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Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial.
Mendell JR, Sahenk Z, Lehman KJ, Lowes LP, Reash NF, Iammarino MA, Alfano LN, Lewis S, Church K, Shell R, Potter RA, Griffin DA, Hogan M, Wang S, Mason S, Darton E, Rodino-Klapac LR. Mendell JR, et al. Among authors: sahenk z. Muscle Nerve. 2024 Jan;69(1):93-98. doi: 10.1002/mus.27955. Epub 2023 Aug 14. Muscle Nerve. 2024. PMID: 37577753 Clinical Trial.
Impaired regeneration in LGMD2A supported by increased PAX7-positive satellite cell content and muscle-specific microrna dysregulation.
Rosales XQ, Malik V, Sneh A, Chen L, Lewis S, Kota J, Gastier-Foster JM, Astbury C, Pyatt R, Reshmi S, Rodino-Klapac LR, Clark KR, Mendell JR, Sahenk Z. Rosales XQ, et al. Among authors: sahenk z. Muscle Nerve. 2013 May;47(5):731-9. doi: 10.1002/mus.23669. Epub 2013 Mar 29. Muscle Nerve. 2013. PMID: 23553538 Free PMC article.
Defective membrane fusion and repair in Anoctamin5-deficient muscular dystrophy.
Griffin DA, Johnson RW, Whitlock JM, Pozsgai ER, Heller KN, Grose WE, Arnold WD, Sahenk Z, Hartzell HC, Rodino-Klapac LR. Griffin DA, et al. Among authors: sahenk z. Hum Mol Genet. 2016 May 15;25(10):1900-1911. doi: 10.1093/hmg/ddw063. Epub 2016 Feb 23. Hum Mol Genet. 2016. PMID: 26911675 Free PMC article.
AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1.
Ozes B, Myers M, Moss K, Mckinney J, Ridgley A, Chen L, Bai S, Abrams CK, Freidin MM, Mendell JR, Sahenk Z. Ozes B, et al. Among authors: sahenk z. Gene Ther. 2022 Apr;29(3-4):127-137. doi: 10.1038/s41434-021-00231-3. Epub 2021 Feb 4. Gene Ther. 2022. PMID: 33542455 Free PMC article.
129 results