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Updated: Jan 10, 2026

Tail Vein Transection Bleeding Model in Fully Anesthetized Hemophilia A Mice
08:13

Tail Vein Transection Bleeding Model in Fully Anesthetized Hemophilia A Mice

Published on: September 30, 2021

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血友病B的因子IX-帕杜亚AAV基因治疗:阶段1/2和3试验

Feng Xue1, Mankai Ju1, Tienan Zhu2

  • 1State Key Laboratory of Experimental Hematology, National Clinical Research Centre for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.

Nature medicine
|November 20, 2025
PubMed
概括

使用B型血友病腺相关病毒 (AAV) 载体的基因疗法在中国队列中证明了安全性和有效性. 该疗法显著降低了出血率,并实现了高零出血率,支持AAV-FIX Padua基因疗法.

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Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus AAV Capsid Variants09:20

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Body:In certain scenarios, in vitro dissolution tests can replace in vivo bioequivalence studies. This is particularly true when a drug product, though available in varying strengths, maintains proportional similarity in its active and inactive ingredients. In such cases, the need for in vivo bioequivalence studies for lower strength variants may be waived, provided dissolution tests and in vivo studies on the highest strength yield satisfactory results.Bioequivalence can be indicated through...
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