|Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene/allele-specific silencing of mutant huntingtin|
JB Carroll, SC Warby, AL Southwell, CN Doty, S Greenlee, N Skotte, ...
Molecular Therapy 19 (12), 2178-2185, 2011
|Intrabody gene therapy ameliorates motor, cognitive, and neuropathological symptoms in multiple mouse models of Huntington's disease|
AL Southwell, J Ko, PH Patterson
Journal of Neuroscience 29 (43), 13589-13602, 2009
|GABA transporter deficiency causes tremor, ataxia, nervousness, and increased GABA-induced tonic conductance in cerebellum|
CS Chiu, S Brickley, K Jensen, A Southwell, S Mckinney, S Cull-Candy, ...
Journal of Neuroscience 25 (12), 3234-3245, 2005
|Atypical expansion in mice of the sensory neuron-specific Mrg G protein-coupled receptor family|
MJ Zylka, X Dong, AL Southwell, DJ Anderson
Proceedings of the National Academy of Sciences 100 (17), 10043-10048, 2003
|Assessment of motor balance and coordination in mice using the balance beam|
TN Luong, HJ Carlisle, A Southwell, PH Patterson
JoVE (Journal of Visualized Experiments), e2376, 2011
|Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS|
ME Østergaard, AL Southwell, H Kordasiewicz, AT Watt, NH Skotte, ...
Nucleic acids research 41 (21), 9634-9650, 2013
|Marked differences in neurochemistry and aggregates despite similar behavioural and neuropathological features of Huntington disease in the full-length BACHD and YAC128 mice|
MA Pouladi, LM Stanek, Y Xie, S Franciosi, AL Southwell, Y Deng, ...
Human molecular genetics 21 (10), 2219-2232, 2012
|Evolution and divergence of sodium channel genes in vertebrates|
GF Lopreato, Y Lu, A Southwell, NS Atkinson, DM Hillis, TP Wilcox, ...
Proceedings of the National Academy of Sciences 98 (13), 7588-7592, 2001
|Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases|
AL Southwell, NH Skotte, CF Bennett, MR Hayden
Trends in molecular medicine 18 (11), 634-643, 2012
|Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients|
NH Skotte, AL Southwell, ME Østergaard, JB Carroll, SC Warby, CN Doty, ...
PloS one 9 (9), e107434, 2014
|Intrabodies binding the proline-rich domains of mutant huntingtin increase its turnover and reduce neurotoxicity|
AL Southwell, A Khoshnan, DE Dunn, CW Bugg, DC Lo, PH Patterson
Journal of Neuroscience 28 (36), 9013-9020, 2008
|In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides|
AL Southwell, NH Skotte, HB Kordasiewicz, ME Østergaard, AT Watt, ...
Molecular Therapy 22 (12), 2093-2106, 2014
|Huntingtin haplotypes provide prioritized target panels for allele-specific silencing in Huntington disease patients of European ancestry|
C Kay, JA Collins, NH Skotte, AL Southwell, SC Warby, NS Caron, ...
Molecular Therapy 23 (11), 1759-1771, 2015
|A fully humanized transgenic mouse model of Huntington disease|
AL Southwell, SC Warby, JB Carroll, CN Doty, NH Skotte, W Zhang, ...
Human molecular genetics 22 (1), 18-34, 2012
|HD iPSC-derived neural progenitors accumulate in culture and are susceptible to BDNF withdrawal due to glutamate toxicity|
VB Mattis, C Tom, S Akimov, J Saeedian, ME Østergaard, AL Southwell, ...
Human molecular genetics 24 (11), 3257-3271, 2015
|HACE1 reduces oxidative stress and mutant Huntingtin toxicity by promoting the NRF2 response|
B Rotblat, AL Southwell, DE Ehrnhoefer, NH Skotte, M Metzler, ...
Proceedings of the National Academy of Sciences 111 (8), 3032-3037, 2014
|Personalized gene silencing therapeutics for Huntington disease|
C Kay, NH Skotte, AL Southwell, MR Hayden
Clinical genetics 86 (1), 29-36, 2014
|Anti-semaphorin 4D immunotherapy ameliorates neuropathology and some cognitive impairment in the YAC128 mouse model of Huntington disease|
AL Southwell, S Franciosi, EB Villanueva, Y Xie, LA Winter, ...
Neurobiology of disease 76, 46-56, 2015
|Hip14l-deficient mice develop neuropathological and behavioural features of Huntington disease|
LM Sutton, SS Sanders, SL Butland, RR Singaraja, S Franciosi, ...
Human molecular genetics 22 (3), 452-465, 2012
|Ultrasensitive measurement of huntingtin protein in cerebrospinal fluid demonstrates increase with Huntington disease stage and decrease following brain huntingtin suppression|
AL Southwell, SEP Smith, TR Davis, NS Caron, EB Villanueva, Y Xie, ...
Scientific reports 5, 12166, 2015