Microglial Replacement and COURIER or SPIT for Neuronal Gene Editing

Michael Renteln

doi:10.20944/preprints202404.1620.v1

Preprint Concept Paper Version 1 Preserved in Portico This version is not peer-reviewed

Version 1 : Received: 24 April 2024 / Approved: 24 April 2024 / Online: 24 April 2024 (11:05:05 CEST)

Adeno-associated viral (AAV) vectors can be used for gene delivery. AAV.CAP-Mac was recently developed; it can cross the blood-brain barrier and transduce cells throughout the CNS. However, some brain regions were not transduced in adult rhesus monkeys. Additionally, AAV vectors can only package 5 kb of DNA. Also, AAV vectors may be genotoxic and cytotoxic, especially at high doses. Finally, AAV vectors are very expensive to produce at sufficiently high titers for treatment. Off-the-shelf cell-based delivery systems wherein the cells can infiltrate the target tissue and be hyper-motile would be ideal. Also, mRNA-based gene editing would be a transient treatment, and thus be much safer.

microglial replacement; adeno-associated viral (AAV) vectors; COURIER; SPIT; base editing; prime editing

Biology and Life Sciences, Biology and Biotechnology

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Microglial Replacement and COURIER or SPIT for Neuronal Gene Editing

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