Treatment of a metabolic liver disease by in vivo genome base editing in adult mice L Villiger, HM Grisch-Chan, H Lindsay, F Ringnalda, CB Pogliano, ... Nature medicine 24 (10), 1519-1525, 2018 | 422 | 2018 |
Hepatocyte growth factor promotes cancer cell migration and angiogenic factors expression: a prognostic marker of human esophageal squamous cell carcinomas Y Ren, B Cao, S Law, Y Xie, PY Lee, L Cheung, Y Chen, X Huang, ... Clinical Cancer Research 11 (17), 6190-6197, 2005 | 197 | 2005 |
Upregulation of macrophage migration inhibitory factor contributes to induced N-Myc expression by the activation of ERK signaling pathway and increased expression of … Y Ren, HM Chan, Z Li, C Lin, J Nicholls, CF Chen, PY Lee, V Lui, ... Oncogene 23 (23), 4146-4154, 2004 | 135 | 2004 |
In vivo prime editing of a metabolic liver disease in mice D Böck, T Rothgangl, L Villiger, L Schmidheini, M Matsushita, N Mathis, ... Science translational medicine 14 (636), eabl9238, 2022 | 134 | 2022 |
Fusion-dependent formation of lipid nanoparticles containing macromolecular payloads JA Kulkarni, D Witzigmann, J Leung, R van der Meel, J Zaifman, ... Nanoscale 11 (18), 9023-9031, 2019 | 124 | 2019 |
Inhibition of tumor growth and metastasis in vitro and in vivo by targeting macrophage migration inhibitory factor in human neuroblastoma Y Ren, HM Chan, J Fan, Y Xie, YX Chen, W Li, GP Jiang, Q Liu, ... Oncogene 25 (25), 3501-3508, 2006 | 91 | 2006 |
Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy N Nair, MY Rincon, H Evens, S Sarcar, S Dastidar, E Samara-Kuko, ... Blood, The Journal of the American Society of Hematology 123 (20), 3195-3199, 2014 | 89 | 2014 |
Treatment of phenylketonuria using minicircle‐based naked‐DNA gene transfer to murine liver HM Viecelli, RP Harbottle, SP Wong, A Schlegel, MK Chuah, ... Hepatology 60 (3), 1035-1043, 2014 | 77 | 2014 |
State-of-the-art 2019 on gene therapy for phenylketonuria HM Grisch-Chan, G Schwank, CO Harding, B Thöny Human gene therapy 30 (10), 1274-1283, 2019 | 51 | 2019 |
Gene therapy for liver diseases using non-viral minicircle-DNA vector HM Viecelli, RP Harbottle, SP Wong, M Chuah, T VandenDriessche, ... Molecular Therapy 21, S136-S136, 2013 | 40 | 2013 |
Gene Therapy for Hepatic Diseases Using Non-Viral Minicircle-DNA Vector HM Viecelli, RP Harbottle, M Chuah, T VandenDriessch, CO Harding, ... Journal of Inherited Metabolic Disease 35, S144-S144, 2012 | 39 | 2012 |
Removing intensity effects and identifying significant genes for Affymetrix arrays in macrophage migration inhibitory factor-suppressed neuroblastoma cells J Fan, Y Chen, HM Chan, PKH Tam, Y Ren Proceedings of the National Academy of Sciences 102 (49), 17751-17756, 2005 | 35 | 2005 |
Pseudoexon exclusion by antisense therapy in 6‐pyruvoyl‐tetrahydropterin synthase deficiency S Brasil, HM Viecelli, D Meili, A Rassi, LR Desviat, B Pérez, M Ugarte, ... Human mutation 32 (9), 1019-1027, 2011 | 31 | 2011 |
Low-dose gene therapy for murine PKU using episomal naked DNA vectors expressing PAH from its endogenous liver promoter HM Grisch-Chan, A Schlegel, T Scherer, G Allegri, R Heidelberger, ... Molecular Therapy-Nucleic Acids 7, 339-349, 2017 | 29 | 2017 |
Involvement of visinin-like protein-1 (VSNL-1) in regulating proliferative and invasive properties of neuroblastoma Y Xie, H Chan, J Fan, Y Chen, J Young, W Li, X Miao, Z Yuan, H Wang, ... Carcinogenesis 28 (10), 2122-2130, 2007 | 26 | 2007 |
Functional Characterization of the spf/ash Splicing Variation in OTC Deficiency of Mice and Man A Rivera-Barahona, R Sánchez-Alcudia, HM Viecelli, V Rüfenacht, ... PLoS One 10 (4), e0122966, 2015 | 25 | 2015 |
A simple dried blood spot-method for in vivo measurement of ureagenesis by gas chromatography–mass spectrometry using stable isotopes G Allegri, S Deplazes, HM Grisch-Chan, D Mathis, R Fingerhut, J Häberle, ... Clinica Chimica Acta 464, 236-243, 2017 | 23 | 2017 |
Hepatocyte transfection in small pigs after weaning by hydrodynamic intraportal injection of naked DNA/minicircle vectors F Stoller, A Schlegel, HM Viecelli, V Rüfenacht, N Cesarovic, C Viecelli, ... Human gene therapy methods 26 (5), 181-192, 2015 | 20 | 2015 |
Treatment of cystathionine β-synthase deficiency in mice using a minicircle-based naked DNA vector HO Lee, L Gallego-Villar, HM Grisch-Chan, J Häberle, B Thöny, ... Human gene therapy 30 (9), 1093-1100, 2019 | 18 | 2019 |
Improvement of DNA vector delivery of DOTAP lipoplexes by short-chain aminolipids J Buck, D Mueller, U Mettal, M Ackermann, HM Grisch-Chan, B Thöny, ... ACS omega 5 (38), 24724-24732, 2020 | 17 | 2020 |