Replacing the SpCas9 HNH domain by deaminases generates compact base editors with an alternative targeting scope

Base editors are RNA-guided deaminases that enable site-specific nucleotide transitions. The targeting scope of these Cas-deaminase fusion proteins critically depends on the availability of a protospacer adjacent motif (PAM) at the target locus and is limited to a window within the CRISPR-Cas R-loop...

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Vydané v:	Molecular therapy. Nucleic acids Ročník 26; s. 502 - 510
Hlavní autori:	Villiger, Lukas, Schmidheini, Lukas, Mathis, Nicolas, Rothgangl, Tanja, Marquart, Kim, Schwank, Gerald
Médium:	Journal Article
Jazyk:	English
Vydavateľské údaje:	Elsevier Inc 03.12.2021 American Society of Gene & Cell Therapy Elsevier
Predmet:	base editing CRISPR-Cas9 gene therapy genome editing HNHx-ABE Original genome editing gene therapy HNHx-ABE base editing CRISPR-Cas9
ISSN:	2162-2531, 2162-2531
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