Ibraheim, Raed http://orcid.org/0000-0002-0961-7299
Tai, Phillip W. L. http://orcid.org/0000-0001-7409-8344
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 http://orcid.org/0000-0001-7754-8945
Wolfe, Scot A. http://orcid.org/0000-0002-7042-201X
Wang, Dan http://orcid.org/0000-0001-9079-2360
Pai, Athma A.
Xue, Wen http://orcid.org/0000-0002-9797-8042
Gao, Guangping http://orcid.org/0000-0003-0097-9012
Sontheimer, Erik J. http://orcid.org/0000-0002-0881-0310
Funding for this research was provided by:
U.S. Department of Health & Human Services | NIH | National Institute of General Medical Sciences (R01GM125797, R01GM115911)
U.S. Department of Health & Human Services | NIH | National Institute of General Medical Sciences
Article History
Received: 3 November 2020
Accepted: 6 October 2021
First Online: 1 November 2021
Competing interests
: The authors declare the following competing interests: A patent application has been filed on technologies related to this work by the University of Massachusetts Medical School [Names of inventors: Erik J. Sontheimer, Raed Ibraheim, Wen Xue, Aamir Mir, Alireza Edraki, Ildar Gainetdinov. Application number: 16/186,352. Status of application: Pending.] The following aspects of the manuscript are covered in the patent application: (1) Truncating of sgRNA sequence to create 121-nt sgRNA versions (Nme.sgRNA-121). (2) Truncating of sgRNA sequence to create 100-nt sgRNA versions (Nme.sgRNA-100). (3) Minimized all-in-one AAV.Nme2Cas9.sgRNA vector backbone to a packaging size of 4.4 Kb including its promoters and regulatory signals. (4) Dual-sgRNA AAV.NmeCas9 vector backbones. G.G. is a scientific co-founder of Voyager Therapeutics, Adrenas Therapeutics, and Aspa Therapeutics and holds equity in these companies. G.G. is an inventor on patents with potential royalties licensed to Voyager Therapeutics, Aspa Therapeutics, and other biopharmaceutical companies. E.J.S. is a co-founder and scientific advisor of Intellia Therapeutics. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. The authors declare no competing interests.