AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life.

"Declaration of interests The authors declare no competing interests."

"The authors would like to sincerely thank the owners of these companion dogs for entrusting their pets to this research study and the primary care veterinarians who helped with case management and sample collection. We also acknowledge research funding from 10.13039/100000050NHLBI (HL158781 to V.R.A. and B.J.S.J., HL156073 to B.S.D., and N01_75N92019D00041 to T.C.N.), the 10.13039/100006108National Center for Advancing Translational Sciences (UL1TR000003 to V.R.A. and M.B.C.) and the Institute for Translational Medicine and Therapeutics of the Perelman School of Medicine and the School of Veterinary Medicine at the University of Pennsylvania (V.R.A. and M.B.C.). We thank Rodney Camire for provision of factor Xa I16L protein and Denise Sabatino for providing bleeding data on colony dogs. Finally, we thank the Children’s Hospital of Philadelphia’s Research Vector Core for their help in producing the AAV vectors."