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Advances in AAV technology for delivering genetically encoded cargo to the nonhuman primate nervous system

Lillian Campos, Cynthia M. Arokiaraj, Miguel R. Chuapoco, Xinhong Chen, Nick Goeden, Viviana Gradinaru, Andrew S. Fox

2023Current Research in Neurobiology46 citationsDOIOpen Access PDF

Abstract

Modern neuroscience approaches including optogenetics, calcium imaging, and other genetic manipulations have facilitated our ability to dissect specific circuits in rodent models to study their role in neurological disease. These approaches regularly use viral vectors to deliver genetic cargo (e.g., opsins) to specific tissues and genetically-engineered rodents to achieve cell-type specificity. However, the translatability of these rodent models, cross-species validation of identified targets, and translational efficacy of potential therapeutics in larger animal models like nonhuman primates remains difficult due to the lack of efficient primate viral vectors. A refined understanding of the nonhuman primate nervous system promises to deliver insights that can guide the development of treatments for neurological and neurodegenerative diseases. Here, we outline recent advances in the development of adeno-associated viral vectors for optimized use in nonhuman primates. These tools promise to help open new avenues for study in translational neuroscience and further our understanding of the primate brain.

Topics & Concepts

Nonhuman primateNeuroscienceBiologyPrimateOptogeneticsTranslational researchGenetically modified organismEvolutionary biologyGeneticsBiotechnologyGeneVirus-based gene therapy researchCRISPR and Genetic EngineeringRNA Interference and Gene Delivery
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