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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

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The evolution of adeno-associated virus capsids for CNS gene therapy

Clinical Trial Designs For Advanced Therapies

Steven J Gray

Adeno-associated virus (AAV) is emerging as a dominant gene therapy delivery vehicle, and the broad tool kit of naturally-occurring AAV capsid variants has allowed tailoring of approaches for specific applications. For example, Glybera® (AAV1) is targeted to muscle, Luxterna™ (AAV2) is targeted to the retina, and Zolgensma® (AAV9) is targeted to the central nervous system (CNS). In the context of CNS gene therapy, the discovery of AAV9 was largely responsible for a shift from direct intraparenchymal brain injections to approaches that more globally target the brain, such as intravenous injection and/or injection into the cerebrospinal fluid (CSF). In fact, one could divide CNS gene therapy into ‘pre-AAV9’ and ‘post-AAV9’ eras, due to the dramatic leap that this vector technology enabled. One can envision a similar future leap coming, as lab-derived improvements to capsids are being made that could further increase the efficiency and specificity of CNS-directed gene therapy. Recent advancements in AAV vectors are discussed.

DOI: 10.18609/cgti.2019.141
Submitted for review: September 4, 2019
Citation: Cell & Gene Therapy Insights 2019; 5(11), 1361-1368.
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