A breakthrough in gene delivery systems has been made by researchers funded by the National Institutes of Health (NIH). The new technology can reach different neural cell types in the human brain and spinal cord with exceptional accuracy, paving the way for precise gene therapy to control errant brain activity.
The system uses a small, stripped-down adeno-associated virus (AAV) to deliver DNA to target cells. It has been tested in intact living systems and includes dozens of delivery systems that selectively target key brain cell types, such as excitatory neurons and inhibitory interneurons.
The toolkit also includes computer programs powered by artificial intelligence (AI) that can identify genetic “light switches” that turn genes on in specific brain cell types. This will significantly accelerate understanding of the human brain and enable access to specific brain cell types in the prefrontal cortex, an area critical for decision-making and uniquely human traits.
The new technology has the potential to transform how scientists study neural circuits and provide precise treatments that target only affected cells in the brain, spinal cord, or brain blood vessels. The toolkit is available at distribution centers, including Addgene, and offers researchers standard operating procedures and user guides for the tools.
This breakthrough was made possible by a large-scale project launched by the NIH’s Brain Research Through Advancing Innovative Neurotechnologies Initiative (BRAIN), which aims to develop precise and reproducible access to cells and circuits in experimental research models of the brain and spinal cord.
Source: https://www.nih.gov/news-events/news-releases/scientists-design-gene-delivery-systems-cells-brain-spinal-cord