by Scientists from Johns Hopkins All Children’s Hospital, in collaboration with an international research team, have made significant progress in understanding the causes of type 2 diabetes. Published in the journal Nature Communications, their findings present a potential strategy for developing new therapies that could restore dysfunctional pancreatic beta-cells or even prevent the development of type 2 diabetes altogether.
The study reveals that individuals with type 2 diabetes have deficient beta-cells, particularly in a protein called phosphatidylinositol transfer protein alpha (PITPNA). This protein plays a critical role in the formation of intracellular granules that contain insulin, enabling the processing and maturation of insulin cargo. By reintroducing PITPNA into the beta-cells of individuals with type 2 diabetes, the production of insulin granules is restored, addressing various deficiencies associated with beta-cell failure and type 2 diabetes.
Understanding how specific genes regulate the function of pancreatic beta-cells, particularly those involved in insulin granule production and maturation like PITPNA, is crucial for developing therapeutic options. The goal is to provide effective treatments for individuals living with type 2 diabetes.
Leading the study was Matthew Poy, Ph.D., an associate professor of Medicine and Biological Chemistry in the Johns Hopkins University School of Medicine and head of the Johns Hopkins All Children’s team within the Institute for Fundamental Biomedical Research. Poy emphasizes that further research is focused on determining whether PITPNA can enhance the functionality of stem-cell-derived pancreatic beta-cells.
Stem cell-based therapies are still in their early stages of clinical development, presenting vast untapped potential. Poy believes that increasing PITPNA levels in stem cell-derived beta-cells could improve their ability to produce and release mature insulin before transplantation in individuals with diabetes.
“Our dream is that increasing PITPNA could improve the efficacy and potency of beta-like stem cells,” says Poy. “This is where our research is heading, but we need to determine whether the capacity of these undifferentiated stem cells, which can be converted into various cell types, can be optimized to become healthy insulin-producing beta-cells. The ultimate goal is to find a cure for type 2 diabetes.”
The study highlights the importance of understanding the underlying causes of type 2 diabetes and exploring innovative treatment approaches. By restoring the function of beta-cells through the introduction of PITPNA, researchers hope to pave the way for more effective therapies and ultimately find a cure for this prevalent disease.
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