Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis SH Cheng, RJ Gregory, J Marshall, S Paul, DW Souza, GA White, ... Cell 63 (4), 827-834, 1990 | 2187 | 1990 |
Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis HB Kordasiewicz, LM Stanek, EV Wancewicz, C Mazur, MM McAlonis, ... Neuron 74 (6), 1031-1044, 2012 | 890 | 2012 |
Expression of cystic fibrosis transmembrane conductance regulator corrects defective chloride channel regulation in cystic fibrosis airway epithelial cells DP Rich, MP Anderson, RJ Gregory, SH Cheng, S Paul, DM Jefferson, ... Nature 347 (6291), 358-363, 1990 | 834 | 1990 |
Phosphorylation of the R domain by cAMP-dependent protein kinase regulates the CFTR chloride channel SH Cheng, DP Rich, J Marshall, RJ Gregory, MJ Welsh, AE Smith Cell 66 (5), 1027-1036, 1991 | 812 | 1991 |
Nomenclature for synthetic gene delivery systems PL Felgner, Y Barenholz, JP Behr, SH Cheng, P Cullis, L Huang, ... Human gene therapy 8 (5), 511-512, 1997 | 652 | 1997 |
Detailed analysis of structures and formulations of cationic lipids for efficient gene transfer to the lung ER Lee, J Marshall, CS Siegel, C Jiang, NS Yew, MR Nichols, ... Human gene therapy 7 (14), 1701-1717, 1996 | 628 | 1996 |
Tyrosine phosphorylation regulates the biochemical and biological properties of pp60c-src H Piwnica-Worms, KB Saunders, TM Roberts, AE Smith, SH Cheng Cell 49 (1), 75-82, 1987 | 608 | 1987 |
Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy MA Passini, J Bu, AM Richards, C Kinnecom, SP Sardi, LM Stanek, Y Hua, ... Science translational medicine 3 (72), 72ra18-72ra18, 2011 | 585 | 2011 |
Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial E Alton, M Stern, R Farley, A Jaffe, SL Chadwick, J Phillips, J Davies, ... The Lancet 353 (9157), 947-954, 1999 | 566 | 1999 |
Targeting nuclear RNA for in vivo correction of myotonic dystrophy TM Wheeler, AJ Leger, SK Pandey, AR MacLeod, M Nakamori, SH Cheng, ... Nature 488 (7409), 111-115, 2012 | 545 | 2012 |
Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial EWFW Alton, DK Armstrong, D Ashby, KJ Bayfield, D Bilton, ... The Lancet Respiratory Medicine 3 (9), 684-691, 2015 | 526 | 2015 |
Expression and characterization of the cystic fibrosis transmembrane conductance regulator RJ Gregory, SH Cheng, DP Rich, J Marshall, S Paul, K Hehir, ... Nature 347 (6291), 382-386, 1990 | 475 | 1990 |
Hypoxia-inducible factor-1 mediates activation of cultured vascular endothelial cells by inducing multiple angiogenic factors M Yamakawa, LX Liu, T Date, AJ Belanger, KA Vincent, GY Akita, ... Circulation research 93 (7), 664-673, 2003 | 470 | 2003 |
A novel cationic lipid greatly enhances plasmid DNA delivery and expression in mouse lung. CJ Wheeler, PL Felgner, YJ Tsai, J Marshall, L Sukhu, SG Doh, J Hartikka, ... Proceedings of the National Academy of Sciences 93 (21), 11454-11459, 1996 | 400 | 1996 |
CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression SC Hyde, IA Pringle, S Abdullah, AE Lawton, LA Davies, A Varathalingam, ... Nature biotechnology 26 (5), 549-551, 2008 | 376 | 2008 |
Acid β‐glucosidase mutants linked to Gaucher disease, Parkinson disease, and Lewy body dementia alter α‐synuclein processing V Cullen, SP Sardi, J Ng, YH Xu, Y Sun, JJ Tomlinson, P Kolodziej, I Kahn, ... Annals of neurology 69 (6), 940-953, 2011 | 374 | 2011 |
Biophysical characterization of cationic lipid: DNA complexes SJ Eastman, C Siegel, J Tousignant, AE Smith, SH Cheng, RK Scheule Biochimica et Biophysica Acta (BBA)-Biomembranes 1325 (1), 41-62, 1997 | 356 | 1997 |
Improved cationic lipid formulations for in vivo gene therapy. PL Felgner, YJ Tsai, L Sukhu, CJ Wheeler, M Manthorpe, J Marshall, ... Annals of the New York Academy of Sciences 772, 126-139, 1995 | 356 | 1995 |
CNS expression of glucocerebrosidase corrects α-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy SP Sardi, J Clarke, C Kinnecom, TJ Tamsett, L Li, LM Stanek, MA Passini, ... Proceedings of the National Academy of Sciences 108 (29), 12101-12106, 2011 | 353 | 2011 |
CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy MA Passini, J Bu, EM Roskelley, AM Richards, SP Sardi, CR O’Riordan, ... The Journal of clinical investigation 120 (4), 1253-1264, 2010 | 349 | 2010 |