@inbook{c4762e4caaf84f25849dd77542968249,
title = "Delivering Base Editors In Vivo by Adeno-Associated Virus Vectors",
abstract = "CRISPR base editors are genome-modifying proteins capable of creating single-base substitutions in DNA but without the requirement for a DNA double-strand break. Given their ability to precisely edit DNA, they hold tremendous therapeutic potential. Here, we describe procedures for delivering base editors in vivo via adeno-associated virus (AAV) vectors, a promising engineered gene delivery vehicle capable of transducing a range of cell types and tissues. We provide step by step protocols for (i) designing and validating base editing systems, (ii) packaging base editors into recombinant AAV vector particles, (iii) delivering AAV to the central nervous system via intrathecal injection, and (iv) quantifying base editing frequencies by next-generation sequencing.",
keywords = "AAV, Base editing, CRISPR, Central nervous system, Genome editing, Intrathecal injections",
author = "Lim, {Colin K.W.} and Miskalis, {Angelo J.} and Pablo Perez-Pinera and Thomas Gaj",
note = "This work was supported by the National Institutes of Health (1U01NS122102-01A1, 1R01NS123556-01A1, and 5R01GM141296), the Muscular Dystrophy Association (MDA602798), and the Judith & Jean Pape Adams Foundation.",
year = "2023",
doi = "10.1007/978-1-0716-2879-9_11",
language = "English (US)",
series = "Methods in Molecular Biology",
publisher = "Humana Press Inc.",
pages = "135--158",
booktitle = "Methods in Molecular Biology",
}