Development of new adeno-associated virus capsid variants for targeted gene delivery to human cardiomyocytes.

"Declaration of interests C.Y.K., S.L.G., M.C.-C., I.E.A., L.L., and E.K. are inventors on patent applications filed by Children’s Medical Research Institute related to AAV capsid sequences and in vivo function of novel AAV variants. L.L. is a cofounder and a scientific advisor at LogicBio Therapeutics. L.L. and I.E.A. are co-founders of Exigen Biotherapeutics. L.L. has a sponsored research agreement with LogicBio Therapeutics. M.C.-C., I.E.A., and L.L. have IP licensed to biopharmaceutical companies. L.L. and I.A.E. have consulted on technologies addressed in this paper. L.L. and I.A.E. have stock and/or equity in companies with technology broadly related to this paper. J.E.H. is a co-founder, scientific advisor, and stockholder in Dynomics. All other authors declare no conflicts of interest."

"We thank CMRI Vector and Genome Engineering Facility for help in vector preparation. Flow cytometry, live cell imaging using the Zeiss microscope, and PCR were performed at the Westmead Scientific Platforms, which are supported by the Westmead Research Hub, the Westmead Institute for Medical Research, the 10.13039/501100001171Cancer Institute New South Wales, the 10.13039/501100000925National Health and Medical Research Council, and the 10.13039/501100001047Ian Potter Foundation. This work was supported by NHMRC Project grant GNT1128864. C.Y.K. was supported by NHMRC Ideas grant APP1184929. L.L. was supported by NHMRC Project grants APP1156431 and APP1161583, and a grant from the National Science Centre, Republic of Poland (OPUS 13) (UMO-2017/25/B/NZ1/02790). J.E.H. is supported by a Snow Medical Fellowship. E.K. was funded by NHMRC Ideas grants APP1184929 and APP1188348 and NSW Health – Cardiovascular Research Capacity Program. R.M. and M.M were funded by the 10.13039/100000002NIH grant R01 NIH GM082946."