Alpha Protein Kinase 3 Gene Therapy Restores Heart Function in Mouse and Human Models of Cardiomyopathy
James W. McNamara, Ellen B. Keen, Rebecca Sutton, Yinghan She, Hannah Huckstep, Lina H.H. Le, Neda R. Mehdiabadi, Madhavan S. Venkatesh, Brendan Griffen, Richard J. Mills, Christina V. Theodoris, James E. Hudson, Drew M. Titmarsh, Sean J. Humphrey, Enzo R. Porrello, David A. Elliott
This study shows that gene therapy with AAV-ALPK3 improves heart function in mouse and human models of cardiomyopathy caused by ALPK3 variants.
Nature Cardiovascular Research · Aug 19, 2026Peer-reviewedPhysiology