Tissue resource for the development of gene therapy treatments
Research type
Research Tissue Bank
IRAS ID
155959
Contact name
Claire Booth
Contact email
Research summary
Tissue resource for development of gene therapy treatments (Gene Therapy Biobank)
REC name
London - Queen Square Research Ethics Committee
REC reference
14/LO/1494
Date of REC Opinion
19 Jan 2015
REC opinion
Further Information Favourable Opinion
Data collection arrangements
We plan to collect primarily blood and bone marrow samples from patients with a genetic disease which may now, or
in the future, be treated with gene or cellular therapies. We may also collect skin biopsies in order to create fibroblast
cell lines for use in preclinical
research. These samples will only be collected if the patient is undergoing either a
clinically indicated blood test or procedure requiring anaesthetic when we would be able to take a bone marrow
aspirate or skin biopsy without causing any distress or discomfort to the patient. These samples will then be
processed and likely frozen until future use. Existing samples may exist from patients who fit the inclusion criteria and
we will seek consent to use these samples.
We anticipate collecting initially approximately 2030
samples each year (inclusive of blood, bone marrow and skin
biopsies). We anticipate using approximately 20 samples each year for experimental purposes. Data will be
collected on the patients diagnosis and genetic mutation (if known) and this will be stored in a secure database
accessed only by the tissue bank manager or those closely involved in the research projects.Research programme
We will be collecting samples from donors with inherited genetic conditions which are currently, or may in the future, be treated with gene or cellular therapies. These samples will be used in the preclinical development (more basic research) and clinical translation of such treatment strategies, for example in safety and efficacy studies allowing more rapid progression to Phase I/II clinical trials.
RTBTitle
Tissue resource for development of gene therapy treatments (Gene Therapy Biobank)
Establishment organisation
UCL Institute of Child Health
Establishment organisation address
30 Guilford Street
London
WC1N 1EH