Development of gene/cell therapies for inherited genetic disorders
Research type
Research Study
Full title
Development of gene and cell therapies for inherited genetic disorders
IRAS ID
173557
Contact name
Claire Booth
Contact email
Sponsor organisation
Great Ormond Street for Children NHS Foundation Trust
Duration of Study in the UK
9 years, 11 months, 14 days
Research summary
An increasing number of genetic disorders are now amenable to treatment by gene and cell therapy techniques. Our research has demonstrated curative therapies for a number of inherited immunodeficiencies. We now want to develop gene and cell therapy for an increasing number of genetic disorders. To do this, we need to model treatment strategies in patient and healthy donor cells. This study will allow us to access surplus and existing material from a number of different sources (including an ethically approved biobank bank based at our centre) for this purpose. This study will involve a number of laboratory research projects which fall within the remit of this ethics application.
REC name
East of England - Cambridge South Research Ethics Committee
REC reference
15/EE/0055
Date of REC Opinion
18 Feb 2015
REC opinion
Further Information Favourable Opinion