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 em 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 no Sci Rep (2015)

A fully humanized transgenic mouse model of Huntington disease

Por Southwell, Amber L., Warby, Simon C., Carroll, Jeffrey B., Doty, Crystal N., Skotte, Niels H., Zhang, Weining, Villanueva, Erika B., Kovalik, Vlad, Xie, Yuanyun, Pouladi, Mahmoud A., Collins, Jennifer A., Yang, X. William, Franciosi, Sonia, Hayden, Michael R.
Publicado em 2013

An enhanced Q175 knock-in mouse model of Huntington disease with higher mutant huntingtin levels and accelerated disease phenotypes

Por Southwell, Amber L., Smith-Dijak, Amy, Kay, Chris, Sepers, Marja, Villanueva, Erika B., Parsons, Matthew P., Xie, Yuanyun, Anderson, Lisa, Felczak, Boguslaw, Waltl, Sabine, Ko, Seunghyun, Cheung, Daphne, Dal Cengio, Louisa, Slama, Ramy, Petoukhov, Eugenia, Raymond, Lynn A., Hayden, Michael R.
Publicado no Hum Mol Genet (2016)

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)