Intra-striatal AAV2.retro administration leads to extensive retrograde transport in the rhesus macaque brain: implications for disease modeling and therapeutic development

por Weiss, Alison R., Liguore, William A., Domire, Jacqueline S., Button, Dana, McBride, Jodi L.
Publicado 2020

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

por Ansseau, Eugénie, Domire, Jacqueline S., Wallace, Lindsay M., Eidahl, Jocelyn O., Guckes, Susan M., Giesige, Carlee R., Pyne, Nettie K., Belayew, Alexandra, Harper, Scott Q.
Publicado 2015

A novel rhesus macaque model of Huntington’s disease recapitulates key neuropathological changes along with motor and cognitive decline

por Weiss, Alison R, Liguore, William A, Brandon, Kristin, Wang, Xiaojie, Liu, Zheng, Domire, Jacqueline S, Button, Dana, Srinivasan, Sathya, Kroenke, Christopher D, McBride, Jodi L
Publicado 2022

Mouse Dux is myotoxic and shares partial functional homology with its human paralog DUX4

por Eidahl, Jocelyn O., Giesige, Carlee R., Domire, Jacqueline S., Wallace, Lindsay M., Fowler, Allison M., Guckes, Susan M., Garwick-Coppens, Sara E., Labhart, Paul, Harper, Scott Q.
Publicado 2016

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

por Wallace, Lindsay M., Saad, Nizar Y., Pyne, Nettie K., Fowler, Allison M., Eidahl, Jocelyn O., Domire, Jacqueline S., Griffin, Danielle A., Herman, Adam C., Sahenk, Zarife, Rodino-Klapac, Louise R., Harper, Scott Q.
Publicado 2017

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

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