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Gene therapy using a novel G6PC-S298C variant enhances the long-term efficacy for treating glycogen storage disease type Ia

The current phase I/II clinical trial for human glycogen storage disease type-Ia (GSD-Ia) (NCT 03517085) uses a recombinant adeno-associated virus (rAAV) vector expressing a codon-optimized human glucose-6-phosphatase-α (G6Pase-α or G6PC). DNA sequence changes introduced by codon-optimization can ne...

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書目詳細資料
發表在:Biochem Biophys Res Commun
Main Authors: Zhang, Lisa, Lee, Cheol, Arnaoutova, Irina, Anduaga, Javier, Starost, Matthew F., Mansfield, Brian C., Chou, Janice Y.
格式: Artigo
語言:Inglês
出版: 2020
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC7309276/
https://ncbi.nlm.nih.gov/pubmed/32430177
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.bbrc.2020.04.124
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