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Updated: Apr 9, 2026

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
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对于血友病B的Fidanacogene Elaparvovec - 一个多年的随访研究

John E J Rasko1,2, Benjamin J Samelson-Jones3,4, Lindsey A George3,4

  • 1University of Sydney, Central Clinical School, Faculty of Medicine and Health, Sydney.

The New England journal of medicine
|April 16, 2025
PubMed
概括

菲达纳可 (Fidanacogene elaparvovec) 基因疗法在B型血友病患者中证明了长期的安全性和有效性. 治疗维持了XIX因子的活性,在3至6年内显著减少了出血事件.

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