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BCKDHA-BCKDHB digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine disease

Science Translational Medicine
2025-02-26 | Journal article | Author
DOI: 10.1126/scitranslmed.ads0539
Contributors: Qin Su; David Chuang; Erik G. Puffenberger; Ran He; Karlla Brigatti; Heather Gray-Edwards; Kevin Strauss; Zhong Jiang; Alisha Gruntman; Lingzhi Ren et al.
Source: check_circle
AAAS - Science
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