R01 AR062123/AR/NIAMS NIH HHS/United States[Grants and Funding] - Search Results

Year	Number of Results
2012	1
2013	1
2015	1
2016	2
2017	1
2018	2
2019	1
2021	2
2023	1
2024	0

1

Post-Translational Modifications of the DUX4 Protein Impact Toxic Function in FSHD Cell Models.

Knox RN, Eidahl JO, Wallace LM, Choudury SG, Rashnonejad A, Daman K, Guggenbiller MJ, Saad NY, Hoover ME, Zhang L, Branson OE, Emerson CP Jr, Freitas MA, Harper SQ. Knox RN, et al. Ann Neurol. 2023 Aug;94(2):398-413. doi: 10.1002/ana.26668. Epub 2023 May 19. Ann Neurol. 2023. PMID: 37186119

2

Human miRNA miR-675 inhibits DUX4 expression and may be exploited as a potential treatment for Facioscapulohumeral muscular dystrophy.

Saad NY, Al-Kharsan M, Garwick-Coppens SE, Chermahini GA, Harper MA, Palo A, Boudreau RL, Harper SQ. Saad NY, et al. Nat Commun. 2021 Dec 8;12(1):7128. doi: 10.1038/s41467-021-27430-1. Nat Commun. 2021. PMID: 34880230 Free PMC article.

3

A stromal progenitor and ILC2 niche promotes muscle eosinophilia and fibrosis-associated gene expression.

Kastenschmidt JM, Coulis G, Farahat PK, Pham P, Rios R, Cristal TT, Mannaa AH, Ayer RE, Yahia R, Deshpande AA, Hughes BS, Savage AK, Giesige CR, Harper SQ, Locksley RM, Mozaffar T, Villalta SA. Kastenschmidt JM, et al. Cell Rep. 2021 Apr 13;35(2):108997. doi: 10.1016/j.celrep.2021.108997. Cell Rep. 2021. PMID: 33852849 Free PMC article.

4

Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models.

Morelli KH, Griffin LB, Pyne NK, Wallace LM, Fowler AM, Oprescu SN, Takase R, Wei N, Meyer-Schuman R, Mellacheruvu D, Kitzman JO, Kocen SG, Hines TJ, Spaulding EL, Lupski JR, Nesvizhskii A, Mancias P, Butler IJ, Yang XL, Hou YM, Antonellis A, Harper SQ, Burgess RW. Morelli KH, et al. J Clin Invest. 2019 Dec 2;129(12):5568-5583. doi: 10.1172/JCI130600. J Clin Invest. 2019. PMID: 31557132 Free PMC article.

5

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.

6

Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD.

Wallace LM, Saad NY, Pyne NK, Fowler AM, Eidahl JO, Domire JS, Griffin DA, Herman AC, Sahenk Z, Rodino-Klapac LR, Harper SQ. Wallace LM, et al. Mol Ther Methods Clin Dev. 2017 Dec 24;8:121-130. doi: 10.1016/j.omtm.2017.12.005. eCollection 2018 Mar 16. Mol Ther Methods Clin Dev. 2017. PMID: 29387734 Free PMC article.

7

Mouse Dux is myotoxic and shares partial functional homology with its human paralog DUX4.

Eidahl JO, Giesige CR, Domire JS, Wallace LM, Fowler AM, Guckes SM, Garwick-Coppens SE, Labhart P, Harper SQ. Eidahl JO, et al. Hum Mol Genet. 2016 Oct 15;25(20):4577-4589. doi: 10.1093/hmg/ddw287. Hum Mol Genet. 2016. PMID: 28173143 Free PMC article.

8

Homologous Transcription Factors DUX4 and DUX4c Associate with Cytoplasmic Proteins during Muscle Differentiation.

Ansseau E, Eidahl JO, Lancelot C, Tassin A, Matteotti C, Yip C, Liu J, Leroy B, Hubeau C, Gerbaux C, Cloet S, Wauters A, Zorbo S, Meyer P, Pirson I, Laoudj-Chenivesse D, Wattiez R, Harper SQ, Belayew A, Coppée F. Ansseau E, et al. PLoS One. 2016 Jan 27;11(1):e0146893. doi: 10.1371/journal.pone.0146893. eCollection 2016. PLoS One. 2016. PMID: 26816005 Free PMC article.

9

Aberrant splicing in transgenes containing introns, exons, and V5 epitopes: lessons from developing an FSHD mouse model expressing a D4Z4 repeat with flanking genomic sequences.

Ansseau E, Domire JS, Wallace LM, Eidahl JO, Guckes SM, Giesige CR, Pyne NK, Belayew A, Harper SQ. Ansseau E, et al. PLoS One. 2015 Mar 5;10(3):e0118813. doi: 10.1371/journal.pone.0118813. eCollection 2015. PLoS One. 2015. PMID: 25742305 Free PMC article.

10

Dose-dependent Toxicity of Humanized Renilla reniformis GFP (hrGFP) Limits Its Utility as a Reporter Gene in Mouse Muscle.

Wallace LM, Moreo A, Clark KR, Harper SQ. Wallace LM, et al. Mol Ther Nucleic Acids. 2013 Apr 16;2(4):e86. doi: 10.1038/mtna.2013.16. Mol Ther Nucleic Acids. 2013. PMID: 23591809 Free PMC article.

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