Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing
Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing
About this item
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Author / Creator
Yin, Hao , Song, Chun-Qing , Suresh, Sneha , Wu, Qiongqiong , Walsh, Stephen , Rhym, Luke Hyunsik , Mintzer, Esther , Bolukbasi, Mehmet Fatih , Zhu, Lihua Julie , Kauffman, Kevin , Mou, Haiwei , Oberholzer, Alicia , Ding, Junmei , Kwan, Suet-Yan , Bogorad, Roman L , Zatsepin, Timofei , Koteliansky, Victor , Wolfe, Scot A , Xue, Wen , Langer, Robert and Anderson, Daniel G
Publisher
New York: Nature Publishing Group US
Journal title
Language
English
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Publication information
Publisher
New York: Nature Publishing Group US
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More information
Scope and Contents
Contents
Efficient Cas9 genome editing
in vivo
is achieved without viral vectors using chemically modified single-guide RNAs.
Efficient genome editing with Cas9–sgRNA
in vivo
has required the use of viral delivery systems, which have limitations for clinical applications. Translational efforts to develop other RNA therapeutics have shown that...
Alternative Titles
Full title
Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing
Authors, Artists and Contributors
Author / Creator
Song, Chun-Qing
Suresh, Sneha
Wu, Qiongqiong
Walsh, Stephen
Rhym, Luke Hyunsik
Mintzer, Esther
Bolukbasi, Mehmet Fatih
Zhu, Lihua Julie
Kauffman, Kevin
Mou, Haiwei
Oberholzer, Alicia
Ding, Junmei
Kwan, Suet-Yan
Bogorad, Roman L
Zatsepin, Timofei
Koteliansky, Victor
Wolfe, Scot A
Xue, Wen
Langer, Robert
Anderson, Daniel G
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_5901668
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_5901668
Other Identifiers
ISSN
1087-0156
E-ISSN
1546-1696
DOI
10.1038/nbt.4005