Every other Tuesday, the Innovation Institute highlights a Pitt technology or a set of technologies from its portfolio of impactful innovations available for licensing or strategic partnerships. This week, we feature a technology that provides a novel approach to enhance gene therapy to treat diabetes.
SUMMARY:
Nuclear respiratory factor (NRF) -array promoter has been shown to drive transdifferentiation of alpha cells into beta cells. Production of beta cells could allow for treatment in type 1 diabetes. Additionally, in type 2 diabetes rejuvenation of beta cells could improve patient outcomes through conversion of alpha cells into insulin-producing and insulin secreting cells.
Stage of Development
Various AAVs have been synthesized with gene inserts containing shorter (4x) or longer (10x) NRF promoter, Pdx1 and MafA sequences, containing <5000 base pairs (bps).
Infection of human alpha cells with AAVs containing these specific gene inserts have been shown to lead to the secretion of insulin within 2 days, demonstrating the potential for double-stranded AAV vectors for use in gene therapy for diabetes.
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