标题
Advances and challenges for hemophilia gene therapy
作者
关键词
-
出版物
HUMAN MOLECULAR GENETICS
Volume -, Issue -, Pages -
出版商
Oxford University Press (OUP)
发表日期
2019-07-19
DOI
10.1093/hmg/ddz157
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9
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- New Therapies for hemophilia
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- Paired CRISPR/Cas9 Nickases Mediate Efficient Site-Specific Integration of F9 into rDNA Locus of Mouse ESCs
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- Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
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- AAV5–Factor VIII Gene Transfer in Severe Hemophilia A
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- Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors
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