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Liver depot gene therapy for Pompe disease
Gene therapy for Pompe disease has advanced to early phase clinical trials, based upon proof-of-concept data indicating that gene therapy could surpass the benefits of the current standard of care, enzyme replacement therapy (ERT). ERT requires frequent infusions of large quantities of recombinant h...
Gorde:
| Argitaratua izan da: | Ann Transl Med |
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| Egile Nagusiak: | , |
| Formatua: | Artigo |
| Hizkuntza: | Inglês |
| Argitaratua: |
AME Publishing Company
2019
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| Gaiak: | |
| Sarrera elektronikoa: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6642935/ https://ncbi.nlm.nih.gov/pubmed/31392200 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.21037/atm.2019.05.02 |
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