Laia Alsina
Head of the Allergy and Clinical Immunology Department at the Hospital Sant Joan de Déu in Barcelona.
The article is excellent in methodology and format. The NEJM journal is an assurance of its quality and the research team (Dr. Puck) has been a reference in the field for more than 30 years.
Gene therapy is a current therapeutic strategy, relevant and necessary when other therapeutic solutions fail in highly lethal diseases such as SCID [Severe Combined Immunodeficiencies]. They should be able to reach the market like gene therapy for ADA-SCID, another common form of SCID.
The relevance of gene therapy is made clear in this review by the world's leading gene therapist, Professor Alain Fisher: https://pubmed.ncbi.nlm.nih.gov/36434109/
The most important limitations are economic. Also the risk of oncogenesis [tumour development] of any gene therapy, which has not been seen in this trial.