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High-throughput transcriptome analyses from ASPIRO, a phase 1/2/3 study of gene replacement therapy for X-linked myotubular myopathy.
Andreoletti G, Romano O, Chou HJ, Sefid-Dashti MJ, Grilli A, Chen C, Lakshman N, Purushothaman P, Varfaj F, Mavilio F, Bicciato S, Urbinati F. Andreoletti G, et al. Among authors: chou hj. Am J Hum Genet. 2023 Oct 5;110(10):1648-1660. doi: 10.1016/j.ajhg.2023.08.008. Epub 2023 Sep 5. Am J Hum Genet. 2023. PMID: 37673065 Free PMC article. Clinical Trial.
54 results