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NK1R-expressing allogeneic bone marrow-derived mesenchymal stem cells
- Development stage
- Unknown
- Lead developer
- University of Miami
- Modality
- Gene Therapies, Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
NK1R-expressing allogeneic bone marrow-derived mesenchymal stem cells is an engineered cell therapy currently under clinical investigation for the treatment of chronic pruritus, particularly in patients with epidermolysis bullosa. This therapy utilizes allogeneic mesenchymal stem cells (MSCs) derived from bone marrow that have been genetically modified to overexpress the neurokinin-1 receptor (NK1R). The primary mechanism of action involves the modified cells acting as a biological sink for Substance P, a neuropeptide that is a key mediator of itch and inflammation. By overexpressing NK1R, the MSCs sequester circulating or local Substance P, preventing it from binding to endogenous receptors on peripheral nerves and immune cells. This targeted neuropeptide neutralization is combined with the inherent immunomodulatory and anti-inflammatory properties of MSCs to address refractory itch and associated skin inflammation.
- Other names
- neurokinin-1 receptor + allogeneic bone marrow-derived mesenchymal stem cell therapyNK1R-overexpressing mesenchymal stem cellsNK-1R-overexpressing mesenchymal stem cellsNK 1R-overexpressing mesenchymal stem cellsNeurokinin-1 receptor-expressing allogeneic BM-MSCsNeurokinin1 receptor-expressing allogeneic BM-MSCsNeurokinin 1 receptor-expressing allogeneic BM-MSCs
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