| Single-dose gene-replacement therapy for spinal muscular atrophy JR Mendell, S Al-Zaidy, R Shell, WD Arnold, LR Rodino-Klapac, TW Prior, ... New England Journal of Medicine 377 (18), 1713-1722, 2017 | 2063 | 2017 |
| Astrocytes from familial and sporadic ALS patients are toxic to motor neurons AM Haidet-Phillips, ME Hester, CJ Miranda, K Meyer, L Braun, A Frakes, ... Nature biotechnology 29 (9), 824-828, 2011 | 898 | 2011 |
| Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS K Meyer, L Ferraiuolo, CJ Miranda, S Likhite, S McElroy, S Renusch, ... Proceedings of the National Academy of Sciences 111 (2), 829-832, 2014 | 384 | 2014 |
| Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose–response study in mice and nonhuman primates K Meyer, L Ferraiuolo, L Schmelzer, L Braun, V McGovern, S Likhite, ... Molecular Therapy 23 (3), 477-487, 2015 | 301 | 2015 |
| Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS KD Foust, DL Salazar, S Likhite, L Ferraiuolo, D Ditsworth, H Ilieva, ... Molecular Therapy 21 (12), 2148-2159, 2013 | 247 | 2013 |
| Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism L Ferraiuolo, K Meyer, TW Sherwood, J Vick, S Likhite, A Frakes, ... Proceedings of the National Academy of Sciences 113 (42), E6496-E6505, 2016 | 193 | 2016 |
| Delayed disease onset and extended survival in the SOD1G93A rat model of amyotrophic lateral sclerosis after suppression of mutant SOD1 in the motor cortex GM Thomsen, G Gowing, J Latter, M Chen, JP Vit, K Staggenborg, ... Journal of Neuroscience 34 (47), 15587-15600, 2014 | 155 | 2014 |
| Major histocompatibility complex class I molecules protect motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis SW Song, CJ Miranda, L Braun, K Meyer, AE Frakes, L Ferraiuolo, ... Nature medicine 22 (4), 397-403, 2016 | 140 | 2016 |
| Translating SOD1 gene silencing toward the clinic: a highly efficacious, off-target-free, and biomarker-supported strategy for fALS T Iannitti, JM Scarrott, S Likhite, IRP Coldicott, KE Lewis, PR Heath, ... Molecular Therapy-Nucleic Acids 12, 75-88, 2018 | 51 | 2018 |
| Gene therapy corrects brain and behavioral pathologies in CLN6-Batten disease JT Cain, S Likhite, KA White, DJ Timm, SS Davis, TB Johnson, ... Molecular Therapy 27 (10), 1836-1847, 2019 | 45 | 2019 |
| AAV9 gene therapy increases lifespan and treats pathological and behavioral abnormalities in a mouse model of CLN8-batten disease TB Johnson, KA White, JJ Brudvig, JT Cain, L Langin, MA Pratt, CD Booth, ... Molecular Therapy 29 (1), 162-175, 2021 | 28 | 2021 |
| Optimization and validation of CAR transduction into human primary NK cells using CRISPR and AAV MN Kararoudi, S Likhite, E Elmas, K Yamamoto, M Schwartz, K Sorathia, ... Cell Reports Methods 2 (6), 2022 | 25 | 2022 |
| 480. Gene therapy for spinal muscular atrophy type 1 shows potential to improve survival and motor functional outcomes JR Mendell, S Al-Zaidy, R Shell, WD Arnold, L Rodino-Klapac, JT Kissel, ... Molecular Therapy 24, S190, 2016 | 23 | 2016 |
| Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice KA White, HR Nelvagal, TA Poole, B Lu, TB Johnson, S Davis, MA Pratt, ... Molecular Therapy-Methods & Clinical Development 20, 497-507, 2021 | 20 | 2021 |
| Neurotoxic astrocytes directly converted from sporadic and familial ALS patient fibroblasts reveal signature diversities and miR-146a theragnostic potential in specific subtypes C Gomes, C Sequeira, S Likhite, CN Dennys, SJ Kolb, PJ Shaw, AR Vaz, ... Cells 11 (7), 1186, 2022 | 19 | 2022 |
| CD33 targeting primary CAR-NK cells generated by CRISPR mediated gene insertion show enhanced anti-AML activity MN Kararoudi, S Likhite, E Elmas, M Schwartz, K Sorathia, K Yamamoto, ... Blood 136, 3, 2020 | 16 | 2020 |
| AAV9-MCT8 delivery at juvenile stage ameliorates neurological and behavioral deficits in a mouse model of MCT8-deficiency XH Liao, P Avalos, O Shelest, R Ofan, M Shilo, C Bresee, S Likhite, JP Vit, ... Thyroid 32 (7), 849-859, 2022 | 15 | 2022 |
| Onasemnogene abeparvovec-xioi: a gene replacement strategy for the treatment of infants diagnosed with spinal muscular atrophy M Schwartz, S Likhite, K Meyer Drugs Today 57 (6), 387, 2021 | 14 | 2021 |
| AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: patients treated early with the proposed therapeutic dose were able to sit unassisted at a younger age L Lowes, S Al-Zaidy, R Shell, W Arnold, L Rodino-Klapac, T Prior, ... Neuromuscular Disorders 27, S208-S209, 2017 | 12 | 2017 |
| CRISPR-targeted CAR gene insertion using Cas9/RNP and AAV6 enhances anti-AML activity of primary NK cells MN Kararoudi, S Likhite, E Elmas, K Yamamoto, M Schwartz, K Sorathia, ... bioRxiv, 2021.03. 17.435886, 2021 | 10 | 2021 |
