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用于乙型血友病治疗的凝血因子 IX

Coagulation Factor IX for Hemophilia B Therapy.

机构信息

Shemyakin and Ovchinnikov Institute of Bioorganic Chemistry, Russian Academy of Sciences.

出版信息

Acta Naturae. 2012 Apr;4(2):62-73.

PMID:22872812
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3408704/
Abstract

Factor IX is a zymogen enzyme of the blood coagulation cascade. Inherited absence or deficit of the IX functional factor causes bleeding disorder hemophilia B, which requires constant protein replacement therapy. Reviewed herein are the current state in the manufacturing of FIX, improved variants of the recombinant protein for therapy, transgenic organisms for obtaining FIX, and the advances in the gene therapy of hemophilia B.

摘要

因子 IX 是血液凝血级联反应的酶原酶。IX 功能因子的遗传性缺乏或减少会导致出血性疾病乙型血友病，需要持续的蛋白质替代疗法。本文综述了 FIX 的制造现状、治疗用重组蛋白的改良变体、用于获得 FIX 的转基因生物以及乙型血友病基因治疗的进展。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fa33/3408704/dc2798168c27/AN20758251-13-062-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fa33/3408704/8fd2d38a5fb4/AN20758251-13-062-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fa33/3408704/9358219beae2/AN20758251-13-062-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fa33/3408704/dc2798168c27/AN20758251-13-062-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fa33/3408704/8fd2d38a5fb4/AN20758251-13-062-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fa33/3408704/9358219beae2/AN20758251-13-062-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fa33/3408704/dc2798168c27/AN20758251-13-062-g003.jpg

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