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AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD.
Giesige CR, Wallace LM, Heller KN, Eidahl JO, Saad NY, Fowler AM, Pyne NK, Al-Kharsan M, Rashnonejad A, Chermahini GA, Domire JS, Mukweyi D, Garwick-Coppens SE, Guckes SM, McLaughlin KJ, Meyer K, Rodino-Klapac LR, Harper SQ. Giesige CR, et al. JCI Insight. 2018 Nov 15;3(22):e123538. doi: 10.1172/jci.insight.123538. JCI Insight. 2018. PMID: 30429376 Free PMC article.
Follistatin gene delivery enhances muscle growth and strength in nonhuman primates.
Kota J, Handy CR, Haidet AM, Montgomery CL, Eagle A, Rodino-Klapac LR, Tucker D, Shilling CJ, Therlfall WR, Walker CM, Weisbrode SE, Janssen PM, Clark KR, Sahenk Z, Mendell JR, Kaspar BK. Kota J, et al. Sci Transl Med. 2009 Nov 11;1(6):6ra15. doi: 10.1126/scitranslmed.3000112. Sci Transl Med. 2009. PMID: 20368179 Free PMC article.
Gene therapy for muscular dystrophy: lessons learned and path forward.
Mendell JR, Rodino-Klapac L, Sahenk Z, Malik V, Kaspar BK, Walker CM, Clark KR. Mendell JR, et al. Neurosci Lett. 2012 Oct 11;527(2):90-9. doi: 10.1016/j.neulet.2012.04.078. Epub 2012 May 17. Neurosci Lett. 2012. PMID: 22609847 Free PMC article. Review.
Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin α2 surrogates.
Chicoine LG, Rodino-Klapac LR, Shao G, Xu R, Bremer WG, Camboni M, Golden B, Montgomery CL, Shontz K, Heller KN, Griffin DA, Lewis S, Coley BD, Walker CM, Clark KR, Sahenk Z, Mendell JR, Martin PT. Chicoine LG, et al. Mol Ther. 2014 Apr;22(4):713-24. doi: 10.1038/mt.2013.246. Epub 2013 Oct 22. Mol Ther. 2014. PMID: 24145553 Free PMC article.
Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery.
Chicoine LG, Montgomery CL, Bremer WG, Shontz KM, Griffin DA, Heller KN, Lewis S, Malik V, Grose WE, Shilling CJ, Campbell KJ, Preston TJ, Coley BD, Martin PT, Walker CM, Clark KR, Sahenk Z, Mendell JR, Rodino-Klapac LR. Chicoine LG, et al. Mol Ther. 2014 Feb;22(2):338-347. doi: 10.1038/mt.2013.244. Epub 2013 Oct 23. Mol Ther. 2014. PMID: 24196577 Free PMC article.
Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.
Wein N, Vulin A, Falzarano MS, Szigyarto CA, Maiti B, Findlay A, Heller KN, Uhlén M, Bakthavachalu B, Messina S, Vita G, Passarelli C, Brioschi S, Bovolenta M, Neri M, Gualandi F, Wilton SD, Rodino-Klapac LR, Yang L, Dunn DM, Schoenberg DR, Weiss RB, Howard MT, Ferlini A, Flanigan KM. Wein N, et al. Nat Med. 2014 Sep;20(9):992-1000. doi: 10.1038/nm.3628. Epub 2014 Aug 10. Nat Med. 2014. PMID: 25108525 Free PMC article.
71 results