Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing

Off-target effects and the feasibility for AAV-mediated delivery are the major barriers impeding the clinical in vivo application of base editors. Here, the authors report the small size AAV-deliverable Cas9-ABE variant that has improved on-target editing efficiency and reduced RNA-off target footpr...

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Autores principales:	Minh Thuan Nguyen Tran, Mohd Khairul Nizam Mohd Khalid, Qi Wang, Jacqueline K. R. Walker, Grace E. Lidgerwood, Kimberley L. Dilworth, Leszek Lisowski, Alice Pébay, Alex W. Hewitt
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2020
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/e11fbcac1a274b3aa056ddd2fd2f8176
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spelling	oai:doaj.org-article:e11fbcac1a274b3aa056ddd2fd2f81762021-12-02T15:14:48ZEngineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing10.1038/s41467-020-18715-y2041-1723https://doaj.org/article/e11fbcac1a274b3aa056ddd2fd2f81762020-09-01T00:00:00Zhttps://doi.org/10.1038/s41467-020-18715-yhttps://doaj.org/toc/2041-1723Off-target effects and the feasibility for AAV-mediated delivery are the major barriers impeding the clinical in vivo application of base editors. Here, the authors report the small size AAV-deliverable Cas9-ABE variant that has improved on-target editing efficiency and reduced RNA-off target footprint.Minh Thuan Nguyen TranMohd Khairul Nizam Mohd KhalidQi WangJacqueline K. R. WalkerGrace E. LidgerwoodKimberley L. DilworthLeszek LisowskiAlice PébayAlex W. HewittNature PortfolioarticleScienceQENNature Communications, Vol 11, Iss 1, Pp 1-10 (2020)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Minh Thuan Nguyen Tran Mohd Khairul Nizam Mohd Khalid Qi Wang Jacqueline K. R. Walker Grace E. Lidgerwood Kimberley L. Dilworth Leszek Lisowski Alice Pébay Alex W. Hewitt Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing
description	Off-target effects and the feasibility for AAV-mediated delivery are the major barriers impeding the clinical in vivo application of base editors. Here, the authors report the small size AAV-deliverable Cas9-ABE variant that has improved on-target editing efficiency and reduced RNA-off target footprint.
format	article
author	Minh Thuan Nguyen Tran Mohd Khairul Nizam Mohd Khalid Qi Wang Jacqueline K. R. Walker Grace E. Lidgerwood Kimberley L. Dilworth Leszek Lisowski Alice Pébay Alex W. Hewitt
author_facet	Minh Thuan Nguyen Tran Mohd Khairul Nizam Mohd Khalid Qi Wang Jacqueline K. R. Walker Grace E. Lidgerwood Kimberley L. Dilworth Leszek Lisowski Alice Pébay Alex W. Hewitt
author_sort	Minh Thuan Nguyen Tran
title	Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing
title_short	Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing
title_full	Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing
title_fullStr	Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing
title_full_unstemmed	Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing
title_sort	engineering domain-inlaid sacas9 adenine base editors with reduced rna off-targets and increased on-target dna editing
publisher	Nature Portfolio
publishDate	2020
url	https://doaj.org/article/e11fbcac1a274b3aa056ddd2fd2f8176
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Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing

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