The prospect of molecular therapy for Angelman syndrome and other monogenic neurologic disorders

por Bailus, Barbara J, Segal, David J
Publicado 2014

Protein Delivery of an Artificial Transcription Factor Restores Widespread Ube3a Expression in an Angelman Syndrome Mouse Brain

por Bailus, Barbara J, Pyles, Benjamin, McAlister, Michelle M, O'Geen, Henriette, Lockwood, Sarah H, Adams, Alexa N, Nguyen, Jennifer Trang T, Yu, Abigail, Berman, Robert F, Segal, David J
Publicado 2016

Genomic Analysis Reveals Disruption of Striatal Neuronal Development and Therapeutic Targets in Human Huntington’s Disease Neural Stem Cells

por Ring, Karen L., An, Mahru C., Zhang, Ningzhe, O’Brien, Robert N., Ramos, Eliana Marisa, Gao, Fuying, Atwood, Robert, Bailus, Barbara J., Melov, Simon, Mooney, Sean D., Coppola, Giovanni, Ellerby, Lisa M.
Publicado 2015

Modulating FKBP5/FKBP51 and autophagy lowers HTT (huntingtin) levels

por Bailus, Barbara J., Scheeler, Stephen M., Simons, Jesse, Sanchez, Maria A., Tshilenge, Kizito-Tshitoko, Creus-Muncunill, Jordi, Naphade, Swati, Lopez-Ramirez, Alejandro, Zhang, Ningzhe, Lakshika Madushani, Kuruwitage, Moroz, Stanislav, Loureiro, Ashley, Schreiber, Katherine H., Hausch, Felix, Kennedy, Brian K., Ehrlich, Michelle E., Ellerby, Lisa M.
Publicado 2021