GeneticsbioRxivPreprint
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…·Elvir Bećirović
The paper uses CRISPRa to activate IRD-linked genes in PBMCs to detect transcripts for improved genetic diagnosis of inherited retinal dystrophies.
DOI 10.1101/2024.11.29.625601