Generation and characterization of a novel Gaa compound heterozygous mouse model recapitulating human Pompe disease

Authors Huang Wenjun, Wang Jie, Zhou Yafei, Xiao Hongyu, Jiang Kaichong, Cui Jiale, Zhang Yanmin, Zhou Rui
Journal Orphanet Journal of Rare Diseases
Publication Date 2026-02-25
DOI 10.1186/s13023-026-04273-x

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