Testing a dual AAV gene therapy vector construct to treat sialidosis and galactosialidosis using small and large animal models

Molecular Genetics and Metabolism 2026

View at publisher
Authors: Jillian Gallagher, Stephanie Brownell, Luz Yvette Lopez Diaz, Hector Ribeiro Benatti, Sarah Nath, Sophia Esteves, Judith Gallant, Ping Xu, Stephanie Bertrand, Ainsley LaFlamme, Rachael E. Gately, Rachel Prestigiacomo
+6 more Alessandra d’Azzo, Cynthia J. Tifft, Scot A. Wolfe, Jennifer W. Koehler, Miguel Sena‐Esteves, Heather L. Gray‐Edwards
DOI: 10.1016/j.ymgme.2025.109418
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Virus-based gene therapy research RNA Interference and Gene Delivery

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109418–109418
Publication date
1 February 2026
Language
EN