@inbook{d07e1acc7cc949948b73a5d165b5ace3,
title = "Identification and Validation of CRISPR/Cas9 Off-Target Activity in Hematopoietic Stem and Progenitor Cells",
abstract = "Targeted genome editing in hematopoietic stem and progenitor cells (HSPCs) using CRISPR/Cas9 can potentially provide a permanent cure for hematologic diseases. However, the utility of CRISPR/Cas9 systems for therapeutic genome editing can be compromised by their off-target effects. In this chapter, we outline the procedures for CRISPR/Cas9 off-target identification and validation in HSPCs. This method is broadly applicable to diverse CRISPR/Cas9 systems and cell types. Using this protocol, researchers can perform computational prediction and experimental identification of potential off-target sites followed by off-target activity quantification by next-generation sequencing.",
keywords = "CRISPR/Cas9, Genome editing, GUIDE-seq, Hematopoietic stem and progenitor cells (HSPCs), Next-generation sequencing (NGS), Off-target",
author = "Park, \{So Hyun\} and Lee, \{Ciaran M.\} and Gang Bao",
note = "Publisher Copyright: {\textcopyright} 2022, Springer Science+Business Media, LLC, part of Springer Nature.",
year = "2022",
doi = "10.1007/978-1-0716-1979-7\_19",
language = "English",
series = "Methods in Molecular Biology",
publisher = "Humana Press Inc.",
pages = "281--306",
booktitle = "Methods in Molecular Biology",
}