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Perinatal systemic gene delivery using adeno-associated viral vectors. Journal article ; 2014 ; Front Mol Neurosci ; Switzerland links: IRIS ; UCL Discovery ; doi ; author's authors [click to collapse] : Karda R,Buckley SM,Mattar CN,Ng J,Massaro G,Hughes MP,Kurian MA,Baruteau J,Gissen P,Chan JK,Bacchelli C,Waddington SN,Rahim AA
Somatotransgenic Bioimaging: Modelling Disease Progression or Therapeutic Amelioration By Continued Tissue-Specific Bioluminescence Imaging in Living Small Animals Conference ; 2014 ; MOLECULAR THERAPY links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Buckley SM,Delhove JM,Karda R,Perocheau D,Rahim AA,Waddington SN,McKay TR
Analysis of transduction profile of AAV2/9 tyrosine hydroxylase promoter in the neonatal mouse brain Conference ; 2014 ; HUMAN GENE THERAPY links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Ng J,Rahim A,Karda R,Buckley S,Baruteau J,Perocheau D,Kurian M,Waddington S
Elongation factor 1 alpha as a potential promoter for gene therapy in a murine model of both urea cycle defects and systemic disease: argininosuccinic aciduria Conference ; 2014 ; HUMAN GENE THERAPY links: IRIS ; author's authors [click to collapse] : Baruteau J,Hanley J,Buckley SM,Gissen P,Howe SJ,Karda R,Mills PB,Ng J,Pechoreau D,Rahim AA,Waddington SN
Somatic Transgenesis of the Central Nervous System for Disease Modelling and Treatment Journal article ; 2014 ; REPRODUCTIVE SCIENCES links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Karda R,Delhove JMKM,Buckley SMK,Ward NJ,Rahim AA,Herbert B,Mckay TR,Hagberg HE,Waddington SN,Johnson MR
The Role of Infection and the TNF Family of Cytokines in Neonatal Hypoxic-Ischemic Brain Injury. Journal article ; 2014 ; REPRODUCTIVE SCIENCES links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Rocha-Ferreira E,Francesch-Domenech E,Thei L,Rahim A,Lange S,Peebles D,Hristova M,Raivich G
Somatic transgenesis of the central nervous system for disease modelling and treatment Conference ; 2013 ; HUMAN GENE THERAPY links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Karda R,Delhove JMKM,Buckley SMK,Ward NJ,Rahim AA,Herbert B,Mckay TR,Hagberg HE,Waddington SN,Johnson MR
Lentiviral vectors for gene therapy of type I Gaucher disease Conference ; 2013 ; HUMAN GENE THERAPY links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Aitchison KL,Rahim AA,Reed M,Burke DG,Hughes DA,Heales S,Kinnon C,Waddington SN,Howe SJ
In utero gene therapy rescue of an acute neonatal lethal mouse model of neuropathic Gaucher Disease Conference ; 2013 ; HUMAN GENE THERAPY links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Rahim AA,Sirka E,Burke DG,Buckley SM,Cullen E,Mukherji S,Herbert B,Aitchison KL,Karlsson S,Hughes DA,Mehta AB,Howe SJ,Heales S,Mills K,Cooper JD,Cheng SH,Waddington SN
β-Glucosidase 2 (GBA2) activity and imino sugar pharmacology. Journal article ; 2013 ; J Biol Chem ; United States links: IRIS ; UCL Discovery ; doi ; author's authors [click to collapse] : Ridley CM,Thur KE,Shanahan J,Thillaiappan NB,Shen A,Uhl K,Walden CM,Rahim AA,Waddington SN,Platt FM,van der Spoel AC
Developing lentiviral vectors for gene thrapy of type I Gaucher disease Conference ; 2013 ; HUMAN GENE THERAPY links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Aitchison K,Rahim A,Reed M,Hughes D,Kinnon C,Waddington S
Mitochondria and quality control defects in a mouse model of Gaucher disease - links to Parkinson’s Disease Journal article ; 2013 ; Cell Metabolism links: IRIS ; UCL Discovery ; doi authors [click to collapse] : Osellame LD,Rahim AA,Hargreaves IP,Gegg ME,Richard-Londt A,Brandner S,Waddington SN,Schapira AHV,Duchen MR
