Biological Aging and the Cellular Pathogenesis of Huntington’s Disease

por Machiela, Emily, Southwell, Amber L.
Publicado 2020

Antisense Oligonucleotide Therapy: From Design to the Huntington Disease Clinic

por Rook, Morgan E., Southwell, Amber L.
Publicado 2022

Striatal Synaptic Dysfunction and Hippocampal Plasticity Deficits in the Hu97/18 Mouse Model of Huntington Disease

por Kolodziejczyk, Karolina, Parsons, Matthew P., Southwell, Amber L., Hayden, Michael R., Raymond, Lynn A.
Publicado 2014

Perturbation with Intrabodies Reveals That Calpain Cleavage Is Required for Degradation of Huntingtin Exon 1

por Southwell, Amber L., Bugg, Charles W., Kaltenbach, Linda S., Dunn, Denise, Butland, Stefanie, Weiss, Andreas, Paganetti, Paolo, Lo, Donald C., Patterson, Paul H.
Publicado 2011

Sudden death due to paralysis and synaptic and behavioral deficits when Hip14/Zdhhc17 is deleted in adult mice

por Sanders, Shaun S., Parsons, Matthew P., Mui, Katherine K. N., Southwell, Amber L., Franciosi, Sonia, Cheung, Daphne, Waltl, Sabine, Raymond, Lynn A., Hayden, Michael R.
Publicado 2016

Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease

por Miniarikova, Jana, Zanella, Ilaria, Huseinovic, Angelina, van der Zon, Tom, Hanemaaijer, Evelyn, Martier, Raygene, Koornneef, Annemart, Southwell, Amber L, Hayden, Michael R, van Deventer, Sander J, Petry, Harald, Konstantinova, Pavlina
Publicado 2016

Mice lacking caspase-2 are protected from behavioral changes, but not pathology, in the YAC128 model of Huntington disease

por Carroll, Jeffrey B, Southwell, Amber L, Graham, Rona K, Lerch, Jason P, Ehrnhoefer, Dagmar E, Cao, Li-Ping, Zhang, Wei-Ning, Deng, Yu, Bissada, Nagat, Henkelman, R Mark, Hayden, Michael R
Publicado 2011

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

Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS

por Østergaard, Michael E., Southwell, Amber L., Kordasiewicz, Holly, Watt, Andrew T., Skotte, Niels H., Doty, Crystal N., Vaid, Kuljeet, Villanueva, Erika B., Swayze, Eric E., Frank Bennett, C., Hayden, Michael R., Seth, Punit P.
Publicado 2013

Ultrasensitive measurement of huntingtin protein in cerebrospinal fluid demonstrates increase with Huntington disease stage and decrease following brain huntingtin suppression

por Southwell, Amber L., Smith, Stephen E.P., Davis, Tessa R., Caron, Nicholas S., Villanueva, Erika B., Xie, Yuanyun, Collins, Jennifer A., Li Ye, Min, Sturrock, Aaron, Leavitt, Blair R., Schrum, Adam G., Hayden, Michael R.
Publicado 2015

Preventing mutant huntingtin proteolysis and intermittent fasting promote autophagy in models of Huntington disease

por Ehrnhoefer, Dagmar E., Martin, Dale D. O., Schmidt, Mandi E., Qiu, Xiaofan, Ladha, Safia, Caron, Nicholas S., Skotte, Niels H., Nguyen, Yen T. N., Vaid, Kuljeet, Southwell, Amber L., Engemann, Sabine, Franciosi, Sonia, Hayden, Michael R.
Publicado 2018

Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease

por Caron, Nicholas S, Southwell, Amber L, Brouwers, Cynthia C, Cengio, Louisa Dal, Xie, Yuanyun, Black, Hailey Findlay, Anderson, Lisa M, Ko, Seunghyun, Zhu, Xiang, van Deventer, Sander J, Evers, Melvin M, Konstantinova, Pavlina, Hayden, Michael R
Publicado 2020

Allele-Specific Suppression of Mutant Huntingtin Using Antisense Oligonucleotides: Providing a Therapeutic Option for All Huntington Disease Patients

por Skotte, Niels H., Southwell, Amber L., Østergaard, Michael E., Carroll, Jeffrey B., Warby, Simon C., Doty, Crystal N., Petoukhov, Eugenia, Vaid, Kuljeet, Kordasiewicz, Holly, Watt, Andrew T., Freier, Susan M., Hung, Gene, Seth, Punit P., Bennett, C. Frank, Swayze, Eric E., Hayden, Michael R.
Publicado 2014