Overview

• The NIH-funded toolkit uses AI-identified DNA enhancers to deliver genes exclusively to targeted neural cell types with unprecedented precision.
• Dozens of adeno-associated virus (AAV) systems have been validated in multiple species, including human surgical tissue, ensuring broad applicability.
• The platform eliminates the need for transgenic animals, democratizing access to advanced genetic research tools for global laboratories.
• Freely available via Addgene, the toolkit includes standard operating procedures and user guides to accelerate neuroscience research.
• This milestone lays the foundation for next-generation therapies addressing root causes of neurological diseases like ALS and Parkinson’s.