Topic: CRISPRa

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

Integrins are biomechanical receptors that connect both physically and functionally the extracellular matrix (ECM) to the intracellular cytoskeleton, regulating synaptic connectivity and plasticity in response to extracellular cues. In particular, in the brain, β3 integrin interacts with the αV subunit to form the αVβ3 integrin heterodimer, which mediates adhesion strengthening of excitatory synapses in cortical pyramidal neurons.Alteration in integrin structure, expression or signalling is involved in the aetiology of various brain pathologies, including autism spectrum disorder (ASD) and epi