CRISPRa-mediated activation of genes associated with inherited retinal dystrophies in acutely isolated human cells for diagnostic purposes
Valentin Johannes Weber, Alice Reschigna, Maximilian J. Gerhardt, Klara Sonnie Hinrichsmeyer, Dina Yehia Otify, Thomas Heigl, Frank Blaser, Isabelle Meneau, Martin Biel, Stylianos Michalakis, Elvir Bećirović
The paper uses CRISPRa to activate IRD-linked genes in PBMCs to detect transcripts for improved genetic diagnosis of inherited retinal dystrophies.
bioRxiv · Nov 29, 2024 · PreprintGenetics