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Gene therapy for hemophilia

Gene therapy offers the potential for a cure for patients with hemophilia by establishing continuous endogenous expression of factor VIII or factor IX (FIX) following transfer of a functional gene to replace the hemophilic patient’s own defective gene. The hemophilias are ideally suited for gene the...

詳細記述

保存先:
書誌詳細
出版年:Hematology Am Soc Hematol Educ Program
第一著者: Nathwani, Amit C.
フォーマット: Artigo
言語:Inglês
出版事項: American Society of Hematology 2019
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC6913446/
https://ncbi.nlm.nih.gov/pubmed/31808868
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/hematology.2019000007
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