CORDIS Project
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This research aims to identify the genetic factors influencing the instability of the Huntington Disease-causing Huntingtin gene. By utilizing advanced genetic screening techniques, the project seeks to uncover the molecular mechanisms behind repeat expansion in neuronal cells.
This proposal 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 te…
UNIVERSITA DEGLI STUDI DI MILANO
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