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Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9
Hemophilia B (HB) is a life-threatening inherited disease caused by mutations in the FIX gene, leading to reduced protein function and abnormal blood clotting. Due to its monogenic nature, HB is one of the primary targets for gene therapy. Indeed, successful correction of HB has been shown in clinic...
Gardado en:
| Publicado en: | J Control Release |
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| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
2019
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| Assuntos: | |
| Acceso en liña: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6636336/ https://ncbi.nlm.nih.gov/pubmed/30771412 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jconrel.2019.02.009 |
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