Nongenotoxic antibody-drug conjugate conditioning enables safe and effective platelet gene therapy of hemophilia A mice

Gene therapy offers the potential to cure hemophilia A (HA). We have shown that hematopoietic stem cell (HSC)–based platelet-specific factor VIII (FVIII) (2bF8) gene therapy can produce therapeutic protein and induce antigen-specific immune tolerance in HA mice, even in the presence of inhibitory an...

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Bibliografische gegevens
Gepubliceerd in:Blood Adv
Hoofdauteurs: Gao, Chunyan, Schroeder, Jocelyn A., Xue, Feng, Jing, Weiqing, Cai, Yuanhua, Scheck, Amelia, Subramaniam, Saravanan, Rao, Sridhar, Weiler, Hartmut, Czechowicz, Agnieszka, Shi, Qizhen
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: American Society of Hematology 2019
Onderwerpen:
Gene Therapy
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6759737/
https://ncbi.nlm.nih.gov/pubmed/31515232
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2019000516
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