Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia

Por Keiser, Megan S., Kordasiewicz, Holly B., McBride, Jodi L.
Publicado no Hum Mol Genet (2016)

Dominant-Negative Suppression of Ca(v)2.1 Currents by α(1)2.1 Truncations Requires the Conserved Interaction Domain for β Subunits

Por Raike, Robert S., Kordasiewicz, Holly B., Thompson, Randall M., Gomez, Christopher M.
Publicado em 2006

Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models

Por Moore, Lauren R., Rajpal, Gautam, Dillingham, Ian T., Qutob, Maya, Blumenstein, Kate G., Gattis, Danielle, Hung, Gene, Kordasiewicz, Holly B., Paulson, Henry L., McLoughlin, Hayley S.
Publicado no Mol Ther Nucleic Acids (2017)

Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression

Por Yu, Dongbo, Pendergraff, Hannah, Liu, Jing, Kordasiewicz, Holly B., Cleveland, Don W., Swayze, Eric E., Lima, Walt F., Crooke, Stanley T., Prakash, Thazha P., Corey, David R.
Publicado em 2012

LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson’s Disease Mouse Model

Por Zhao, Hien Tran, John, Neena, Delic, Vedad, Ikeda-Lee, Karli, Kim, Aneeza, Weihofen, Andreas, Swayze, Eric E., Kordasiewicz, Holly B., West, Andrew B., Volpicelli-Daley, Laura A.
Publicado no Mol Ther Nucleic Acids (2017)

PMP22 antisense oligonucleotides reverse Charcot-Marie-Tooth disease type 1A features in rodent models

Por Zhao, Hien Tran, Damle, Sagar, Ikeda-Lee, Karli, Kuntz, Steven, Li, Jian, Mohan, Apoorva, Kim, Aneeza, Hung, Gene, Scheideler, Mark A., Scherer, Steven S., Svaren, John, Swayze, Eric E., Kordasiewicz, Holly B.
Publicado no J Clin Invest (2017)

Sustained therapeutic reversal of Huntington’s disease by transient repression of huntingtin synthesis

Por 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.
Publicado em 2012

Peripheral huntingtin silencing does not ameliorate central signs of disease in the B6.Htt(Q111/+) mouse model of Huntington’s disease

Por Coffey, Sydney R., Bragg, Robert M., Minnig, Shawn, Ament, Seth A., Cantle, Jeffrey P., Glickenhaus, Anne, Shelnut, Daniel, Carrillo, José M., Shuttleworth, Dominic D., Rodier, Julie-Anne, Noguchi, Kimihiro, Bennett, C. Frank, Price, Nathan D., Kordasiewicz, Holly B., Carroll, Jeffrey B.
Publicado no PLoS One (2017)

In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides

Por Southwell, Amber L, Skotte, Niels H, Kordasiewicz, Holly B, Østergaard, Michael E, Watt, Andrew T, Carroll, Jeffrey B, Doty, Crystal N, Villanueva, Erika B, Petoukhov, Eugenia, Vaid, Kuljeet, Xie, Yuanyun, Freier, Susan M, Swayze, Eric E, Seth, Punit P, Bennett, Clarence Frank, Hayden, Michael R
Publicado no Mol Ther (2014)

Tau Reduction Prevents Neuronal Loss and Reverses Pathological Tau Deposition and Seeding in Mice with Tauopathy

Por DeVos, Sarah L., Miller, Rebecca L., Schoch, Kathleen M., Holmes, Brandon B., Kebodeaux, Carey S., Wegener, Amy J., Chen, Guo, Shen, Tao, Tran, Hien, Nichols, Brandon, Zanardi, Tom A., Kordasiewicz, Holly B., Swayze, Eric E., Bennett, C. Frank, Diamond, Marc I., Miller, Timothy M.
Publicado no Sci Transl Med (2017)

MicroRNA Profiling Reveals Marker of Motor Neuron Disease in ALS Models

Por Hoye, Mariah L., Koval, Erica D., Wegener, Amy J., Hyman, Theodore S., Yang, Chengran, O'Brien, David R., Miller, Rebecca L., Cole, Tracy, Schoch, Kathleen M., Shen, Tao, Kunikata, Tomonori, Richard, Jean-Philippe, Gutmann, David H., Maragakis, Nicholas J., Kordasiewicz, Holly B., Dougherty, Joseph D., Miller, Timothy M.
Publicado no J Neurosci (2017)

ALS-linked TDP-43 mutations produce aberrant RNA splicing and adult-onset motor neuron disease without aggregation or loss of nuclear TDP-43

Por Arnold, Eveline S., Ling, Shuo-Chien, Huelga, Stephanie C., Lagier-Tourenne, Clotilde, Polymenidou, Magdalini, Ditsworth, Dara, Kordasiewicz, Holly B., McAlonis-Downes, Melissa, Platoshyn, Oleksandr, Parone, Philippe A., Da Cruz, Sandrine, Clutario, Kevin M., Swing, Debbie, Tessarollo, Lino, Marsala, Martin, Shaw, Christopher E., Yeo, Gene W., Cleveland, Don W.
Publicado em 2013

Tcf4 Regulates Synaptic Plasticity, DNA Methylation, and Memory Function

Por Kennedy, Andrew J., Rahn, Elizabeth J., Paulukaitis, Brynna S., Savell, Katherine E., Kordasiewicz, Holly B., Wang, Jing, Lewis, John W., Posey, Jessica, Strange, Sarah K., Guzman-Karlsson, Mikael C., Phillips, Scott E., Decker, Kyle, Motley, S. Timothy, Swayze, Eric E., Ecker, David J., Michael, Todd P., Day, Jeremy J., Sweatt, J. David
Publicado no Cell Rep (2016)