Preclinical Development and Safety of Laterally Spreading Dual AAV.SPR-MYO7A Vectors for the Treatment of Usher Syndrome Type 1B
Kaitlyn R. Calabro, Eva Andrés‐Mateos, Christopher Kozlowski, Paige S. Nemec, Emily M. Xu, Sean M. Crosson, Diego S. Fajardo, Hannah L. Friend, James J. Peterson, Wei Li, Hangning Zhang, Hugh Lee Sweeney, Victoria A. Stevenson, Howard Rind, Matthew Leahy, , Shannon E. Boye
This paper develops and tests dual AAV vectors for treating USH1B by reconstituting full-length MYO7A expression, showing safety and efficacy in non-human primates.
bioRxiv · Oct 29, 2026 · PreprintGenetics