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Emergent data influences the risk/benefit assessment of hemophilia gene therapy using recombinant adeno-associated virus

After decades of investigation, gene therapy has received regulatory approval to treat hemophilia. However, since gene therapy investigations were initially conceived, other avenues of treatment have revolutionized the care of hemophilia. Emergent data is showing that gene therapy may not be as bene...

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Detalles Bibliográficos
Autor principal: Puetz, John
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10667906/
https://www.ncbi.nlm.nih.gov/pubmed/38020165
http://dx.doi.org/10.3389/fmed.2023.1256919