Extension of the crRNA enhances Cpf1 gene editing in vitro and in vivo

Hyo Min Park, Hui Liu, Joann Wu, Anthony Chong, Vanessa Mackley, Christof Fellmann, Anirudh Rao, Fuguo Jiang, Hunghao Chu, Niren Murthy () and Kunwoo Lee ()
Additional contact information
Hyo Min Park: GenEdit Inc.
Hui Liu: GenEdit Inc.
Joann Wu: GenEdit Inc.
Anthony Chong: GenEdit Inc.
Vanessa Mackley: GenEdit Inc.
Christof Fellmann: University of California, Berkeley
Anirudh Rao: University of California, Berkeley
Fuguo Jiang: University of California, Berkeley
Hunghao Chu: GenEdit Inc.
Niren Murthy: University of California, Berkeley
Kunwoo Lee: GenEdit Inc.

Nature Communications, 2018, vol. 9, issue 1, 1-12

Abstract: Abstract Engineering of the Cpf1 crRNA has the potential to enhance its gene editing efficiency and non-viral delivery to cells. Here, we demonstrate that extending the length of its crRNA at the 5′ end can enhance the gene editing efficiency of Cpf1 both in cells and in vivo. Extending the 5′ end of the crRNA enhances the gene editing efficiency of the Cpf1 RNP to induce non-homologous end-joining and homology-directed repair using electroporation in cells. Additionally, chemical modifications on the extended 5′ end of the crRNA result in enhanced serum stability. Also, extending the 5′ end of the crRNA by 59 nucleotides increases the delivery efficiency of Cpf1 RNP in cells and in vivo cationic delivery vehicles including polymer nanoparticle. Thus, 5′ extension and chemical modification of the Cpf1 crRNA is an effective method for enhancing the gene editing efficiency of Cpf1 and its delivery in vivo.

Date: 2018
References: Add references at CitEc
Citations: View citations in EconPapers (1)

Downloads: (external link)
https://www.nature.com/articles/s41467-018-05641-3 Abstract (text/html)

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:nat:natcom:v:9:y:2018:i:1:d:10.1038_s41467-018-05641-3

Ordering information: This journal article can be ordered from
https://www.nature.com/ncomms/

DOI: 10.1038/s41467-018-05641-3

Access Statistics for this article

Nature Communications is currently edited by Nathalie Le Bot, Enda Bergin and Fiona Gillespie

More articles in Nature Communications from Nature
Bibliographic data for series maintained by Sonal Shukla () and Springer Nature Abstracting and Indexing ().