Efficient gene therapy-based method for the delivery of therapeutics to primate cortex

Area of Science:

• Neuroscience
• Gene Therapy
• Primate Research

Background:

• Transducing the primate cortex with adeno-associated virus (AAV) vectors is difficult due to its large size.
• Developing effective gene delivery methods to the cortex is crucial for treating neurological disorders.

Purpose of the Study:

• To investigate a novel method for achieving widespread gene expression in the primate cortex using AAV vectors.
• To overcome the challenges associated with delivering gene therapy vectors to the large cortical area.

Main Methods:

• A single infusion of adeno-associated virus serotype 2 (AAV2) vector was administered into the thalamus of primates.
• Gene expression levels and distribution in the cortex were analyzed following the vector infusion.

Main Results:

• A single thalamic AAV2 vector infusion resulted in widespread transgene expression across the primate cortex.
• The expression was mediated by the transduction of numerous, widely dispersed thalamocortical projection neurons.

Conclusions:

• Targeting the thalamus with AAV2 vectors provides an effective strategy for widespread cortical gene transduction in primates.
• This approach holds significant potential for developing new treatments for genetic and neurodegenerative diseases affecting the brain.