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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...
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| Publicado no: | Ann Transl Med |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
AME Publishing Company
2019
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| Assuntos: | |
| Acesso em linha: | 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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