Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed re...
Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo
About this item
Full title
Author / Creator
Ibraheim, Raed , Tai, Phillip W. L. , Mir, Aamir , Javeed, Nida , Wang, Jiaming , Rodríguez, Tomás C. , Namkung, Suk , Nelson, Samantha , Khokhar, Eraj Shafiq , Mintzer, Esther , Maitland, Stacy , Chen, Zexiang , Cao, Yueying , Tsagkaraki, Emmanouela , Wolfe, Scot A. , Wang, Dan , Pai, Athma A. , Xue, Wen , Gao, Guangping and Sontheimer, Erik J.
Publisher
London: Nature Publishing Group UK
Journal title
Language
English
Formats
Publication information
Publisher
London: Nature Publishing Group UK
Subjects
More information
Scope and Contents
Contents
Adeno-associated virus (AAV) vectors are important delivery platforms for therapeutic genome editing but are severely constrained by cargo limits. Simultaneous delivery of multiple vectors can limit dose and efficacy and increase safety risks. Here, we describe single-vector, ~4.8-kb AAV platforms that express Nme2Cas9 and either two sgRNAs for seg...
Alternative Titles
Full title
Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo
Authors, Artists and Contributors
Author / Creator
Tai, Phillip W. L.
Mir, Aamir
Javeed, Nida
Wang, Jiaming
Rodríguez, Tomás C.
Namkung, Suk
Nelson, Samantha
Khokhar, Eraj Shafiq
Mintzer, Esther
Maitland, Stacy
Chen, Zexiang
Cao, Yueying
Tsagkaraki, Emmanouela
Wolfe, Scot A.
Wang, Dan
Pai, Athma A.
Xue, Wen
Gao, Guangping
Sontheimer, Erik J.
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_81e517a4261d442a884395258d491bfe
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_81e517a4261d442a884395258d491bfe
Other Identifiers
ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/s41467-021-26518-y
