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DLG4-1 is a preclinical therapeutic candidate developed by Grann, targeting the Disks large homolog 4 (DLG4) protein, also known as Postsynaptic Density Protein 95 (PSD-95). DLG4 is a critical scaffolding protein in the postsynaptic density of excitatory synapses, where it anchors ion channels and signaling proteins, playing a vital role in synaptic maturation and plasticity. Mutations in the DLG4 gene cause DLG4-related synaptopathy (SHINE syndrome), a condition characterized by intellectual disability, developmental delay, and epilepsy. DLG4-1 is designed to modulate DLG4 expression using Grann's RNA-targeted platform to address the underlying genetic cause of the disorder. The program is currently in the in vitro preclinical stage of development.
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