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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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Dettagli Bibliografici
Pubblicato in:Ann Transl Med
Autori principali: Kishnani, Priya S., Koeberl, Dwight D.
Natura: Artigo
Lingua:Inglês
Pubblicazione: AME Publishing Company 2019
Soggetti:
Accesso online: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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