Current Therapies in Hemophilia: From Plasma-Derived Factor Modalities to CRISPR/Cas Alternatives
Author:
Affiliation:
1. División de Genética, Centro de Investigación Biomédica de Occidente, Instituto Mexicano del Seguro Social
Publisher
Tohoku University Medical Press
Subject
General Biochemistry, Genetics and Molecular Biology,General Medicine
Link
https://www.jstage.jst.go.jp/article/tjem/256/3/256_256.197/_pdf
Reference79 articles.
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2. Bergmann, T., Ehrke-Schulz, E., Gao, J., Schiwon, M., Schildgen, V., David, S., Schildgen, O. & Ehrhardt, A.(2018) Designer nuclease-mediated gene correction via homology-directed repair in an in vitro model of canine hemophilia B. J. Gene Med., 20, e3020.
3. Blanchette, V.S., Key, N.S., Ljung, L.R., Manco-Johnson, M.J., van den Berg, H.M. & Srivastava, A.; Subcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders of the Scientific and Standardization Committee of the International Society on Thrombosis and Hemostasis (2014) Definitions in hemophilia: communication from the SSC of the ISTH. J. Thromb. Haemost., 12, 1935-1939.
4. Chen, H., Shi, M., Gilam, A., Zheng, Q., Zhang, Y., Afrikanova, I., Li, J., Gluzman, Z., Jiang, R., Kong, L.J. & Chen-Tsai, R.Y.(2019) Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII. Sci. Rep., 9, 16838.
5. Chen, J., An, B., Yu, B., Peng, X., Yuan, H., Yang, Q., Chen, X., Yu, T., Wang, L., Zhang, X., Wang, H., Zou, X., Pang, D., Ouyang, H. & Tang, X.(2021) CRISPR/Cas9-mediated knockin of human factor IX into swine factor IX locus effectively alleviates bleeding in hemophilia B pigs. Haematologica, 106, 829-837.
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1. The More Recent History of Hemophilia Treatment;Seminars in Thrombosis and Hemostasis;2022-09-15
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