Aberrant splicing in transgenes containing introns, exons, and V5 epitopes: lessons from developing an FSHD mouse model expressing a D4Z4 repeat with flanking genomic sequences.

by Eugénie Ansseau, Jacqueline S Domire, Lindsay M Wallace, Jocelyn O Eidahl, Susan M Guckes, Carlee R Giesige, Nettie K Pyne, Alexandra Belayew, Scott Q Harper
Published 2015-01-01

Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD

by Lindsay M. Wallace, Nizar Y. Saad, Nettie K. Pyne, Allison M. Fowler, Jocelyn O. Eidahl, Jacqueline S. Domire, Danielle A. Griffin, Adam C. Herman, Zarife Sahenk, Louise R. Rodino-Klapac, Scott Q. Harper
Published 2018-03-01

Discovery of a CLN7 model of Batten disease in non-human primates

by Jodi L. McBride, Martha Neuringer, Betsy Ferguson, Steven G. Kohama, Ian J. Tagge, Robert C. Zweig, Laurie M. Renner, Trevor J. McGill, Jonathan Stoddard, Samuel Peterson, Weiping Su, Larry S. Sherman, Jacqueline S. Domire, Rebecca M. Ducore, Lois M. Colgin, Anne D. Lewis
Published 2018-11-01