Genetic ENgineering Of Skin (GENOS)
Research type
Research Study
Full title
Characterisation and genetic correction/modification of cells from patients with inherited skin diseases: A Feasibility study
IRAS ID
288555
Contact name
John McGrath
Contact email
Sponsor organisation
King's College London
Duration of Study in the UK
1 years, 0 months, 0 days
Research summary
Patients with genetic (inherited) skin diseases (GSD), caused by underlying genetic defects, often endure lifelong pain and suffering as currently there is no cure for these individuals. In order to find an effective treatment for these patients, we aim to try and correct these defective genes in order to restore their function and hopefully improve patient symptoms. To do this, we will take a skin sample (biopsy) from patients with GSD and grow skin cells (known as keratinocytes and fibroblasts) in the laboratory. These cells will be expanded further using established techniques. To increase the survival of these cells, some will be converted into a different type called induced pluripotent stem cells (that can continue to divide and be used to create many more cells). The defective gene in these skin cells that is responsible for the GSD will be changed or fixed in two ways: [1] using a vector (such as a disabled inactive virus) carrying a working copy of the gene; or [2] using molecular scissors or enzymes (called endonucleases). These modified or corrected skin cells will then be assessed to see whether the genetic manipulation has led to an improvement in function and behaviour of the cells. Other sources of cells that can be genetically manipulated include cells from blood. Some fully anonymised samples may be sent to our collaborators’ laboratories as and when their expertise is required. If these techniques can be shown to result in a recovery of function in the cells then this will create a platform for future studies that take our gene modification and genomic editing work into clinical trials to benefit patients. The current application, however, is limited to clinical sampling and laboratory correction/testing and does not include clinical trial work.
REC name
London - Camden & Kings Cross Research Ethics Committee
REC reference
21/PR/1636
Date of REC Opinion
21 Jan 2022
REC opinion
Further Information Favourable Opinion