If the JMML is cause by a mutation replacing or “correcting” that gene should cure it. How far are we from that kind of intervention?
To date, this kind of gene-therapy is impossible and, to our knowledge, no group is investigating this approach. Gene-therapy has been employed in some genetic disorders, where it is sufficient to correct a small number of cells. In JMML, it would be necessary to correct all leukemia cells, which would be impossible at this time point. Additionally, gene-therapy currently has a risk of inducing leukemia.
