Viral Vector-Based Delivery of CRISPR/Cas9 and Donor DNA for Homology-Directed Repair in an In Vitro Model for Canine Hemophilia B-Reference-Cited by-同舟云学术

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Viral Vector-Based Delivery of CRISPR/Cas9 and Donor DNA for Homology-Directed Repair in an In Vitro Model for Canine Hemophilia B

Published:2019-03 Issue: Volume:14 Page:364-376
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Gao Jian,Bergmann Thorsten,Zhang Wenli,Schiwon Maren,Ehrke-Schulz Eric,Ehrhardt Anja

Funder

China Scholarship Council

Witten/Herdecke University

Else Kröner-Fresenius Foundation

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference57 articles.

1. Epidemiology of coagulation disorders;Nathwani;Baillieres Clin. Haematol.,1992

2. Abnormal joint and bone wound healing in hemophilia mice is improved by extending factor IX activity after hemarthrosis;Sun;Blood,2017

3. In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs;Kay;Science,1993

4. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector;Kay;Nat. Genet.,2000

5. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B;Nathwani;N. Engl. J. Med.,2011

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