AAV9-based PMM2 gene replacement augments PMM2 expression and improves glycosylation in primary fibroblasts of patients with phosphomannomutase 2 deficiency (PMM2-CDG)

Zhong, M.; Balakrishnan, B.; Guo, A.J.; Lai, K.

doi:10.1016/j.ymgmr.2023.101035

Molecular Genetics and Metabolism Reports

2024

DOI: 10.1016/j.ymgmr.2023.101035

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AAV9-based PMM2 gene replacement augments PMM2 expression and improves glycosylation in primary fibroblasts of patients with phosphomannomutase 2 deficiency (PMM2-CDG)

M. Zhong,

B. Balakrishnan,

A.J. Guo

et al.

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AAV9-based PMM2 gene replacement augments PMM2 expression and improves glycosylation in primary fibroblasts of patients with phosphomannomutase 2 deficiency (PMM2-CDG)

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References 17 publications

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