| Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an adeno-associated virus vector expressing human SMN C Hinderer, N Katz, EL Buza, C Dyer, T Goode, P Bell, LK Richman, ... Human gene therapy 29 (3), 285-298, 2018 | 669 | 2018 |
| The neurotropic properties of AAV-PHP. B are limited to C57BL/6J mice J Hordeaux, Q Wang, N Katz, EL Buza, P Bell, JM Wilson Molecular Therapy 26 (3), 664-668, 2018 | 375 | 2018 |
| Adeno-associated virus-induced dorsal root ganglion pathology J Hordeaux, EL Buza, C Dyer, T Goode, TW Mitchell, L Richman, ... Human gene therapy 31 (15-16), 808-818, 2020 | 176 | 2020 |
| Evaluation of intrathecal routes of administration for adeno-associated viral vectors in large animals C Hinderer, P Bell, N Katz, CH Vite, JP Louboutin, E Bote, H Yu, Y Zhu, ... Human gene therapy 29 (1), 15-24, 2018 | 135 | 2018 |
| Toxicology study of intra-cisterna magna adeno-associated virus 9 expressing human alpha-L-iduronidase in rhesus macaques J Hordeaux, C Hinderer, T Goode, N Katz, EL Buza, P Bell, R Calcedo, ... Molecular Therapy Methods & Clinical Development 10, 79-88, 2018 | 93 | 2018 |
| Self-reactive VH4-34–expressing IgG B cells recognize commensal bacteria JN Schickel, S Glauzy, YS Ng, N Chamberlain, C Massad, I Isnardi, ... Journal of Experimental Medicine 214 (7), 1991-2003, 2017 | 78 | 2017 |
| Delivery of an adeno-associated virus vector into cerebrospinal fluid attenuates central nervous system disease in mucopolysaccharidosis type II mice C Hinderer, N Katz, JP Louboutin, P Bell, H Yu, M Nayal, K Kozarsky, ... Human Gene Therapy 27 (11), 906-915, 2016 | 67 | 2016 |
| PTPN22 inhibition resets defective human central B cell tolerance JN Schickel, M Kuhny, A Baldo, JM Bannock, C Massad, H Wang, N Katz, ... Science immunology 1 (1), aaf7153-aaf7153, 2016 | 63 | 2016 |
| Rational design of organotin polyesters AF Baldwin, TD Huan, R Ma, A Mannodi-Kanakkithodi, M Tefferi, N Katz, ... Macromolecules 48 (8), 2422-2428, 2015 | 63 | 2015 |
| Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model C Hinderer, P Bell, JP Louboutin, N Katz, Y Zhu, G Lin, R Choa, J Bagel, ... Molecular genetics and metabolism 119 (1-2), 124-130, 2016 | 44 | 2016 |
| Exome-wide evaluation of rare coding variants using electronic health records identifies new gene–phenotype associations J Park, AM Lucas, X Zhang, K Chaudhary, JH Cho, G Nadkarni, A Dobbyn, ... Nature medicine 27 (1), 66-72, 2021 | 41 | 2021 |
| Translational feasibility of lumbar puncture for intrathecal AAV administration C Hinderer, N Katz, C Dyer, T Goode, J Johansson, P Bell, L Richman, ... Molecular Therapy Methods & Clinical Development 17, 969-974, 2020 | 33 | 2020 |
| A single injection of an optimized adeno-associated viral vector into cerebrospinal fluid corrects neurological disease in a murine model of GM1 gangliosidosis C Hinderer, B Nosratbakhsh, N Katz, JM Wilson Human Gene Therapy 31 (21-22), 1169-1177, 2020 | 24 | 2020 |
| Standardized method for intra-cisterna magna delivery under fluoroscopic guidance in nonhuman primates N Katz, T Goode, C Hinderer, J Hordeaux, JM Wilson Human Gene Therapy Methods 29 (5), 212-219, 2018 | 18 | 2018 |
| Manganese homeostasis: from rare single-gene disorders to complex phenotypes and diseases N Katz, DJ Rader The Journal of Clinical Investigation 129 (12), 5082-5085, 2019 | 16 | 2019 |
| Abnormal polyamine metabolism is unique to the neuropathic forms of MPS: potential for biomarker development and insight into pathogenesis C Hinderer, N Katz, JP Louboutin, P Bell, J Tolar, PJ Orchard, TC Lund, ... Human molecular genetics 26 (19), 3837-3849, 2017 | 11 | 2017 |
| Compositions useful in treatment of spinal muscular atrophy JM Wilson, C Hinderer, N Katz, Q Wang US Patent 11,578,341, 2023 | 2 | 2023 |
| Exome-by-phenome-wide rare variant gene burden association with electronic health record phenotypes J Park, N Katz, X Zhang, AM Lucas, A Verma, RL Judy, RL Kember, ... bioRxiv, 798330, 2019 | 2 | 2019 |
| Correction: Self-reactive VH4-34–expressing IgG B cells recognize commensal bacteria JN Schickel, S Glauzy, YS Ng, N Chamberlain, C Massad, I Isnardi, ... The Journal of Experimental Medicine 214 (7), 2161, 2017 | 1 | 2017 |
| Gene therapy for ceroid lipofuscinoses JM Wilson, N Katz, C Hinderer, J Hordeaux US Patent App. 18/148,077, 2023 | | 2023 |
