| A comprehensive review: sphingolipid metabolism and implications of disruption in sphingolipid homeostasis BM Quinville, NM Deschenes, AE Ryckman, JS Walia International journal of molecular sciences 22 (11), 5793, 2021 | 134 | 2021 |
| PARP inhibitor drugs in the treatment of breast, ovarian, prostate and pancreatic cancers: an update of clinical trials D Kamel, C Gray, JS Walia, V Kumar Current drug targets 19 (1), 21-37, 2018 | 124 | 2018 |
| Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors M Yoshimitsu, T Sato, K Tao, JS Walia, VI Rasaiah, GT Sleep, GJ Murray, ... Proceedings of the National Academy of Sciences 101 (48), 16909-16914, 2004 | 121 | 2004 |
| Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates JS Walia, N Altaleb, A Bello, C Kruck, MC LaFave, GK Varshney, ... Molecular Therapy 23 (3), 414-422, 2015 | 89 | 2015 |
| Glutaminase Deficiency Caused by Short Tandem Repeat Expansion in GLS ABP van Kuilenburg, M Tarailo-Graovac, PA Richmond, BI Drögemöller, ... New England Journal of Medicine 380 (15), 1433-1441, 2019 | 83 | 2019 |
| Novel vector design and hexosaminidase variant enabling self-complementary adeno-associated virus for the treatment of Tay-Sachs disease S Karumuthil-Melethil, S Nagabhushan Kalburgi, P Thompson, M Tropak, ... Human gene therapy 27 (7), 509-521, 2016 | 68 | 2016 |
| Experiences of caregivers of children with inherited metabolic diseases: a qualitative study S Siddiq, BJ Wilson, ID Graham, M Lamoureux, SD Khangura, K Tingley, ... Orphanet journal of rare diseases 11, 1-10, 2016 | 66 | 2016 |
| Construction of a hybrid β-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo MB Tropak, S Yonekawa, S Karumuthil-Melethil, P Thompson, ... Molecular Therapy Methods & Clinical Development 3, 2016 | 57 | 2016 |
| Mobile element insertion detection in 89,874 clinical exomes RI Torene, K Galens, S Liu, K Arvai, C Borroto, J Scuffins, Z Zhang, ... Genetics in Medicine 22 (5), 974-978, 2020 | 53 | 2020 |
| Metabolism of glycosphingolipids and their role in the pathophysiology of lysosomal storage disorders AE Ryckman, I Brockhausen, JS Walia International Journal of Molecular Sciences 21 (18), 6881, 2020 | 46 | 2020 |
| Autologous transplantation of Lentivector/acid ceramidase–transduced hematopoietic cells in nonhuman Primates JS Walia, A Neschadim, O Lopez-Perez, A Alayoubi, X Fan, S Carpentier, ... Human gene therapy 22 (6), 679-687, 2011 | 42 | 2011 |
| De novo and bi-allelic pathogenic variants in NARS1 cause neurodevelopmental delay due to toxic gain-of-function and partial loss-of-function effects A Manole, S Efthymiou, E O’Connor, MI Mendes, M Jennings, ... The American Journal of Human Genetics 107 (2), 311-324, 2020 | 41 | 2020 |
| Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice KJL Osmon, E Woodley, P Thompson, K Ong, S Karumuthil-Melethil, ... Human gene therapy 27 (7), 497-508, 2016 | 40 | 2016 |
| IL‐12 immunotherapy of murine leukaemia: comparison of systemic versus gene modified cell therapy A Labbe, M Nelles, J Walia, L Jia, C Furlonger, T Nonaka, JA Medin, ... Journal of cellular and molecular medicine 13 (8b), 1962-1976, 2009 | 39 | 2009 |
| Relaxin-3 and receptors in the human and rhesus brain and reproductive tissues JD Silvertown, A Neschadim, HN Liu, P Shannon, JS Walia, JCH Kao, ... Regulatory peptides 159 (1-3), 44-53, 2010 | 36 | 2010 |
| Tumor protection following vaccination with low doses of lentivirally transduced DCs expressing the self-antigen erbB2 ME Mossoba, JS Walia, VI Rasaiah, N Buxhoeveden, R Head, C Ying, ... Molecular Therapy 16 (3), 607-617, 2008 | 33 | 2008 |
| Battery of behavioral tests assessing general locomotion, muscular strength, and coordination in mice KJL Osmon, M Vyas, E Woodley, P Thompson, JS Walia JoVE (Journal of Visualized Experiments), e55491, 2018 | 28 | 2018 |
| Functional expression of mouse relaxin and mouse relaxin-3 in the lung from an Ebola virus glycoprotein-pseudotyped lentivirus via tracheal delivery JD Silvertown, JS Walia, AJ Summerlee, JA Medin Endocrinology 147 (8), 3797-3808, 2006 | 28 | 2006 |
| Familial PDGFRA-mutation syndrome: somatic and gastrointestinal phenotype PN Manley, S Abu-Abed, R Kirsch, A Hawrysh, N Perrier, H Feilotter, ... Human Pathology 76, 52-57, 2018 | 26 | 2018 |
| Clinical spectrum of KIAA2022 pathogenic variants in males: Case report of two boys with KIAA2022 pathogenic variants and review of the literature M Lorenzo, I Stolte‐Dijkstra, P van Rheenen, RG Smith, T Scheers, ... American Journal of Medical Genetics Part A 176 (6), 1455-1462, 2018 | 26 | 2018 |
