2597ライソゾーム病改訂第2版
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100 │D ライソゾーム病の治療857-862 8) Fumagalli F, Calbi V, Natali Sora MG, et al.: Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and ex-panded access. .Lancet 2022; 399: 372-383 9) Gentner B, Tucci F, Galimberti S, et al.: Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome. N Engl J Med 2021; 385: 1929-1940.

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