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The Gene Transfer Technology Group听is focused upon translational gene therapy for a range of inherited genetic diseases affecting children. 听
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Dr Simon Waddington
Research Group Lead
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Gene Transfer Technology Group and Collaborators
Left to right: Steve Howe, Riccardo Privolizzi, Aura Zelco, Rajvinder Karda, Natalie Suff, Julien Baruteau, Giulia Massaro, Jo Ng, Sam Cuka, Michael Hughes, Simon Waddington, Dany Perocheau, Ahad Rahim, Suzy Buckley
Brain Disease
Dravet Syndrome
An MRC DPFS, grant, awarded to Drs Simon Waddington and听Dr Rajvinder Karda听and Dr Stephanie Schorge (UCL Institute of Neurology) is funding a translational project to develop gene therapy for the incurable inherited childhood epilepsy, Dravet Syndrome. Dr Karda and her PhD student, Juan Antinao Diaz, are developing novel lentiviral vectors to deliver SCN1A gene, which encodes voltage-gated sodium channel 1.1 (NaV1.1) to affected neurons.
Childhood movement disorders
Dr Jo Ng is leading preclinical studies to develop gene therapy for Dopamine Transporter Deficiency Syndrome, a disease first identified by at Great Ormond Street Hospital.
Immunoperoxidase staining of green fluorescent protein in dorsal root ganglia and spinal cord
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Gaucher Disease
In close collaboration with听听(UCL School of Pharmacy), we are working on project to听develop new gene therapy strategies for Neuronopathic Gaucher Disease, a debilitating neurodegenerative disease affecting children. For more information see the Gauchers Association website at听.
Adenovirus vectors
In collaboration with 听and 听(University of Glasgow), Dr (University of Cardiff) and Professor John McVey (University of Surrey) we elucidated one of the听fundamental mechanisms by which adenovirus vectors transduce cells. We continue to collaborate with Dr Parker in optimising new vector iterations
Cartoon of adenovirus vector
Advanced Preclinical Models
We 听collaborate with Drs听听and (National University of Singapore) who are concentrating on characterisation of fetal stem cells and the development of NHP models of gene therapy for treatment of early clotting diseases and for thalassemia.听
Somatotransgenic Bioimaging
We have developed an application of gene听transfer whereby signalling pathways in diseased organs and tumours can be听quantified continually and non-invasively. This project is being driven by Dr Suzy Buckley in collaboration with 听(Manchester Metropolitan University). Recently, these tools were exploited by in order to generate liver-directed biosensors to study transcription factors mediating onset and resolution of liver fibrosis.听听has been using this technology to interrogate the signalling pathways activated during hypoxic ischaemic encephalopathy. We have just published the first tranche of work in Scientific Reports in our paper entitled "". We are happy to freely share any of the constructs generated over the course of this project. More details can be found听here.
Neurogenetic bioimaging
Further information
There are many other resources which explain the concept of gene therapy for different diseases. The 听represents the gene and cell therapy community in the UK. The represents activities across the whole of Europe. There are also resources accessible to a lay audience such as project recently completed by Nowgen which examines the potential for gene therapy to treat diseases including cystic fibrosis.听
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