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Updated: May 9, 2026

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
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伦蒂病毒性造血干细胞基因治疗的好处是元色性白血病变异症

Alessandra Biffi1, Eugenio Montini, Laura Lorioli

  • 1San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy. biffi.alessandra@hsr.it

Science (New York, N.Y.)
|July 13, 2013
PubMed
概括

使用lentiviral载体的基因疗法成功地纠正了甲基染色性白血病 (MLD) 患者的造血干细胞 (HSC) 中的arylsulfatase A (ARSA) 缺乏. 这种方法防止了治疗个体的疾病进展,为MLD提供了潜在的治疗策略.

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