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Effects of Chronic, Maximal Phosphorodiamidate Morpholino Oligomer (PMO) Dosing on Muscle Function and Dystrophin Restoration in a Mouse Model of Duchenne Muscular Dystrophy.
Benny Klimek ME, Vila MC, Edwards K, Boehler J, Novak J, Zhang A, Van der Meulen J, Tatum K, Quinn J, Fiorillo A, Burki U, Straub V, Lu QL, Hathout Y, van Den Anker J, Partridge TA, Morales M, Hoffman E, Nagaraju K. Benny Klimek ME, et al. Among authors: zhang a. J Neuromuscul Dis. 2021;8(s2):S369-S381. doi: 10.3233/JND-210701. J Neuromuscul Dis. 2021. PMID: 34569970
Discovery of serum protein biomarkers in the mdx mouse model and cross-species comparison to Duchenne muscular dystrophy patients.
Hathout Y, Marathi RL, Rayavarapu S, Zhang A, Brown KJ, Seol H, Gordish-Dressman H, Cirak S, Bello L, Nagaraju K, Partridge T, Hoffman EP, Takeda S, Mah JK, Henricson E, McDonald C. Hathout Y, et al. Among authors: zhang a. Hum Mol Genet. 2014 Dec 15;23(24):6458-69. doi: 10.1093/hmg/ddu366. Epub 2014 Jul 15. Hum Mol Genet. 2014. PMID: 25027324 Free PMC article.
Elusive sources of variability of dystrophin rescue by exon skipping.
Vila MC, Klimek MB, Novak JS, Rayavarapu S, Uaesoontrachoon K, Boehler JF, Fiorillo AA, Hogarth MW, Zhang A, Shaughnessy C, Gordish-Dressman H, Burki U, Straub V, Lu QL, Partridge TA, Brown KJ, Hathout Y, van den Anker J, Hoffman EP, Nagaraju K. Vila MC, et al. Among authors: zhang a. Skelet Muscle. 2015 Dec 1;5:44. doi: 10.1186/s13395-015-0070-6. eCollection 2015. Skelet Muscle. 2015. PMID: 26634117 Free PMC article.
Clinical utility of serum biomarkers in Duchenne muscular dystrophy.
Hathout Y, Seol H, Han MH, Zhang A, Brown KJ, Hoffman EP. Hathout Y, et al. Among authors: zhang a. Clin Proteomics. 2016 Apr 5;13:9. doi: 10.1186/s12014-016-9109-x. eCollection 2016. Clin Proteomics. 2016. PMID: 27051355 Free PMC article. Review.
Author Correction: Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle.
Novak JS, Hogarth MW, Boehler JF, Nearing M, Vila MC, Heredia R, Fiorillo AA, Zhang A, Hathout Y, Hoffman EP, Jaiswal JK, Nagaraju K, Cirak S, Partridge TA. Novak JS, et al. Among authors: zhang a. Nat Commun. 2018 Jan 15;9(1):208. doi: 10.1038/s41467-017-02206-8. Nat Commun. 2018. PMID: 29335405 Free PMC article.
Author Correction: Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle.
Novak JS, Hogarth MW, Boehler JF, Nearing M, Vila MC, Heredia R, Fiorillo AA, Zhang A, Hathout Y, Hoffman EP, Jaiswal JK, Nagaraju K, Cirak S, Partridge TA. Novak JS, et al. Among authors: zhang a. Nat Commun. 2018 Mar 23;9(1):1256. doi: 10.1038/s41467-018-03709-8. Nat Commun. 2018. PMID: 29572439 Free PMC article.
Morpholino-induced exon skipping stimulates cell-mediated and humoral responses to dystrophin in mdx mice.
Vila MC, Novak JS, Benny Klimek M, Li N, Morales M, Fritz AG, Edwards K, Boehler JF, Hogarth MW, Kinder TB, Zhang A, Mazala D, Fiorillo AA, Douglas B, Chen YW, van den Anker J, Lu QL, Hathout Y, Hoffman EP, Partridge TA, Nagaraju K. Vila MC, et al. Among authors: zhang a. J Pathol. 2019 Jul;248(3):339-351. doi: 10.1002/path.5263. Epub 2019 Apr 16. J Pathol. 2019. PMID: 30883742 Free PMC article.
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