Review written by Amy Ciceu (NEU, 2024)
Adeno-associated viruses (AAVs) are some of the most widely used recombinant viral technologies—those that combine different genes to produce unique viral vectors, or tools that convey genetic material into cells—in modern neuroscience. Because they are incapable of being replicated within cells, recombinant AAVs are commonly used in neuroscience research as a means of expressing genes in specific cells. By expressing genes that heighten or dampen the function of certain cells, researchers are able to identify the functions of particular neural circuits.1 This approach can provide scientists with insight into the mechanisms underlying neurobiological processes. To make such circuit-related discoveries, AAVs are typically injected into certain brain regions of animal subjects. After an incubation period, some AAVs encoding fluorescent tracers can induce cells to fluoresce under specialized microscopes, enabling scientists to visualize particular neural circuitry. Furthermore, AAVs have demonstrated clinical potential; for example, AAVs have been used to replenish certain proteins in the treatment of diseases like congenital blindness 2, 3 and spinal muscular atrophy.4, 5 AAV is also currently being investigated as a potential means of treating other brain disorders, including Parkinson’s disease, Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), and more.6
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