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

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