Cellular/gene therapy: haematological disorders and immunodeficiency
Research type
Research Study
Full title
Development of cellular / gene therapies for haematological malignancies and immunodeficiencies
IRAS ID
184180
Contact name
Vanshree Patel
Contact email
Sponsor organisation
Great Ormond Street for Childrem NHS Foundation Trust
Duration of Study in the UK
10 years, 0 months, 1 days
Research summary
A large number of haematological disorders and immunodeficiencies can be potentially treated with gene and cellular therapies. We wish to expand the number of conditions we can treat in this way as we believe they may be more effective and / or less toxic than standard treatment including chemoradiotherapy or haematopoietic progenitor cell (HPC) transplantation with standard conditioning regimens. We are developing novel gene and cell therapies as well as novel conditioning regimes for HPC transplantation which offer patients alternative treatment options and which may be curative.To progress potential treatments from the laboratory to the clinic requires extensive pre-clinical and clinical studies. For such laboratory studies, it is possible to use surplus / existing tissue samples from a variety of sources. We are therefore applying for approval to use such samples, where consent from the donor has already been obtained.
REC name
London - Bromley Research Ethics Committee
REC reference
16/LO/1290
Date of REC Opinion
5 Jul 2016
REC opinion
Favourable Opinion