Molecular characterization of mutations that cause globoid cell leukodystrophy and pharmacological rescue using small molecule chemical chaperones

Wing C. Lee, Dongcheul Kang, Ena Causevic, Aimee R. Herdt, Elizabeth A. Eckman, Christopher B. Eckman

Research output: Contribution to journal › Article › peer-review

64 Scopus citations

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Medicine & Life Sciences