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Translational Potential of Immune Tolerance Induction by AAV Liver-Directed Factor VIII Gene Therapy for Hemophilia A

Hemophilia A (HA) is an X-linked bleeding disorder due to deficiencies in coagulation factor VIII (FVIII). The major complication of current protein-based therapies is the development of neutralizing anti-FVIII antibodies, termed inhibitors, that block the hemostatic effect of therapeutic FVIII. Inh...

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Pubblicato in:Front Immunol
Autori principali: Samelson-Jones, Benjamin J., Arruda, Valder R.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Frontiers Media S.A. 2020
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7212376/
https://ncbi.nlm.nih.gov/pubmed/32425925
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fimmu.2020.00618
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