Search Results - "Bailus, Barbara J."

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

“…We utilized induced pluripotent stem cells (iPSCs) derived from Huntington’s disease (HD) patients as a human model of HD and determined that the disease…”
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In Vivo Applications of Cell-Penetrating Zinc-Finger Transcription Factors by Ren, Chonghua, Adams, Alexa N, Pyles, Benjamin, Bailus, Barbara J, O'Geen, Henriette, Segal, David J

“…Artificial transcription factors based on zinc finger, TALE, and CRISPR/Cas9 programmable DNA-binding platforms have been widely used to regulate the…”
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Modulating FKBP5/FKBP51 and autophagy lowers HTT (huntingtin) levels by 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.

“…Current disease-modifying therapies for Huntington disease (HD) focus on lowering mutant HTT (huntingtin; mHTT) levels, and the immunosuppressant drug…”
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iPSC-based drug screening for Huntington's disease by Zhang, Ningzhe, Bailus, Barbara J, Ring, Karen L, Ellerby, Lisa M

“…Huntington's disease (HD) is an autosomal dominant neurodegenerative disorder, caused by an expansion of the CAG repeat in exon 1 of the huntingtin gene. The…”
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Protein Delivery of an Artificial Transcription Factor Restores Widespread Ube3a Expression in an Angelman Syndrome Mouse Brain by 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

“…Angelman syndrome (AS) is a neurological genetic disorder caused by loss of expression of the maternal copy of UBE3A in the brain. Due to brain-specific…”
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Purified Protein Delivery to Activate an Epigenetically Silenced Allele in Mouse Brain by Pyles, Benjamin, Bailus, Barbara J, O'Geen, Henriette, Segal, David J

“…The ability to activate or repress specific genes in the brain could have a tremendous impact for understanding and treating neurological disorders. Artificial…”
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The prospect of molecular therapy for Angelman syndrome and other monogenic neurologic disorders by Bailus, Barbara J, Segal, David J

“…Angelman syndrome is a monogenic neurologic disorder that affects 1 in 15,000 children, and is characterized by ataxia, intellectual disability, speech…”
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iPSC-based drug screening for Huntington׳s disease by Zhang, Ningzhe, Bailus, Barbara J, Ring, Karen L, Ellerby, Lisa M

“…Abstract Huntington׳s disease (HD) is an autosomal dominant neurodegenerative disorder, caused by an expansion of the CAG repeat in exon 1 of the huntingtin…”
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