Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.
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Migliavacca M, et al. Among authors: duse m.
Nat Med. 2024 Feb;30(2):488-497. doi: 10.1038/s41591-023-02789-4. Epub 2024 Feb 14.
Nat Med. 2024.
PMID: 38355973
Free PMC article.