As a potential treatment for Parkinson’s disease, scientists at the University of North Carolina (UNC) at Chapel Hill have created smarter immune cells that produce and deliver a neuron-healing protein to the brain, while also teaching nerve cells to begin making the protein for themselves. Associate Professor Elena Batrakova, Ph.D., and her team at the UNC Eshelman School of Pharmacy’s Center for Nanotechnology in Drug Delivery genetically modified white blood cells called macrophages to produce glial cell–derived neurotrophic factor (GDNF), and deliver it to the brain. Glial cells provide support and protection for nerve cells throughout the brain and body, and GDNF can heal and stimulate the growth of damaged neurons. “Currently, there are no treatments that can halt or reverse the course of Parkinson’s disease. There are only therapies to address quality of life, such as dopamine replacement,” Dr. Batrakova said. “However, studies have shown that delivering neurotrophic factor to the brain not only promotes the survival of neurons, but also reverses the progression of Parkinson’s disease.” In addition to delivering GDNF, the engineered macrophages can “teach” neurons to make the protein for themselves by delivering both the tools and the instructions needed: DNA, messenger RNA, and transcription factor. Successfully delivering the treatment to the brain is the key to the success of GDNF therapy, Dr. Batrakova said. Using immune cells avoids the body’s natural defenses. The repurposed macrophages are also able to penetrate the blood-brain barrier, something most medicines cannot do. The reprogrammed cells travel to the brain and produce tiny vesicles called exosomes that contain GDNF. The cells release the exosomes, which are then able to deliver the GDNF proteins to neurons in the brain.
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