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Platelet-targeted hyperfunctional FIX gene therapy for hemophilia B mice even with preexisting anti-FIX immunity

Gene therapy may lead to a cure for hemophilia B (HB) if it is successful. Data from clinical trials using adeno-associated virus (AAV)–mediated liver-targeted FIX gene therapy are very encouraging. However, this protocol can be applied only to adults who do not have liver disease or anti-AAV antibo...

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Publicat a:Blood Adv
Autors principals: Schroeder, Jocelyn A., Chen, Juan, Chen, Yingyu, Cai, Yuanhua, Yu, Hongyin, Mattson, Jeremy G., Monahan, Paul E., Shi, Qizhen
Format: Artigo
Idioma:Inglês
Publicat: American Society of Hematology 2021
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7948262/
https://ncbi.nlm.nih.gov/pubmed/33646304
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2020004071
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