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Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species.
Tabebordbar M, Lagerborg KA, Stanton A, King EM, Ye S, Tellez L, Krunnfusz A, Tavakoli S, Widrick JJ, Messemer KA, Troiano EC, Moghadaszadeh B, Peacker BL, Leacock KA, Horwitz N, Beggs AH, Wagers AJ, Sabeti PC. Tabebordbar M, et al. Among authors: beggs ah. Cell. 2021 Sep 16;184(19):4919-4938.e22. doi: 10.1016/j.cell.2021.08.028. Epub 2021 Sep 9. Cell. 2021. PMID: 34506722 Free PMC article.
SPEG interacts with myotubularin, and its deficiency causes centronuclear myopathy with dilated cardiomyopathy.
Agrawal PB, Pierson CR, Joshi M, Liu X, Ravenscroft G, Moghadaszadeh B, Talabere T, Viola M, Swanson LC, Haliloğlu G, Talim B, Yau KS, Allcock RJ, Laing NG, Perrella MA, Beggs AH. Agrawal PB, et al. Among authors: beggs ah. Am J Hum Genet. 2014 Aug 7;95(2):218-26. doi: 10.1016/j.ajhg.2014.07.004. Epub 2014 Jul 31. Am J Hum Genet. 2014. PMID: 25087613 Free PMC article.
Muscle dysfunction in a zebrafish model of Duchenne muscular dystrophy.
Widrick JJ, Alexander MS, Sanchez B, Gibbs DE, Kawahara G, Beggs AH, Kunkel LM. Widrick JJ, et al. Among authors: beggs ah. Physiol Genomics. 2016 Nov 1;48(11):850-860. doi: 10.1152/physiolgenomics.00088.2016. Epub 2016 Oct 7. Physiol Genomics. 2016. PMID: 27764767 Free PMC article.
399 results