IL10RA gene therapy

IL10RA gene therapy refers to an experimental therapeutic approach aimed at treating monogenic inflammatory bowel disease (IBD) caused by deficiency in the interleukin-10 receptor subunit alpha (IL10RA). A specific strategy developed by researchers at the University Hospital, LMU, Munich, utilizes a CRISPR-Cas9/rAAV6-mediated genome-editing system. This approach involves using CRISPR-Cas9 to create a targeted double-strand break at the endogenous IL10RA locus and a recombinant adeno-associated virus serotype 6 (rAAV6) vector to provide a wild-type IL10RA cDNA donor template. By integrating the functional cDNA at the native translational start site, the therapy restores IL-10-mediated STAT3 signaling and anti-inflammatory functions in immune cells, such as macrophages. This ex vivo strategy, tested in iPSCs and CD34+ hematopoietic stem cells, is intended as a potentially safer and more accessible alternative to allogeneic hematopoietic stem cell transplantation.

Other names

CRISPR–Cas9/AAV6-mediated IL10RA correctionIL10RA genome editingIL-10RA genome editingIL 10RA genome editing