Optimization of trans-Splicing for Huntington's Disease RNA Therapy

por Rindt, Hansjörg, Tom, Colton M., Lorson, Christian L., Mattis, Virginia B.
Publicado 2017

Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA

por Mattis, Virginia B, Fosso, Marina Y, Chang, Cheng-Wei, Lorson, Christian L
Publicado 2009

Characterization of Neurodevelopmental Abnormalities in iPSC-Derived Striatal Cultures from Patients with Huntington’s Disease

por Mathkar, Pranav P., Suresh, Divya, Dunn, James, Tom, Colton M., Mattis, Virginia B.
Publicado 2019

Stimulation of GABA-Induced Ca(2+) Influx Enhances Maturation of Human Induced Pluripotent Stem Cell-Derived Neurons

por Rushton, David J., Mattis, Virginia B., Svendsen, Clive N., Allen, Nicholas D., Kemp, Paul J.
Publicado 2013

Huntington’s Disease Patient-Derived Astrocytes Display Electrophysiological Impairments and Reduced Neuronal Support

por Garcia, Veronica J., Rushton, David J., Tom, Colton M., Allen, Nicholas D., Kemp, Paul J., Svendsen, Clive N., Mattis, Virginia B.
Publicado 2019

Optimizing maturity and dose of iPSC-derived dopamine progenitor cell therapy for Parkinson’s disease

por Hiller, Benjamin M., Marmion, David J., Thompson, Cayla A., Elliott, Nathaniel A., Federoff, Howard, Brundin, Patrik, Mattis, Virginia B., McMahon, Christopher W., Kordower, Jeffrey H.
Publicado 2022

The Interaction of Aging and Cellular Stress Contributes to Pathogenesis in Mouse and Human Huntington Disease Neurons

por Machiela, Emily, Jeloka, Ritika, Caron, Nicholas S., Mehta, Shagun, Schmidt, Mandi E., Baddeley, Helen J. E., Tom, Colton M., Polturi, Nalini, Xie, Yuanyun, Mattis, Virginia B., Hayden, Michael R., Southwell, Amber L.
Publicado 2020

A patient-derived cellular model for Huntington’s disease reveals phenotypes at clinically relevant CAG lengths

por Hung, Claudia Lin-Kar, Maiuri, Tamara, Bowie, Laura Erin, Gotesman, Ryan, Son, Susie, Falcone, Mina, Giordano, James Victor, Gillis, Tammy, Mattis, Virginia, Lau, Trevor, Kwan, Vickie, Wheeler, Vanessa, Schertzer, Jonathan, Singh, Karun, Truant, Ray
Publicado 2018

The difficulty to model Huntington’s disease in vitro using striatal medium spiny neurons differentiated from human induced pluripotent stem cells

por Le Cann, Kim, Foerster, Alec, Rösseler, Corinna, Erickson, Andelain, Hautvast, Petra, Giesselmann, Sebastian, Pensold, Daniel, Kurth, Ingo, Rothermel, Markus, Mattis, Virginia B., Zimmer-Bensch, Geraldine, von Hörsten, Stephan, Denecke, Bernd, Clarner, Tim, Meents, Jannis, Lampert, Angelika
Publicado 2021

Inducible Expression of GDNF in Transplanted iPSC-Derived Neural Progenitor Cells

por Akhtar, Aslam Abbasi, Gowing, Genevieve, Kobritz, Naomi, Savinoff, Steve E., Garcia, Leslie, Saxon, David, Cho, Noell, Kim, Gibum, Tom, Colton M., Park, Hannah, Lawless, George, Shelley, Brandon C., Mattis, Virginia B., Breunig, Joshua J., Svendsen, Clive N.
Publicado 2018