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Gene therapy – are we ready now?
INTRODUCTION: Haemophilia therapy has evolved from rudimentary transfusion‐based approaches to an unprecedented level of innovation with glimmers of functional cure brought by gene therapy. After decades of misfires, gene therapy has normalized factor (F)VIII and factor (F)IX levels in some individu...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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John Wiley and Sons Inc.
2022
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9325484/ https://www.ncbi.nlm.nih.gov/pubmed/35521736 http://dx.doi.org/10.1111/hae.14530 |