ICH GEPID Study Version 1.0
Research type
Research Study
Full title
Gene Editing in Primary Immunodeficiency
IRAS ID
296973
Contact name
Adrian Thrasher
Contact email
Sponsor organisation
Institute of Child Health
Duration of Study in the UK
4 years, 0 months, 0 days
Research summary
Primary Immunodeficiencies affect roughly 1 in 15,000 people in the United Kingdom. These diseases cause frequent and serious infections. Genetic causes are increasingly being identified in patients. Conventional therapy depends on the individual condition but can involve life-long infusions of immunoglobulin, prophylactic antibiotics and stem cell transplants. All these treatments come with risks and often patients will still have infections. Currently a safe and definitive treatment is lacking.
Recent progress in gene therapy has led to promising treatments in several Immunodeficiencies where the causative genetic mutation has been identified. This involves putting the correct gene into cells. However, conventional gene therapy has limitations as we cannot control where the gene is inserted.
An alternative novel approach is to use accurate gene editing technology in order to insert the correct gene into the location it is normally found in or directly repair a mutation. This project will be a pre-clinical study to establish this technique for the treatment of Primary Immunodeficiencies.
We would like to trial our technique in cells from patients to see if we can restore their ability to function correctly. We will compare these corrected cells with stem cells from patients without the disease. In order to test the corrected cells and compare them to stem cells from healthy donors, we will inject them into mice which don’t have an immune system and monitor them over several months to determine if their immune system is restored. All these experiments will follow strict Home Office rules which are designed to minimise distress caused to the animals.REC name
North West - Liverpool Central Research Ethics Committee
REC reference
21/NW/0240
Date of REC Opinion
1 Nov 2021
REC opinion
Further Information Favourable Opinion