Experimental cell therapy tested for rare genetic blood disorder

This early-stage study is testing the safety of an experimental cell therapy called EXG34217 in people with genetic disorders that cause bone marrow failure. The therapy involves collecting a patient's own blood cells, modifying them in a lab to potentially lengthen their telomeres (a key part of cells linked to aging), and then infusing them back. The main goal is to see if this process is safe and tolerable for up to 12 participants, while also checking if it improves blood counts.