Establishing an experimental sgRNA expression screening assay for CRISPR activation in vitro
Elin Arvidsson, Diana Duarte Lobo, Ermelinda Sabarese, Fabio Duarte, Rui Jorge Nobre, Cecilia Lundberg, Luis Quintino · FENS Forum 2024
Neurodegenerative disorders (NDs) are characterized by a progressive loss of neurons leading to broad symptoms affecting cognition and motor function. Despite extensive research efforts, treatment options remain palliative, and due to increasing incidence of NDs worldwide, novel therapies are necessary. Gene therapy represents an attractive approach for tackling the complex pathology of NDs. CRISPR-mediated gene activation (CRISPRa) encompasses a growing field of biotechnological approaches with exciting implications for gene therapy through upregulation of genetic targets of interest. However
ePoster · Neuroscience