Research teams funded by the National Institutes of Health (NIH) have developed gene delivery systems that target neural cell types in the human brain and spinal cord with high precision. These systems represent a significant advancement towards precise gene therapy for brain disorders, moving beyond treatments that only address symptoms.
The newly created delivery systems introduce genetic material into specific neural cells. This approach could revolutionize how scientists study neural circuits, providing tools to illuminate brain cell structures and control behaviors and cognition without genetically modified animals.
John Ngai, Director of the NIH’s Brain Research Through Advancing Innovative Neurotechnologies Initiative (The BRAIN Initiative), explained, “Imagine this new platform as a delivery truck dropping off specialized genetic packages in specific cell neighborhoods in the brain and spinal cord.” He added that these systems enable access to specific cells previously unreachable at such a scale.
These tools use adeno-associated virus (AAV) to deliver DNA across various species and experimental models. They have been validated in living systems, marking an essential step toward widespread application. The toolkit includes resources for studying individual cells and pathways affected by neurological diseases like ALS, Parkinson's disease, Alzheimer's disease, Huntington's disease, seizure disorders, and neuropsychiatric conditions.
Some AAV-based treatments are already approved for conditions like spinal muscular atrophy. The new resources aim to refine treatment precision by targeting only affected cells in the nervous system.
Available through distribution centers such as Addgene, this toolkit offers researchers standardized procedures and guides. The work is part of a large-scale project supported by The BRAIN Initiative to design molecular tools beneficial to research laboratories worldwide. Experts from molecular biology, neuroscience, and artificial intelligence collaborated on this project.
Eight related papers were published on May 21 in journals including Neuron and Cell Reports Methods. The NIH BRAIN Initiative aims to advance neurotechnology development for better understanding of brain function in health and disease.
The NIH is the primary federal agency supporting medical research across 27 Institutes and Centers under the U.S. Department of Health and Human Services.
