Delivering Base Editors In Vivo by Adeno-Associated Virus Vectors

Colin K.W. Lim, Angelo J. Miskalis, Pablo Perez-Pinera, Thomas Gaj

Research output: Chapter in Book/Report/Conference proceedingChapter


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.

Original languageEnglish (US)
Title of host publicationMethods in Molecular Biology
PublisherHumana Press Inc.
Number of pages24
StatePublished - 2023

Publication series

NameMethods in Molecular Biology
ISSN (Print)1064-3745
ISSN (Electronic)1940-6029


  • AAV
  • Base editing
  • Central nervous system
  • Genome editing
  • Intrathecal injections

ASJC Scopus subject areas

  • Genetics
  • Molecular Biology


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