Isogenic GAA-KO Murine Muscle Cell Lines Mimicking Severe Pompe Mutations as Preclinical Models for the Screening of Potential Gene Therapy Strategies.
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Int J Mol Sci. 2022 Jun 4;23(11):6298. doi: 10.3390/ijms23116298.
Int J Mol Sci. 2022.
PMID: 35682977
Free PMC article.