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AAV-hIR is an experimental gene therapy candidate designed to deliver the human insulin receptor (hIR) gene to metabolic tissues, primarily the liver. It utilizes an adeno-associated virus (AAV) vector, specifically the AAV8 serotype, to restore or increase the expression of insulin receptors. This approach is intended to overcome insulin resistance and improve glucose homeostasis in patients with Type 2 Diabetes Mellitus (T2DM). Preclinical studies in mouse models, including inducible IR-knockout and ob/ob mice, have demonstrated that a single dose of AAV-hIR can significantly improve diabetic phenotypes with a long-lasting profile and a low risk of hypoglycemia in healthy subjects.
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