Miceli MC - Search Results

1

Targeting RyR Activity Boosts Antisense Exon 44 and 45 Skipping in Human DMD Skeletal or Cardiac Muscle Culture Models.

Barthélémy F, Wang RT, Hsu C, Douine ED, Marcantonio EE, Nelson SF, Miceli MC. Barthélémy F, et al. Among authors: miceli mc. Mol Ther Nucleic Acids. 2019 Dec 6;18:580-589. doi: 10.1016/j.omtn.2019.09.020. Epub 2019 Sep 28. Mol Ther Nucleic Acids. 2019. PMID: 31678734 Free PMC article.

2

Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse.

Wang DW, Mokhonova EI, Kendall GC, Becerra D, Naeini YB, Cantor RM, Spencer MJ, Nelson SF, Miceli MC. Wang DW, et al. Among authors: miceli mc. Mol Ther Nucleic Acids. 2018 Jun 1;11:180-191. doi: 10.1016/j.omtn.2018.02.002. Epub 2018 Feb 13. Mol Ther Nucleic Acids. 2018. PMID: 29858053 Free PMC article.

3

Validation and Detection of Exon Skipping Boosters in DMD Patient Cell Models and mdx Mouse.

Barthelemy F, Wang D, Nelson SF, Miceli MC. Barthelemy F, et al. Among authors: miceli mc. Methods Mol Biol. 2018;1828:309-326. doi: 10.1007/978-1-4939-8651-4_19. Methods Mol Biol. 2018. PMID: 30171550

4

DMD genotype correlations from the Duchenne Registry: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype.

Wang RT, Barthelemy F, Martin AS, Douine ED, Eskin A, Lucas A, Lavigne J, Peay H, Khanlou N, Sweeney L, Cantor RM, Miceli MC, Nelson SF. Wang RT, et al. Among authors: miceli mc. Hum Mutat. 2018 Sep;39(9):1193-1202. doi: 10.1002/humu.23561. Epub 2018 Jul 12. Hum Mutat. 2018. PMID: 29907980 Free PMC article.

5

Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy.

Kendall GC, Mokhonova EI, Moran M, Sejbuk NE, Wang DW, Silva O, Wang RT, Martinez L, Lu QL, Damoiseaux R, Spencer MJ, Nelson SF, Miceli MC. Kendall GC, et al. Among authors: miceli mc. Sci Transl Med. 2012 Dec 12;4(164):164ra160. doi: 10.1126/scitranslmed.3005054. Sci Transl Med. 2012. PMID: 23241744

6

The case for eteplirsen: Paving the way for precision medicine.

Miceli MC, Nelson SF. Miceli MC, et al. Mol Genet Metab. 2016 Jun;118(2):70-1. doi: 10.1016/j.ymgme.2016.04.001. Epub 2016 Apr 14. Mol Genet Metab. 2016. PMID: 27102846 No abstract available.

7

FDA Approval of Eteplirsen for Muscular Dystrophy.

Nelson SF, Miceli MC. Nelson SF, et al. Among authors: miceli mc. JAMA. 2017 Apr 11;317(14):1480. doi: 10.1001/jama.2017.2601. JAMA. 2017. PMID: 28399245 No abstract available.

8

Large in-frame 5' deletions in DMD associated with mild Duchenne muscular dystrophy: Two case reports and a review of the literature.

Gibbs EM, Barthélémy F, Douine ED, Hardiman NC, Shieh PB, Khanlou N, Crosbie RH, Nelson SF, Miceli MC. Gibbs EM, et al. Among authors: miceli mc. Neuromuscul Disord. 2019 Nov;29(11):863-873. doi: 10.1016/j.nmd.2019.09.009. Epub 2019 Sep 24. Neuromuscul Disord. 2019. PMID: 31672265 Free PMC article. Review.

9

A well-tolerated core needle muscle biopsy process suitable for children and adults.

Barthelemy F, Woods JD, Nieves-Rodriguez S, Douine ED, Wang R, Wanagat J, Miceli MC, Nelson SF. Barthelemy F, et al. Among authors: miceli mc. Muscle Nerve. 2020 Dec;62(6):688-698. doi: 10.1002/mus.27041. Epub 2020 Sep 20. Muscle Nerve. 2020. PMID: 32820569 Free PMC article.

10

Quantitative immuno-mass spectrometry imaging of skeletal muscle dystrophin.

Bishop DP, Westerhausen MT, Barthelemy F, Lockwood T, Cole N, Gibbs EM, Crosbie RH, Nelson SF, Miceli MC, Doble PA, Wanagat J. Bishop DP, et al. Among authors: miceli mc. Sci Rep. 2021 Jan 13;11(1):1128. doi: 10.1038/s41598-020-80495-8. Sci Rep. 2021. PMID: 33441839 Free PMC article.

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