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Professor Adrian Thrasher MB BS PhD MRCPCH FRCP FMedSCi Wellcome Trust Senior Clinical Fellow NIHR Senior Investigator Consultant Immunologist GOSH Director of Centre for Immunodeficiency Director of Gene Therapy Programme ICH/GOSH a.thrasher@ich.ucl.ac.uk 020 7813 8490 Funding Sources: Wellcome Trust, MRC, BBSRC, CGD Research Trust, EUFP6/7, NIHR, Mason Medical Research Foundation, Moulton Trust.

Conducting trials of somatic gene therapy for various forms of PID including SCID-X1, CGD, ADA-SCID, and WAS. Other research interests include the pathophysiology of primary immunodeficiency syndromes especially WAS, the actin cytoskeleton in haematopoietic cells (with Dr Siobhan Burns), the development of somatic gene therapy (with Professor Bobby Gaspar, Professor Christine Kinnon, Dr Waseem Qasim, and for ocular disease with Professor Robin Ali), and thymus transplantation (with Dr Graham Davies).

Selected publications

Professor Bobby Gaspar
Professor of Paediatrics and Immunology
Hon Consultant in Paediatric Immunology

h.gaspar@ich.ucl.ac.uk
020 7905 2319

Research in my group spans many aspects of primary immunodeficiencies including genetic diagnosis, molecular and cellular pathogenesis, haematopoietic stem cell transplantation and development of gene therapy.

Current lab projects:

1. Understanding the molecular basis of TACI defects in Common Variable Immunodeficiency using in vitro and in vivo models
2. Development of T cell strategies to improve outcome following stem cell transplantation including genetic modification of T cells using suicide genes and generation of virus specific T cells
3. Development of gene therapy models for X-linked lymphoproliferative disease (XLP) and haemophagocytic lymphohistiocytosis (HLH)
4. Gene therapy for severe combined immunodeficiency (X-linked SCID and ADA SCID) – this includes implementation of clinical trials and development of new safer viral constructs
   

Clinical projects

1. Outcome of stem cell transplantation for ADA deficiency
2. Understanding the systemic nature of ADA deficiency
3. Long term cognitive outcome in patients receiving stem cell transplants

Professor Christine Kinnon
Professor of Molecular Immunology and Head of Unit
Molecular Immunology Unit
Institute of Child Health

C.Kinnon@ich.ucl.ac.uk

Prof Kinnon became a Professor in 1998 and runs a large Unit of over 60 clinicians and scientists. The main interest of her research has been to identify the genes which can cause immunodeficiency. With their identification came the possibility of developing new treatments, including gene therapy. This has resulted in developing new vectors for gene transfer and in the characterisation of haematopoietic stem cells, with the first successful UK trials of gene therapy ongoing at ICH/GOS.

Dr Siobhan Burns
Clinician Scientist

S.burns@ich.ucl.ac.uk

My research group has an interest in the pathogenesis of immune defects. We are particularly interested in cells of the innate immune system including dendritic cells and neutrophils. Several of our projects focus on cell migration and in particular, how the actin cytoskeleton regulates motility.  We are currently working to understand how defects of dendritic cell function contribute to the basis of immunodeficiencies that we see in the clinic.

Dr E Graham Davies
Honorary Reader in Molecular Immunology

gdavies@ich.ucl.nhs.uk

Research Interests: Thymus transplantation in Primary Immunodeficiencies; Cryptosporidial disease in Primary Immunodeficiencies.Long term outcomes after bone marrow transplantation.

Dr Waseem Qasim
BMedSci (Hons), MBBS, MRCP (UK), MRCPCH, PhD
Leukaemia Research Fund & Institute of Child Health Clinical Senior Lecturer
Honorary Consultant in Paediatric Immunology and Bone Marrow Transplantation

W.Qasim@ich.ucl.ac.uk
020 7905 2794

My laboratory research is focussed on developing novel cell and gene based therapies that can be used in the paediatric haematopoietic stem cell transplantation setting. Gene delivery and vector development work is centred on murine retroviral and HIV-1 lentiviral derived systems, and much of this work is directed towards the modification of human T cells. Translational work to test T cell suicide gene therapy in the clinic is at an advanced stage, and plans for adoptive cell therapy to protect against viral reactivation after transplant are being formulated. Other laboratory based projects include redirection of T cell specificity through the introduction of recombinant receptor genes, or reprogramming cells through manipulation of regulatory transcription factors such as FOXP3 and the tracking of cells following the introduction of image-enhancing genes.

Dr Penny Titman
Consultant Clinical Psychologist

titmap@gosh.nhs.uk
020 7829 8896

Penny’s research interests include the psychological impact of immunodeficiencies on children and families. She has carried out research on the long term outcome for children with severe congenital immunodeficiencies treated by HSCT and on quality of life of children affected by an immunodeficiency.

Cellular and gene therapies

Dr Kimberly Gilmour
Clinical Scientist in Immunology
gilmok@gosh.nhs.uk
Dr Kathryn Parsley
Clinical Scientist in Gene therapy
k.parsley@ich.ucl.ac.uk
Dr Sue Swift
Quality Assurance Officer
s.swift@ich.ucl.ac.uk

Comprising two clean room facilities, research in the Cellular Therapies Laboratories is focussed on cell manipulations for Phase 1/2 gene and cell therapy trials. Trials currently underway or to begin shortly include gene therapy trials for ADA-SCID, X-SCID, Wiskott-Aldrich syndrome as well as dendritic cell therapy trials for osteosarcoma and glioma. Manipulations and cell culture for thymic transplantation are being undertaken here. In addition, routine cell manipulations including stem cell isolation, freezing of clinical material and cell preparation for transplant to treat haematological, immunology and metabolic diseases are performed in this facility.

Ms Michelle Quaye
Clinical Trial Coordinator

m.quaye@ich.ucl.ac.uk
020 7905 2292

Clinical trials of investigational medicinal products must comply with a large number of regulations, standards and principals of good practice. The clinical trial coordinator ensures efficient submission, organisation and regulatory compliance of a number of new and ongoing gene therapy clinical trials within the Molecular Immunology department at ICH and Great Ormond Street Hospital for Children.