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Authors
Smidler, Andrea L.
Marrogi, Eryney A.
Scott, Sean
Mameli, Enzo
Abernathy, Daniel
Akbari, Omar S.
Church, George M.
Catteruccia, Flaminia
Esvelt, Kevin
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Funding
Funding for this research was provided by:
Foundation for the National Institutes of Health (AI120480-02, R01 AI104956, R01 AI104956, R01AI151004, R01 AI104956, R00-DK102669-04)
Burroughs Wellcome Fund (IRSA1016432, IRSA1016432, IRSA1016432)
Howard Hughes Medical Institute (OPP1158190)
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Article History
Received: 4 March 2025
Accepted: 2 September 2025
First Online: 8 October 2025
Declarations
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: O.S.A is a founder of Agragene, Inc. and Synvect, Inc. with equity interest. The terms of this arrangement have been reviewed and approved by the University of California, San Diego in accordance with its conflict of interest policies. G.M.C. has the following patents related to this work: WO2015006290A1 (“Multiplex RNA-guided genome engineering”) and WO2016089866A1 (“RNA-guided systems for in vivo gene editing”). A complete list of G.M.C’s conflict of interest can be found at arep.med.harvard.edu/gmc/tech.html. K.M.E. and A.L.S have pending patents related to the described work: WO2015105928A1 (“RNA-guided gene drives”) and K.M.E. has an additional patent WO2015006294A2 (“Orthogonal Cas9 proteins for RNA-guided gene regulation and editing”). All other authors (E.M., S.S., E.M., D.A., F.C.) declare no competing interests.
: All animals were handled in accordance with the Guide for the Care and Use of Laboratory Animals as recommended by the National Institutes of Health and approved by the UCSD Institutional Animal Care and Use Committee (IACUC, Animal Use Protocol #S17187) and UCSD Biological Use Authorization (BUA #R2401).
