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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...
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| Publicado no: | J Control Release |
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| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2019
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| Assuntos: | |
| Acesso em linha: | 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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