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Steven J. Howe
University College London
94Publications
25H-index
3,746Citations
Publications 96
Newest
Fabry disease (FD) is caused by mutations in the GLA gene that encodes lysosomal α-galactosidase-A (α-gal-A). A number of pathogenic mechanisms have been proposed and these include loss of mitochondrial respiratory chain activity. For FD, gene therapy is beginning to be applied as a treatment. In view of the loss of mitochondrial function reported in FD, we have considered here the impact of loss of mitochondrial respiratory chain activity on the ability of a GLA lentiviral vector to increase ce...
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#1Hh Abdul-Razak (RHUL: Royal Holloway, University of London)H-Index: 2
#2C Rocca (RHUL: Royal Holloway, University of London)H-Index: 1
Last.Rafael J. Yáñez-Muñoz (RHUL: Royal Holloway, University of London)H-Index: 20
view all 23 authors...
Genome editing is the introduction of directed modifications in the genome, a process boosted to therapeutic levels by designer nucleases. Building on the experience of ex vivo gene therapy for severe combined immunodeficiencies, it is likely that genome editing of haematopoietic stem/progenitor cells (HSPC) for correction of inherited blood diseases will be an early clinical application. We show molecular evidence of gene correction in a mouse model of primary immunodeficiency. In vitro experim...
1 CitationsSource
#1John R. Counsell (UCL: University College London)H-Index: 4
#2Rajvinder Karda (UCL: University College London)H-Index: 5
Last.Steven J. Howe (UCL: University College London)H-Index: 25
view all 19 authors...
Viral vectors are rapidly being developed for a range of applications in research and gene therapy. Prototype foamy virus (PFV) vectors have been described for gene therapy, although their use has mainly been restricted to ex vivo stem cell modification. Here we report direct in vivo transgene delivery with PFV vectors carrying reporter gene constructs. In our investigations, systemic PFV vector delivery to neonatal mice gave transgene expression in the heart, xiphisternum, liver, pancreas, and ...
1 CitationsSource
#1Me Alonso-Ferrero (UCL: University College London)H-Index: 2
#2Niek P. van Til (UU: Utrecht University)H-Index: 4
Last.Steven J. Howe (UCL: University College London)H-Index: 25
view all 11 authors...
Integration-deficient lentiviruses (IdLVs) deliver genes effectively to tissues but are lost rapidly from dividing cells. This property can be harnessed to express transgenes transiently to manipulate cell biology. Here, we demonstrate the utility of short-term gene expression to improve functional potency of hematopoietic stem and progenitor cells (HSPCs) during transplantation by delivering HOXB4 and Angptl3 using IdLVs to enhance the engraftment of HSPCs. Constitutive overexpression of either...
2 CitationsSource
#1John R. CounsellH-Index: 4
#2Zeinab AsgarianH-Index: 2
Last.Olivier DanosH-Index: 47
view all 11 authors...
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#1John R. CounsellH-Index: 4
#2Zeinab AsgarianH-Index: 2
Last.Olivier Danos (UCL: University College London)H-Index: 15
view all 11 authors...
Duchenne Muscular Dystrophy (DMD) is caused by a lack of dystrophin expression in patient muscle fibres. Current DMD gene therapy strategies rely on the expression of internally deleted forms of dystrophin, missing important functional domains. Viral gene transfer of full-length dystrophin could restore wild-type functionality, although this approach is restricted by the limited capacity of recombinant viral vectors. Lentiviral vectors can package larger transgenes than adeno-associated viruses,...
20 CitationsSource
#1Conrad A. VinkH-Index: 5
#2John R. Counsell (UCL: University College London)H-Index: 4
Last.Steven J. Howe (UCL: University College London)H-Index: 25
view all 11 authors...
Lentiviral vector genomic RNA requires sequences that partially overlap wild-type HIV-1 gag and env genes for packaging into vector particles. These HIV-1 packaging sequences constitute 19.6% of the wild-type HIV-1 genome and contain functional cis elements that potentially compromise clinical safety. Here, we describe the development of a novel lentiviral vector (LTR1) with a unique genomic structure designed to prevent transfer of HIV-1 packaging sequences to patient cells, thus reducing the t...
8 CitationsSource
#1Hh Abdul-RazakH-Index: 2
#2C RoccaH-Index: 1
view all 23 authors...
#2Conrad A. VinkH-Index: 5
Last.Steven J. HoweH-Index: 25
view all 10 authors...
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