shRNA NKCC1

shRNA NKCC1 is a gene-silencing therapeutic approach that utilizes short hairpin RNA (shRNA) to downregulate the expression of the sodium-potassium-chloride co-transporter 1 (NKCC1). In the context of glioblastoma, NKCC1 expression is elevated in glioma-intrinsic neurons, leading to increased intracellular chloride levels. This shift causes GABA-A receptor-mediated currents to become depolarizing and excitatory, driving neuronal hyperexcitability and tumor-associated epilepsy. By silencing NKCC1, this approach aims to restore inhibitory GABAergic tone and reduce neuronal synchrony. Preclinical studies conducted at the University of California, San Francisco, have demonstrated that NKCC1 knockdown reduces tumor burden and network burst synchrony in patient-derived xenograft models and organoid co-cultures.

Other names

NKCC1 shRNANKCC-1 shRNANKCC 1 shRNAshRNA-mediated NKCC1 knockdown