|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
|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
|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
|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
|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
|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
|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
|Fetal gene therapy for neurodegenerative disease of infants|
G Massaro, CNZ Mattar, AMS Wong, E Sirka, SMK Buckley, BR Herbert, ...
Nature medicine 24 (9), 1317-1323, 2018
|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
|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
|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
|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
|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
|Using stem cells to grow artificial tissue for peripheral nerve repair|
KS Bhangra, F Busuttil, JB Phillips, AA Rahim
Stem cells international 2016, 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
|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
|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
|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
|Luciferin detection after intranasal vector delivery is improved by intranasal rather than intraperitoneal luciferin administration|
SMK Buckley, SJ Howe, AA Rahim, H Buning, J McIntosh, SP Wong, ...
Human gene therapy 19 (10), 1050-1056, 2008
|Increased glucocerebrosidase (GBA) 2 activity in GBA1 deficient mice brains and in Gaucher leucocytes|
DG Burke, AA Rahim, SN Waddington, S Karlsson, I Enquist, K Bhatia, ...
Journal of Inherited Metabolic Disease: Official Journal of the Society for …, 2013