CRISPR/Cas9 editing of the mutant huntingtin allele in vitro and in vivo AM Monteys, SA Ebanks, MS Keiser, BL Davidson Molecular therapy 25 (1), 12-23, 2017 | 342 | 2017 |
Viral vectors for gene transfer YH Chen, MS Keiser, BL Davidson Current protocols in mouse biology 8 (4), e58, 2018 | 177 | 2018 |
Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia MS Keiser, HB Kordasiewicz, JL McBride Human molecular genetics 25 (R1), R53-R64, 2016 | 122 | 2016 |
Broad therapeutic benefit after RNAi expression vector delivery to deep cerebellar nuclei: implications for spinocerebellar ataxia type 1 therapy MS Keiser, RL Boudreau, BL Davidson Molecular Therapy 22 (3), 588-595, 2014 | 88 | 2014 |
RNAi prevents and reverses phenotypes induced by mutant human ataxin‐1 MS Keiser, AM Monteys, R Corbau, P Gonzalez‐Alegre, BL Davidson Annals of neurology 80 (5), 754-765, 2016 | 68 | 2016 |
RNAi or overexpression: alternative therapies for Spinocerebellar Ataxia Type 1 MS Keiser, JC Geoghegan, RL Boudreau, KA Lennox, BL Davidson Neurobiology of disease 56, 6-13, 2013 | 60 | 2013 |
Broad distribution of ataxin 1 silencing in rhesus cerebella for spinocerebellar ataxia type 1 therapy MS Keiser, JH Kordower, P Gonzalez-Alegre, BL Davidson Brain 138 (12), 3555-3566, 2015 | 54 | 2015 |
The long non-coding RNA NEAT1 is elevated in polyglutamine repeat expansion diseases and protects from disease gene-dependent toxicities C Cheng, RM Spengler, MS Keiser, AM Monteys, JM Rieders, ... Human molecular genetics 27 (24), 4303-4314, 2018 | 53 | 2018 |
Altered Purkinje cell miRNA expression and SCA1 pathogenesis E Rodriguez-Lebron, G Liu, M Keiser, MA Behlke, BL Davidson Neurobiology of disease 54, 456-463, 2013 | 53 | 2013 |
Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brain MS Keiser, PT Ranum, CM Yrigollen, EM Carrell, GR Smith, AL Muehlmatt, ... Nature medicine 27 (11), 1982-1989, 2021 | 51 | 2021 |
Adeno‐Associated Virus Production, Purification, and Titering YH Chen, MS Keiser, BL Davidson Current protocols in mouse biology 8 (4), e56, 2018 | 30 | 2018 |
Recent advances in RNA interference therapeutics for CNS diseases PS Ramachandran, MS Keiser, BL Davidson Neurotherapeutics 10 (3), 473-485, 2013 | 28 | 2013 |
RNA interference of human α-synuclein in mouse YC Kim, A Miller, LCRF Lins, SW Han, MS Keiser, RL Boudreau, ... Frontiers in neurology 8, 13, 2017 | 26 | 2017 |
APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease RJ Jackson, MS Keiser, JC Meltzer, DP Fykstra, SE Dierksmeier, ... Molecular Therapy 32 (5), 1373-1386, 2024 | 21 | 2024 |
Techniques for intracranial stereotaxic injections of Adeno‐Associated viral vectors in adult mice MS Keiser, YH Chen, BL Davidson Current protocols in mouse biology 8 (4), e57, 2018 | 18 | 2018 |
Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates PT Ranum, L Tecedor, MS Keiser, YH Chen, DE Leib, X Liu, BL Davidson Molecular Therapy 31 (3), 609-612, 2023 | 15 | 2023 |
Combined overexpression of ATXN1L and mutant ATXN1 knockdown by AAV rescue motor phenotypes and gene signatures in SCA1 mice EM Carrell, MS Keiser, AB Robbins, BL Davidson Molecular Therapy-Methods & Clinical Development 25, 333-343, 2022 | 12 | 2022 |
Haplotyping SNPs for allele-specific gene editing of the expanded huntingtin allele using long-read sequencing L Fang, AM Monteys, A Dürr, M Keiser, C Cheng, A Harapanahalli, ... Human Genetics and Genomics Advances 4 (1), 2023 | 11 | 2023 |
Design and validation of a multi-point injection technology for MR-guided convection enhanced delivery in the brain K Prezelski, M Keiser, JM Stein, TH Lucas, B Davidson, ... Frontiers in medical technology 3, 725844, 2021 | 11 | 2021 |
VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSF EM Carrell, YH Chen, PT Ranum, SL Coffin, LN Singh, L Tecedor, ... Molecular Therapy-Nucleic Acids 33, 296-304, 2023 | 5 | 2023 |