Stem Cells in Focus: The Role of Glia Cells in a Potential Treatment for Angelman Syndrome
Dr. Alvin Wang discusses the role of glial cells, specifically oligodendrocytes, in the potential treatment of Angelman Syndrome. Oligodendrocytes play a crucial role in insulating nerve fibers in the brain with myelin, which allows for efficient transmission of signals. However, individuals with Angelman Syndrome experience a delay in myelination, affecting learning and behavior. Dr. Wang’s research team is using induced pluripotent stem cells (iPSCs) to study oligodendrocytes and develop new drugs. They have discovered that the UBE3A gene, associated with Angelman Syndrome, is active in early oligodendrocyte development and helps these cells grow and mature properly. By screening different drugs, they have found some that restore normal growth to Angelman Syndrome cells and have shown promising results in mice. This research opens up new avenues for treating Angelman Syndrome by targeting glial dysfunctions alongside restoring neuron UBE3A expression.
In this talk, we will explore the role of glial cells, specifically oligodendrocytes, in the potential treatment of Angelman Syndrome. Dr. Alvin Wang, a neurologist turned neurobiologist from Brown University, will guide us through this fascinating topic. We will discuss the function of glial cells in the brain, the use of human-induced pluripotent stem cells (iPSCs) in studying the disease, and the research funded by FAST (Foundation for Angelman Syndrome Therapeutics).
Part 1: Glial Cells and Oligodendrocytes
Glial cells are not just passive supporters in the brain; they actively moderate the flow of signals, much like the components on a circuit board. Oligodendrocytes, a type of glial cell, play a crucial role in insulating and speeding up the transmission of signals in the brain. They wrap around nerve fibers, forming layers of myelin that protect and enhance the messages sent by our brains. Any disruption in this myelination process can affect learning and behavior, as seen in individuals with Angelman Syndrome.
Part 2: Human-Induced Pluripotent Stem Cells (iPSCs)
To study oligodendrocytes closely, researchers have turned to iPSCs, a game-changing tool that allows the creation of humanized models in the lab. iPSCs are derived from skin or blood samples and can be reprogrammed to become any type of cell, including oligodendrocytes. This method provides a unique opportunity to study the disease and test potential drugs. Dr. Wang’s lab has been using iPSCs to screen different drugs and has discovered promising candidates that can counteract the issues caused by Angelman Syndrome.
Part 3: Research and Potential Treatment
Dr. Wang’s research team, generously funded by FAST, is leveraging their expertise in stem cell technology to dissect the disease mechanism in oligodendrocytes. They have developed an efficient way to create human oligodendrocytes from iPSCs, allowing them to mimic the natural development process in a shorter timeframe. By studying the effects of Angelman Syndrome gene mutations on these cells, they have gained insights into the disease and discovered drugs that can restore normal growth to affected cells. These findings have shown significant improvements in mice with Angelman Syndrome and offer hope for the development of a treatment that could make a difference for individuals living with the condition.
Dr. Wang’s research highlights the importance of glial cells, particularly oligodendrocytes, in understanding and potentially treating Angelman Syndrome. The use of iPSCs has revolutionized the study of the disease, providing valuable insights and potential drug candidates. This research, made possible by the support of FAST and collaboration with other institutions, opens up new avenues for correcting glial dysfunctions and complementing existing efforts to restore neuron UBE3A expression. With continued research and development, there is hope for improved treatments for individuals with Angelman Syndrome.
- Title: Stem Cells in Focus: The Role of Glia Cells in a Potential Treatment for Angelman Syndrome
- Author(s): Yu-Wen Alvin Huang
- Author(s)’ affiliation: Brown University
- Publication date: 2023-11-12
- Collection: 2023 FAST Science Summit