Tag: MECP2

AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice

Wang, D., Tai, P. W. L. & Gao, G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat. Rev. Drug Discov. 18, 358–378 (2019). CAS  PubMed  PubMed Central  Google Scholar  Li, C. & Samulski, R. J. Engineering adeno-associated virus vectors for gene therapy. Nat. Rev. Genet. 21, 255–272…

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In Rett Models, Regulatory RNAs Appear To Be Altered

Regulatory RNAs involved in nerve cell signaling and structure were altered in cell and mouse models of Rett syndrome, with these changes also evident in tissue from deceased patients, a study reported. These findings aid in understanding the biological processes impaired in Rett and may reveal potential biomarkers to help…

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Environmental Factor – September 2021: Intramural Papers of the Month

IntramuralBy Nicholas Alagna, Kelley Christensen, Mimi Huang, Janelle Weaver, and Qing Xu DNTP develops whole-genome sequencing of cell-free DNA in blood Researchers from the Division of the National Toxicology Program have developed a protocol for whole-genome sequencing of low quantities of circulating cell-free DNA. Small amounts of DNA are typically…

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