-causing Huntingtin (Htt) exon 1, with its pure and unstable CAG repeat, to be shaped the way it is today. Specifically, we intend to screen for genetic elements affecting Htt repeat length instability in dividing and postmitotic neuronal cells. The novelty of our approach relies on the construction of a human embryonic stem (hES) cell platform that couples highly efficient CRISPR/Cas9 technology with genome-wide screenings and third generation sequencing, to test the contribution of thousands of unequivocally barcoded cis and trans modifiers on Htt exon 1 repeats instability.