Publication date: Feb 29, 2020
A research group led by Dr. Gong Chen, a former professor at Penn State University and now leading a brain repair center at Jinan University in China, has developed a novel gene therapy to regenerate functional new neurons in mouse models of HD.
We are developing a series of NeuroD1-based gene therapies to reprogram brain internal glial cells directly into functional new neurons to treat a variety of brain disorders, including Huntington’s disease, Alzheimer’s disease, stroke, ALS, and many more,” said Dr. Chen.
“Because every single neuron in our brain is surrounded by supporting glial cells, such direct glia-to-neuron conversion technology offers great advantages over stem cell transplantation therapy in terms of high efficiency of neuroregeneration and no worries about immunorejection,” Dr. Chen added.
Dr. Chen is one of the early pioneers making use of internal brain glial cells to regenerate functional new neurons by overexpressing neural transcription factors in the mouse brain.
“In order to generate GABAergic neurons, we combined NeuroD1 together with another transcription factor Dlx2, which is known to generate GABAergic neurons during early brain development, and successfully converted striatal astrocytes into GABAergic neurons in HD mice,” said the first author of this article Dr. Zheng Wu.
In this HD mouse study, Dr. Chen and colleagues reported that 80% of the AAV-infected striatal astrocytes were directly converted into GABAergic neurons and the remaining astrocytes can proliferate to replenish themselves.