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Rare bleeding disorders and advances in gene therapy

Liu, Tiana; Yang, Zhigangb

Blood Coagulation & Fibrinolysis: December 2019 - Volume 30 - Issue 8 - p 371–378
doi: 10.1097/MBC.0000000000000852
REVIEW ARTICLE
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Rare bleeding disorders usually begin in childhood and manifest as varying degrees of bleeding, which can be life-threatening in severe cases. With the development of gene editing technology, it is expected that hereditary coagulation factor disorders will someday be fundamentally cured by gene therapy. On account of their rarity, comprehension of these diseases is essential for the application of new treatment strategies. We have compiled the features of some newly discovered mutations of prothrombin, factor VII, and factor X in recent years. In addition, this review introduces the advances and obstacles in gene therapy.

aLaboratory of Hematology, Guangdong Medical University

bDepartment of Hematology and Rheumatology, Affiliated Zhanjiang Central People's Hospital of Guangdong Medical University, Zhanjiang, Guangdong, China

Correspondence to Zhigang Yang, Department of Hematology and Rheumatology, Affiliated Zhanjiang Central People's Hospital of Guangdong Medical University, Zhanjiang, Guangdong, China. Tel: +86 759-3152128; e-mail: yangzg@gdmu.edu.cn

Received 24 June, 2019

Revised 8 August, 2019

Accepted 21 August, 2019

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