Developing gene therapy for autoinflammation
Research type
Research Study
Full title
Developing gene and cell therapy for genetic autoinflammatory disorders
IRAS ID
336390
Contact name
Despina Eleftheriou
Contact email
Sponsor organisation
UCL GOS Institute of Child Health
Duration of Study in the UK
10 years, 0 months, 1 days
Research summary
Autoinflammatory diseases are genetic disorders characterized by a malfunctioning immune system, leading to intense and recurring episodes of inflammation that manifest in symptoms such as fever, rash, or joint swelling. These conditions also pose the risk of amyloidosis, a potentially fatal accumulation of a blood protein in vital organs. The currently available drugs are effective for some patients with autoinflammation, but a significant proportion of patients do not respond to these therapies. As a result, patients are still dying from these conditions and their only hope is bone marrow transplantation, a procedure that is itself associated with 20% chance of dying from complications. Gene therapy, where a working copy of the relevant gene is inserted into a patient’s DNA to replace the faulty disease-causing gene or where a faulty gene is corrected or silenced, may offer these patients a better and safer treatment, with less complications.
Our objective is to develop curative genetic therapies for these autoinflammatory diseases. To achieve this, we need to conduct preclinical studies (laboratory based experiments) to assess the treatment's efficacy in both patient and healthy control cells, as well as in animals. The aim is to determine if gene therapy is effective and ascertain the appropriate dosage for a positive impact. Additionally, we will evaluate its safety by monitoring for any adverse effects. If the preclinical results show promise we will then proceed to design clinical trials involving human subjects. At this stage our study is purely preclinical no therapies will be given to any patients.
REC name
East Midlands - Nottingham 1 Research Ethics Committee
REC reference
24/EM/0068
Date of REC Opinion
29 May 2024
REC opinion
Further Information Favourable Opinion