Key Points
- This project is exploring a new ultrasound-based approach to improve the safety and effectiveness of stem cell therapies.
- Researchers introduced a novel genetic switch wherein a single, brief ultrasound treatment left the targeted gene permanently active.
Ultrasound Control of Gene Expression in Human iPSCs via Heat Shock Promoters
Stem cell therapy has the potential to transform treatment for neurodegenerative diseases, but key barriers still limit its use in the brain, including for Parkinson’s disease. To overcome these hurdles, the Focused Ultrasound Foundation is funding a project led by Yun Jing, PhD, and Xiaojun (Lance) Lian, PhD, at Penn State University that is exploring a new ultrasound-based approach to improve the safety and effectiveness of stem cell therapies.
In a new publication from this project, the team demonstrates how focused ultrasound can remotely “switch on” genes inside human induced pluripotent stem cells (or iPSCs) using the cells’ natural heat-response pathways. While previous studies combining focused ultrasound with heat shock promoters (HSPs) were aimed at short-lived, temporary gene activation, here the researchers introduced a clever genetic switch that turns this short pulse into permanent gene expression. They found that a single, brief ultrasound treatment left the gene permanently active.
This advance opens the possibility of guiding stem cells to develop into the right cell types at the right time, without drugs or invasive tools. Such precise control over iPSC differentiation could pave the way for new, safer therapies for Parkinson’s disease and other neurodegenerative disorders, and may help overcome long-standing challenges of cell survival and integration that have limited stem cell therapies to date.
“Having demonstrated this technology in vitro, we have now progressed to in vivo studies, conducting demonstrations of our focused ultrasound–based technology using a mouse model of Parkinson’s disease,” said Dr. Jing. “We hope to report on the results of this animal study soon.”
This study was funded by the National Science Foundation, National Institutes of Health, the Huck Innovative and Transformational Seed Fund, the Focused Ultrasound Foundation, and the Pennsylvania Department of Health Tobacco CURE Funds.
Dr. Jing participated in the Foundation’s November 2023 Cell and Gene Therapy Workshop.
“Prof. Jing is developing technology to tackle a critical challenge in cell therapies for neurodegenerative diseases: cell engraftment,” said Frédéric Padilla, PhD, the Foundation’s director of the Gene and Cell Therapy Program. “If successful, this approach could have wide-ranging applications. Neurodegenerative diseases are a priority area for our Gene and Cell Therapy Program at the Focused Ultrasound Foundation.”
See Biotechnology and Bioengineering (Open Source)
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