Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis
Kordasiewicz, Holly B., Stanek, Lisa M., Wancewicz, Edward V., Mazur, Curt, McAlonis, Melissa M., Pytel, Kimberly A., Artates, Jonathan W., Weiss, Andreas, Cheng, Seng H., Shihabuddin, Lamya S., Hung, Gene, Bennett, C. Frank, Cleveland, Don W.
Published in Neuron (Cambridge, Mass.) (21.06.2012)
Published in Neuron (Cambridge, Mass.) (21.06.2012)
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Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy
Passini, Marco A, Bu, Jie, Richards, Amy M, Kinnecom, Cathrine, Sardi, S Pablo, Stanek, Lisa M, Hua, Yimin, Rigo, Frank, Matson, John, Hung, Gene, Kaye, Edward M, Shihabuddin, Lamya S, Krainer, Adrian R, Bennett, C Frank, Cheng, Seng H
Published in Science translational medicine (02.03.2011)
Published in Science translational medicine (02.03.2011)
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CNS expression of glucocerebrosidase corrects α-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy
Sardi, S. Pablo, Clarke, Jennifer, Kinnecom, Cathrine, Tamsett, Thomas J., Li, Lingyun, Stanek, Lisa M., Passini, Marco A., Grabowski, Gregory A., Schlossmacher, Michael G., Sidman, Richard L., Cheng, Seng H., Shihabuddin, Lamya S.
Published in Proceedings of the National Academy of Sciences - PNAS (19.07.2011)
Published in Proceedings of the National Academy of Sciences - PNAS (19.07.2011)
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Extensive Transduction and Enhanced Spread of a Modified AAV2 Capsid in the Non-human Primate CNS
Naidoo, Jerusha, Stanek, Lisa M., Ohno, Kousaku, Trewman, Savanah, Samaranch, Lluis, Hadaczek, Piotr, O’Riordan, Catherine, Sullivan, Jennifer, San Sebastian, Waldy, Bringas, John R., Snieckus, Christopher, Mahmoodi, Amin, Mahmoodi, Amir, Forsayeth, John, Bankiewicz, Krystof S., Shihabuddin, Lamya S.
Published in Molecular therapy (03.10.2018)
Published in Molecular therapy (03.10.2018)
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Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease
Stanek, Lisa M, Sardi, Sergio P, Mastis, Bryan, Richards, Amy R, Treleaven, Christopher M, Taksir, Tatyana, Misra, Kuma, Cheng, Seng H, Shihabuddin, Lamya S
Published in Human gene therapy (01.05.2014)
Published in Human gene therapy (01.05.2014)
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Marked differences in neurochemistry and aggregates despite similar behavioural and neuropathological features of Huntington disease in the full-length BACHD and YAC128 mice
POULADI, Mahmoud A, STANEK, Lisa M, HAYDEN, Michael R, YUANYUN XIE, FRANCIOSI, Sonia, SOUTHWELL, Amber L, YU DENG, BUTLAND, Stefanie, WEINING ZHANG, CHENG, Seng H, SHIHABUDDIN, Lamya S
Published in Human molecular genetics (15.05.2012)
Published in Human molecular genetics (15.05.2012)
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Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain
Sullivan, Jennifer A., Stanek, Lisa M., Lukason, Michael J., Bu, Jie, Osmond, Shayla R., Barry, Elizabeth A., O’Riordan, Catherine R., Shihabuddin, Lamya S., Cheng, Seng H., Scaria, Abraham
Published in Gene therapy (01.06.2018)
Published in Gene therapy (01.06.2018)
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Partial rescue of some features of Huntington Disease in the genetic absence of caspase-6 in YAC128 mice
Wong, Bibiana K.Y, Ehrnhoefer, Dagmar E, Graham, Rona K, Martin, Dale D.O, Ladha, Safia, Uribe, Valeria, Stanek, Lisa M, Franciosi, Sonia, Qiu, Xiaofan, Deng, Yu, Kovalik, Vlad, Zhang, Weining, Pouladi, Mahmoud A, Shihabuddin, Lamya S, Hayden, Michael R
Published in Neurobiology of disease (01.04.2015)
Published in Neurobiology of disease (01.04.2015)
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Time-resolved FRET screening identifies small molecular modifiers of mutant Huntingtin conformational inflexibility in patient-derived cells
Wilbertz, Johannes H., Frappier, Julia, Muller, Sandra, Gratzer, Sabine, Englaro, Walter, Stanek, Lisa M., Calamini, Barbara
Published in SLAS discovery (01.06.2022)
Published in SLAS discovery (01.06.2022)
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The engineered AAV2-HBKO promotes non-invasive gene delivery to large brain regions beyond ultrasound targeted sites
Kofoed, Rikke Hahn, Noseworthy, Kate, Wu, Kathleen, Sivadas, Shuruthisai, Stanek, Lisa, Elmer, Bradford, Hynynen, Kullervo, Shihabuddin, Lamya S., Aubert, Isabelle
Published in Molecular therapy. Methods & clinical development (08.12.2022)
Published in Molecular therapy. Methods & clinical development (08.12.2022)
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Antisense oligonucleotide-mediated correction of transcriptional dysregulation is correlated with behavioral benefits in the YAC128 mouse model of Huntington's disease
Stanek, Lisa M, Yang, Wendy, Angus, Stuart, Sardi, Pablo S, Hayden, Michael R, Hung, Gene H, Bennett, C Frank, Cheng, Seng H, Shihabuddin, Lamya S
Published in Journal of Huntington's disease (2013)
Published in Journal of Huntington's disease (2013)
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Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease
Hadaczek, Piotr, Stanek, Lisa, Ciesielska, Agnieszka, Sudhakar, Vivek, Samaranch, Lluis, Pivirotto, Philip, Bringas, John, O'Riordan, Catherine, Mastis, Bryan, San Sebastian, Waldy, Forsayeth, John, Cheng, Seng H, Bankiewicz, Krystof S, Shihabuddin, Lamya S
Published in Molecular therapy. Methods & clinical development (01.01.2016)
Published in Molecular therapy. Methods & clinical development (01.01.2016)
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Efficient and Precise Processing of the Optimized Primary Artificial MicroRNA in a Huntingtin-Lowering Adeno-Associated Viral Gene Therapy In Vitro and in Mice and Nonhuman Primates
Wang, Wei, Zhou, Pengcheng, Wang, Xin, Chen, Fen, Christensen, Emily, Thompson, Jeffrey, Ren, Xiaoqin, Kells, Adrian, Stanek, Lisa, Carter, Todd, Hou, Jay, Sah, Dinah W Y
Published in Human gene therapy (01.01.2022)
Published in Human gene therapy (01.01.2022)
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A50 Hypothalamic suppression of mutant huntingtin restored proteins involved in energy metabolism
Stricker-Shaver, Janice, Fabry, Benedikt, Yu-Taeger, Libo, Singer, Elisabeth, Stanek, Lisa, Calaminus, Carsten, Pichler, Bernd J, Shihabuddin, Lamya S, Riess, Olaf, Nguyen, Huu Phuc
Published in Journal of neurology, neurosurgery and psychiatry (01.09.2018)
Published in Journal of neurology, neurosurgery and psychiatry (01.09.2018)
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