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Chunk #53 — Conclusion

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Targeting tau: Clinical trials and novel therapeutic approaches.
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In order to circumvent these challenges, companies are using gene therapy approaches by vectorizing antibodies to directly express them in the brain. Companies are developing novel AAV capsids that cross the blood-brain barrier after systemic administration, and engineering the viruses to improve transduction of cells in the CNS for enhanced cellular specificity. Preclinical studies will need to address the feasibility of this approach. These antibodies could possibly be actively transported out of the brain even if they are locally produced. Moreover, potential deleterious effects of expressing antibodies in cells that do not typically have this function will need to be addressed before being tested in the clinic.