Scientists create targeted gene delivery for the brain

nih.gov

Scientists have developed new gene delivery systems for the brain and spinal cord, potentially enabling targeted therapies for neurological disorders. This breakthrough, funded by the NIH, could revolutionize treatment approaches. The new systems use adeno-associated viruses to deliver genetic material to specific cell types with high accuracy. This allows researchers to study neural circuits and potentially treat diseases like ALS and Alzheimer's more precisely than current methods. The research provides a toolkit of delivery systems and AI-powered programs, accelerating brain research. The tools are available for broad use, building on existing gene therapy successes and paving the way for more targeted treatments.


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