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Impaired activity of the fusogenic micropeptide Myomixer causes myopathy resembling Carey-Fineman-Ziter syndrome.
Ramirez-Martinez A, Zhang Y, van den Boogaard MJ, McAnally JR, Rodriguez-Caycedo C, Chai AC, Chemello F, Massink MP, Cuppen I, Elferink MG, van Es RJ, Janssen NG, Walraven-van Oijen LP, Liu N, Bassel-Duby R, van Jaarsveld RH, Olson EN. Ramirez-Martinez A, et al. Among authors: chemello f. J Clin Invest. 2022 Jun 1;132(11):e159002. doi: 10.1172/JCI159002. J Clin Invest. 2022. PMID: 35642635 Free PMC article.
CRISPR-Editing Therapy for Duchenne Muscular Dystrophy.
Chemello F, Olson EN, Bassel-Duby R. Chemello F, et al. Hum Gene Ther. 2023 May;34(9-10):379-387. doi: 10.1089/hum.2023.053. Hum Gene Ther. 2023. PMID: 37060194 Free PMC article. Review.
Single-swap editing for the correction of common Duchenne muscular dystrophy mutations.
Chai AC, Chemello F, Li H, Nishiyama T, Chen K, Zhang Y, Sánchez-Ortiz E, Alomar A, Xu L, Liu N, Bassel-Duby R, Olson EN. Chai AC, et al. Among authors: chemello f. Mol Ther Nucleic Acids. 2023 Apr 19;32:522-535. doi: 10.1016/j.omtn.2023.04.009. eCollection 2023 Jun 13. Mol Ther Nucleic Acids. 2023. PMID: 37215149 Free PMC article.
41 results