Erratum to: Increased glucocerebrosidase (GBA) 2 activity in GBA1 deficient mice brains and in Gaucher leucocytes. Journal article ; 2013 ; J Inherit Metab Dis ; Netherlands links: IRIS ; UCL Discovery ; doi ; author's authors [click to collapse] : Burke DG,Rahim AA,Waddington SN,Karlsson S,Enquist I,Bhatia K,Mehta A,Vellodi A,Heales S
Increased glucocerebrosidase (GBA) 2 activity in GBA1 deficient mice brains and in Gaucher leucocytes. Journal article ; 2013 ; J Inherit Metab Dis ; Netherlands links: IRIS ; UCL Discovery ; doi ; author's authors [click to collapse] : Burke DG,Rahim AA,Waddington SN,Karlsson S,Enquist I,Bhatia K,Mehta A,Vellodi A,Heales S
Accumulation of damaged mitochondria in neuropathic Gaucher disease, the most prevalent risk factor for Parkinson's disease, is due to defective cellular degradation machinery Conference ; 2012 ; BIOCHIMICA ET BIOPHYSICA ACTA-BIOENERGETICS links: IRIS ; UCL Discovery ; doi ; author's authors [click to collapse] : Osellame LD,Rahim A,Gegg ME,Waddington S,Schapira AHV,Duchen MR
Codon optimised FVIII constructs for treatment of haemophilia A Conference ; 2012 ; HUMAN GENE THERAPY links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Ward N,Buckley S,Rahim A,McVey J,Thrasher A,Waddington S
Intravenous administration of AAV2/9 to fetal and neonatal mice leads to extensive transduction of visceral organs and intestinal epithelia Conference ; 2012 ; HUMAN GENE THERAPY links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Rahim AA,Wong AM,Hofer K,Buckley SM,Mukherji S,Cooper JD,Waddington SN
Developing lentiviral vectors for gene therapy of Type I Gaucher Disease Conference ; 2012 ; HUMAN GENE THERAPY links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Aitchison KL,Rahim AA,Hughes D,Kinnon C,Waddington SN,Howe SJ
Optimised Factor VIII Vectors for Gene Therapy of Haemophilia A Conference ; 2012 ; MOLECULAR THERAPY links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Ward NJ,Buckley SMK,Rahim AA,McVey JH,Waddington SN
Systemic Gene Expression after Intravenous Injection of Adeno-Associated Virus 2/9 to Fetal and Neonatal Mice Conference ; 2012 ; MOLECULAR THERAPY links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Rahim AA,Wong AM,Hoefer K,Buckley SM,Mattar CN,Chan JK,Cooper JD,Waddington SN
Choice of surrogate and physiological markers for prenatal gene therapy. Journal article ; 2012 ; Methods Mol Biol ; United States links: IRIS ; UCL Discovery ; doi ; author's authors [click to collapse] : Delhove JM,Rahim AA,McKay TR,Waddington SN,Buckley SM
Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems. Journal article ; 2013 ; Gene Ther ; England links: IRIS ; UCL Discovery ; doi ; author's authors [click to collapse] : Mattar CN,Waddington SN,Biswas A,Johana N,Ng XW,Fisk AS,Fisk NM,Tan LG,Rahim AA,Buckley SM,Tan MH,Lu J,Choolani M,Chan JK
Optimised vectors for the treatment of haemophilia A Journal article ; 2011 ; HUMAN GENE THERAPY ; MARY ANN LIEBERT INC links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Ward NJ,Buckley SMK,Rahim AA,McVey JH,Thrasher AJ,Waddington SN
Systemic gene expression following intravenous administration of AAV2/9 to fetal and neonatal mice and non-human primates Journal article ; 2011 ; HUMAN GENE THERAPY ; MARY ANN LIEBERT INC links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Rahim AA,Wong AMS,Hoefer K,Buckley SMK,Mattar CN,Chan JKY,Cooper JD,Waddington SN
Viral expression of GM-CSF: neuroinflammatory response and effects on regeneration Journal article ; 2011 ; HUMAN GENE THERAPY ; MARY ANN LIEBERT INC links: IRIS ; UCL Discovery ; author's authors [click to collapse] : Acosta-Saltos C,Gonitel R,Chavda S,Acosta-Saltos A,Rahim A,Thrasher A,Anderson P,Raivich G
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