Literature DB >> 34591611

Hemophilia as a blueprint for gene therapy.

Margaret V Ragni1,2.   

Abstract

Advances in hemophilia gene therapy could enable progress in other inherited disorders.

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Year:  2021        PMID: 34591611     DOI: 10.1126/science.abg0856

Source DB:  PubMed          Journal:  Science        ISSN: 0036-8075            Impact factor:   47.728


  1 in total

1.  Targeted B-domain deletion restores F8 function in human endothelial cells and mice.

Authors:  Zhiqing Hu; Zhuo Li; Yong Wu; Junya Zhao; Lingqian Wu; Miaojin Zhou; Desheng Liang
Journal:  Signal Transduct Target Ther       Date:  2022-06-20
  1 in total

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