| Mitochondria and quality control defects in a mouse model of Gaucher disease—links to Parkinson’s disease LD Osellame, AA Rahim, IP Hargreaves, ME Gegg, A Richard-Londt, ... Cell metabolism 17 (6), 941-953, 2013 | 299 | 2013 |
| Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice M Themis, SN Waddington, M Schmidt, C Von Kalle, Y Wang, F Al-Allaf, ... Molecular Therapy 12 (4), 763-771, 2005 | 243 | 2005 |
| Physical parameters affecting ultrasound/microbubble-mediated gene delivery efficiency in vitro A Rahim, SL Taylor, NL Bush, GR Ter Haar, JC Bamber, CD Porter Ultrasound in medicine & biology 32 (8), 1269-1279, 2006 | 196 | 2006 |
| Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis SE Mole, G Anderson, HA Band, SF Berkovic, JD Cooper, SMK Holthaus, ... The Lancet Neurology 18 (1), 107-116, 2019 | 116 | 2019 |
| Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors AA Rahim, AMS Wong, SJ Howe, SMK Buckley, AD Acosta-Saltos, ... Gene therapy 16 (4), 509-520, 2009 | 106 | 2009 |
| Fetal gene therapy for neurodegenerative disease of infants G Massaro, CNZ Mattar, A Wong, E Sirka, SMK Buckley, BR Herbert, ... Nature medicine 24 (9), 1317-1323, 2018 | 103 | 2018 |
| Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system AA Rahim, AMS Wong, K Hoefer, SMK Buckley, CN Mattar, SH Cheng, ... The FASEB Journal 25 (10), 3505-3518, 2011 | 99 | 2011 |
| Nuclear localisation sequence templated nonviral gene delivery vectors: investigation of intracellular trafficking events of LMD and LD vector systems M Keller, RP Harbottle, E Perouzel, M Colin, I Shah, A Rahim, L Vaysse, ... Chembiochem 4 (4), 286-298, 2003 | 88 | 2003 |
| β-Glucosidase 2 (GBA2) activity and imino sugar pharmacology CM Ridley, KE Thur, J Shanahan, NB Thillaiappan, A Shen, K Uhl, ... Journal of Biological Chemistry 288 (36), 26052-26066, 2013 | 83 | 2013 |
| A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency J Tordo, C O’Leary, ASLM Antunes, N Palomar, P Aldrin-Kirk, M Basche, ... Brain 141 (7), 2014-2031, 2018 | 69 | 2018 |
| Spatial and acoustic pressure dependence of microbubble‐mediated gene delivery targeted using focused ultrasound AA Rahim, SL Taylor, NL Bush, GR ter Haar, JC Bamber, CD Porter The Journal of Gene Medicine: A cross‐disciplinary journal for research on …, 2006 | 68 | 2006 |
| Using stem cells to grow artificial tissue for peripheral nerve repair KS Bhangra, F Busuttil, JB Phillips, AA Rahim Stem cells international 2016, 2016 | 60 | 2016 |
| Targeted retroviral gene delivery using ultrasound SL Taylor, AA Rahim, NL Bush, JC Bamber, CD Porter The Journal of Gene Medicine: A cross‐disciplinary journal for research on …, 2007 | 60 | 2007 |
| Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems CN Mattar, SN Waddington, A Biswas, N Johana, XW Ng, AS Fisk, ... Gene therapy 20 (1), 69-83, 2013 | 58 | 2013 |
| Delineating pathological pathways in a chemically induced mouse model of Gaucher disease A Vardi, H Zigdon, A Meshcheriakova, AD Klein, C Yaacobi, R Eilam, ... The Journal of pathology 239 (4), 496-509, 2016 | 50 | 2016 |
| Current therapies for the soluble lysosomal forms of neuronal ceroid lipofuscinosis AMS Wong, AA Rahim, SN Waddington, JD Cooper Biochemical Society transactions 38 (6), 1484-1488, 2010 | 50 | 2010 |
| Epilepsy gene therapy using an engineered potassium channel A Snowball, E Chabrol, RC Wykes, T Shekh-Ahmad, JH Cornford, A Lieb, ... Journal of neuroscience 39 (16), 3159-3169, 2019 | 48 | 2019 |
| Influence of coagulation factor X on in vitro and in vivo gene delivery by adenovirus (Ad) 5, Ad35, and chimeric Ad5/Ad35 vectors JA Greig, SMK Buckley, SN Waddington, AL Parker, D Bhella, R Pink, ... Molecular Therapy 17 (10), 1683-1691, 2009 | 48 | 2009 |
| In vivo bioimaging with tissue-specific transcription factor activated luciferase reporters SMK Buckley, JMKM Delhove, DP Perocheau, R Karda, AA Rahim, ... Scientific reports 5 (1), 1-13, 2015 | 46 | 2015 |
| AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann–Pick type C1 disease MP Hughes, DA Smith, L Morris, C Fletcher, A Colaco, M Huebecker, ... Human molecular genetics 27 (17), 3079-3098, 2018 | 45 | 2018 |
