Gene therapy for adenosine deaminase deficiency
Deficiency of adenosine deaminase type 2 (DADA2) is a rare, inherited disorder characterised by abnormal inflammation of various tissues and immunodeficiency. It is caused by mutations in the ADA2 gene, rendering it an ideal candidate for gene therapy. The EU-funded DADA2GT project is exploring a gene therapy strategy for correcting ADA2 mutations in homologous haematopoietic stem cells using gene-editing technologies or gene addition by lentiviral vectors. In a clinical setting, these gene-corrected cells will be infused back to the patient. DADA2GT is a proof-of-concept study with a key goal to evaluate the efficacy of gene correction at the preclinical level using patient-derived cells.
Fields of science
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- medical and health sciencesmedical biotechnologycells technologiesstem cells
- natural sciencesbiological sciencesgeneticsmutation
- medical and health sciencesbasic medicineneurologystroke
- natural sciencesbiological sciencesgeneticsgenomes