Llwytho...
Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects
Over the last decade, pioneering liver-directed gene therapy trials for haemophilia B have achieved sustained clinical improvement after a single systemic injection of adeno-associated virus (AAV) derived vectors encoding the human factor IX cDNA. These trials demonstrate the potential of AAV techno...
Wedi'i Gadw mewn:
| Cyhoeddwyd yn: | J Inherit Metab Dis |
|---|---|
| Prif Awduron: | , , , |
| Fformat: | Artigo |
| Iaith: | Inglês |
| Cyhoeddwyd: |
Springer Netherlands
2017
|
| Pynciau: | |
| Mynediad Ar-lein: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5500673/ https://ncbi.nlm.nih.gov/pubmed/28567541 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-017-0053-3 |
| Tagiau: |
Ychwanegu Tag
Dim Tagiau, Byddwch y cyntaf i dagio'r cofnod hwn!
|