Cargando…

How to translate and implement the current science of gene therapy into haemophilia care?

Gene-based therapy opens an entirely new paradigm in managing people with haemophilia (PWH), offering them the possibility of a functional cure by enabling continuous expression of factor VIII (FVIII) or factor IX (FIX) after transfer of a functional gene designed to replace the PWH’s own defective...

Descripción completa

Detalles Bibliográficos
Autores principales: Hermans, Cedric, Gruel, Yves, Frenzel, Laurent, Krumb, Evelien
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9841832/
https://www.ncbi.nlm.nih.gov/pubmed/36654740
http://dx.doi.org/10.1177/20406207221145627