Reacción a "Gene therapy is effective long-term in children with a serious rare disease"

This is an excellent article in terms of patient numbers and follow-up. Furthermore, all aspects of clinical and immunological efficacy and safety have been well assessed.

It complements the current knowledge we have regarding lentivirus-based gene therapy in severe combined immunodeficiency due to ADA deficiency.

Its main implication is to place gene therapy as the first line of treatment ahead of stem cell transplantation in this condition, as previously proposed due to its excellent results.

There are no major limitations, although the risk of oncogenicity due to clonal activation should continue to be assessed with even longer follow-up. This long-term safety point is very important and I believe it is not sufficiently addressed in the press release.