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Authors
Osman, Mahmoud Fawzi
Ibrahim, Khalid
Gleeson, Claire
Ibrahim, Haytham
Haque, Ikram Ul
Alhamad, Noora
Ben-Omran, Tawfeg https://orcid.org/0000-0001-8522-7738
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Article History
Received: 13 July 2025
Revised: 17 October 2025
Accepted: 7 November 2025
First Online: 27 November 2025
Competing interests
: The authors declare no competing interests.
: Legal guardians of all patients signed an informed consent after a detailed discussion of the potential benefits, risks, and alternative treatment options for gene therapy. This consent process ensured that families were fully aware of the nature of the therapy, the pre- and post-infusion requirements, and the importance of regular follow-up. All study methods performed in this study was based on approved treatment protocols and guidelines utilized in Qatar and worldwide in accordance with the Helsinki Declaration. (Sidra ethics board ID: SDR600204 and ethical approval number: 1997345).
