AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich's ataxia.

"Declaration of interests J.C.C., M.R.R., M.C.S., S.L., F.B., C.A.J., M.C., B.M., and C.F. are employees of and/or own equity in Astellas Gene Therapies. P.P. and F.U. are former employees of Astellas Gene Therapies. H.M. is an employee of Diverge Translational Science Laboratory. M.W.L. is the founder, owner, and chief executive officer of Diverge Translational Science Laboratory; participated in scientific advisory boards sponsored by Solid Biosciences, Vial, Astellas Gene Therapies, and Ichorion Therapeutics (formerly); received research support from Astellas Gene Therapies, Solid Biosciences, Taysha Gene Therapies, Kate Therapeutics, Prothelia, Ultragenyx, Dynacure, Rocket Pharmaceuticals, Carbon Biosciences, and Voyager Therapeutics; and a paid consultant to Astellas Gene Therapies, Encoded Therapeutics, Lacerta Therapeutics, Affinia Therapeutics, AGADA Biosciences, Modis Therapeutics, Dynacure, BioMarin, Vertex Pharmaceuticals, Locanabio, Rocket Pharmaceuticals, and Entrada Therapeutics. D.R.L. has received grant funding from Astellas, PTC, Reata, Design, and Novartis."

"This research was funded by Astellas Gene Therapies. Medical writing support was provided by Laurie LaRusso, MS, ELS, of Chestnut Medical Communications, paid for by Astellas Gene Therapies. Additional editorial support and preparation of artwork files were provided by Joanne Fitz-Gerald, B.Pharm. and Jonathan A.C. Lee, Ph.D. of FourWave Medical Communications, funded by Astellas Gene Therapies."