| Comprehensive genetic testing in the clinical evaluation of 1119 patients with hearing loss CM Sloan-Heggen, AO Bierer, AE Shearer, DL Kolbe, CJ Nishimura, ... Human genetics 135, 441-450, 2016 | 491 | 2016 |
| RNA interference prevents autosomal-dominant hearing loss SB Shibata, PT Ranum, H Moteki, B Pan, AT Goodwin, SS Goodman, ... The American Journal of Human Genetics 98 (6), 1101-1113, 2016 | 118 | 2016 |
| Insights into the biology of hearing and deafness revealed by single-cell RNA sequencing PT Ranum, AT Goodwin, H Yoshimura, DL Kolbe, WD Walls, JY Koh, ... Cell reports 26 (11), 3160-3171. e3, 2019 | 103 | 2019 |
| Enhanced viral-mediated cochlear gene delivery in adult mice by combining canal fenestration with round window membrane inoculation H Yoshimura, SB Shibata, PT Ranum, RJH Smith Scientific reports 8 (1), 2980, 2018 | 98 | 2018 |
| CDKL5 regulates flagellar length and localizes to the base of the flagella in Chlamydomonas LW Tam, PT Ranum, PA Lefebvre Molecular biology of the cell 24 (5), 588-600, 2013 | 80 | 2013 |
| Nonsyndromic Hearing Loss and Deafness, DFNA3–RETIRED CHAPTER, FOR HISTORICAL REFERENCE ONLY RJH Smith, PT Ranum | 75 | 2016 |
| Regulated control of gene therapies by drug-induced splicing AM Monteys, AA Hundley, PT Ranum, L Tecedor, A Muehlmatt, E Lim, ... Nature 596 (7871), 291-295, 2021 | 73 | 2021 |
| Targeted allele suppression prevents progressive hearing loss in the mature murine model of human TMC1 deafness H Yoshimura, SB Shibata, PT Ranum, H Moteki, RJH Smith Molecular therapy 27 (3), 681-690, 2019 | 72 | 2019 |
| HOMER2, a stereociliary scaffolding protein, is essential for normal hearing in humans and mice H Azaiez, AR Decker, KT Booth, AC Simpson, AE Shearer, PLM Huygen, ... PLoS genetics 11 (3), e1005137, 2015 | 63 | 2015 |
| 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 | 39 | 2021 |
| Intravenous rAAV2/9 injection for murine cochlear gene delivery SB Shibata, H Yoshimura, PT Ranum, AT Goodwin, RJH Smith Scientific reports 7 (1), 9609, 2017 | 34 | 2017 |
| New lipid‐producing, cold‐tolerant yellow‐green alga isolated from the rocky mountains of colorado DR Nelson, S Mengistu, P Ranum, G Celio, M Mashek, D Mashek, ... Biotechnology progress 29 (4), 853-861, 2013 | 20 | 2013 |
| 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 | 8 | 2023 |
| Immortalized striatal precursor neurons from Huntington’s disease patient-derived iPS cells as a platform for target identification and screening for experimental therapeutics SS Akimov, M Jiang, AJ Kedaigle, N Arbez, LO Marque, CR Eddings, ... Human molecular genetics 30 (24), 2469-2487, 2021 | 8 | 2021 |
| Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching JM Dawicki-McKenna, AJ Felix, EA Waxman, C Cheng, DA Amado, ... Nature Communications 14 (1), 2628, 2023 | 7 | 2023 |
| Mutation-agnostic RNA interference with engineered replacement rescues Tmc1-related hearing loss Y Iwasa, MJ Klimara, H Yoshimura, WD Walls, R Omichi, CA West, ... Life Science Alliance 6 (3), 2023 | 5 | 2023 |
| 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, ... bioRxiv, 2023.08. 14.552850, 2023 | 4 | 2023 |
| 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 | 3 | 2023 |
| Mapping PTBP splicing in human brain identifies targets for therapeutic splice switching including SYNGAP1 JM Dawicki-McKenna, AJ Felix, EA Waxman, C Cheng, DA Amado, ... bioRxiv, 2022.07. 15.500219, 2022 | 1 | 2022 |
| A Manual Technique for Isolation and Single-Cell RNA Sequencing Analysis of Cochlear Hair Cells and Supporting Cells C West, PT Ranum, R Omichi, Y Iwasa, MJ Kilmara, D Walls, JY Koh, ... Developmental, Physiological, and Functional Neurobiology of the Inner Ear …, 2022 | 1 | 2022 |
