Publication
11/11/2020
MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates.
Hordeaux J, Buza EL, Jeffrey B, Song C, Jahan T, Yuan Y, Zhu Y, Bell P, Li M, Chichester JA, Calcedo R, Wilson JM
Science Translational Medicine
Publication
9/16/2020
Adeno-associated virus serotype 1-based gene therapy for FTD caused by GRN mutations.
Hinderer C, Miller R, Dyer C, Johansson J, Bell P, Buza E, Wilson JM
Annals Of Clinical And Translational Neurology
Publication
4/18/2020
Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration.
Hinderer C, Katz N, Dyer C, Goode T, Johansson J, Bell P, Richman L, Buza E, Wilson JM
Molecular Therapy. Methods & Clinical Development
Publication
2/12/2020
A Mutation-Independent CRISPR-Cas9-Mediated Gene Targeting Approach to Treat a Murine Model of Ornithine Transcarbamylase Deficiency
Wang L, Yang Y, Breton C, Bell P, Li M, Zhang J, Che Y, Saveliev A, He Z, White J, Latshaw C, Xu C, McMenamin D, Yu H, Morizono H, Batshaw ML, Wilson JM
Science Advances
