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Self-regulating gene therapy ameliorates phenotypes and overcomes gene dosage sensitivity in a mouse model of Rett syndrome |
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期刊:Science Translational Medicine 作者:Paul D. Ross; Kamal K.E. Gadalla; Sophie R. Thomson; Jim Selfridge; Noha Bahey; et al 出版日期:2025-04-02 |
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(2025-6-4)