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"Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic First Workshop Report: Examining current findings and opportunities around the emerging D2.B10-Dmdmdx/J (D2/mdx) model in context of the classic C57BL/10ScSn-Dmdmdx/J (Bl10/mdx).

Gordish-Dressman H, Willmann R, Pazze LD, Kreibich A, van Putten M, Heydemann A, Bogdanik L, Lutz C, Davies K, Demonbruen AR, Duan D, Elsey D, Fukada SI, Girgenrath M, Gonzalez JP, Grounds MD, Nichols A, Partridge T, Passini M, Sanarica F, Schnell FJ, Wells DJ, Yokota T, Young CS, Zhong Z, Spurney C, Spencer M, De Luca A, Nagaraju K, Aartsma-Rus A.

J Neuromuscul Dis. 2018 Sep 3. doi: 10.3233/JND-180324. [Epub ahead of print]


Synthesis, Characterization, and Function of an RNA-Based Transfection Reagent.

Jain HV, Boehler JF, Nagaraju K, Beaucage SL.

Curr Protoc Nucleic Acid Chem. 2018 Mar;72(1):4.81.1-4.81.29. doi: 10.1002/cpnc.51.


Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone.

Fiorillo AA, Tully CB, Damsker JM, Nagaraju K, Hoffman EP, Heier CR.

Physiol Genomics. 2018 Sep 1;50(9):735-745. doi: 10.1152/physiolgenomics.00134.2017. Epub 2018 Jun 8.


Ryanodine channel complex stabilizer compound S48168/ARM210 as a disease modifier in dystrophin-deficient mdx mice: proof-of-concept study and independent validation of efficacy.

Capogrosso RF, Mantuano P, Uaesoontrachoon K, Cozzoli A, Giustino A, Dow T, Srinivassane S, Filipovic M, Bell C, Vandermeulen J, Massari AM, De Bellis M, Conte E, Pierno S, Camerino GM, Liantonio A, Nagaraju K, De Luca A.

FASEB J. 2018 Feb;32(2):1025-1043. doi: 10.1096/fj.201700182RRR. Epub 2018 Jan 3.


An amphipathic trans-acting phosphorothioate RNA element delivers an uncharged phosphorodiamidate morpholino sequence in mdx mouse myotubes.

Jain HV, Boehler JF, Verthelyi D, Nagaraju K, Beaucage SL.

RSC Adv. 2017;7:42519-42528. doi: 10.1039/C7RA04247G. Epub 2017 Sep 4.


Mitochondria mediate cell membrane repair and contribute to Duchenne muscular dystrophy.

Vila MC, Rayavarapu S, Hogarth MW, Van der Meulen JH, Horn A, Defour A, Takeda S, Brown KJ, Hathout Y, Nagaraju K, Jaiswal JK.

Cell Death Differ. 2017 Feb;24(2):330-342. doi: 10.1038/cdd.2016.127. Epub 2016 Nov 11.


Effect of the IL-1 Receptor Antagonist Kineret® on Disease Phenotype in mdx Mice.

Benny Klimek ME, Sali A, Rayavarapu S, Van der Meulen JH, Nagaraju K.

PLoS One. 2016 May 23;11(5):e0155944. doi: 10.1371/journal.pone.0155944. eCollection 2016.


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.

Skelet Muscle. 2015 Dec 1;5:44. doi: 10.1186/s13395-015-0070-6. eCollection 2015.


Effect of genetic background on the dystrophic phenotype in mdx mice.

Coley WD, Bogdanik L, Vila MC, Yu Q, Van Der Meulen JH, Rayavarapu S, Novak JS, Nearing M, Quinn JL, Saunders A, Dolan C, Andrews W, Lammert C, Austin A, Partridge TA, Cox GA, Lutz C, Nagaraju K.

Hum Mol Genet. 2016 Jan 1;25(1):130-45. doi: 10.1093/hmg/ddv460. Epub 2015 Nov 12.


TNF-α-Induced microRNAs Control Dystrophin Expression in Becker Muscular Dystrophy.

Fiorillo AA, Heier CR, Novak JS, Tully CB, Brown KJ, Uaesoontrachoon K, Vila MC, Ngheim PP, Bello L, Kornegay JN, Angelini C, Partridge TA, Nagaraju K, Hoffman EP.

Cell Rep. 2015 Sep 8;12(10):1678-90. doi: 10.1016/j.celrep.2015.07.066. Epub 2015 Aug 28.


The use of urinary and kidney SILAM proteomics to monitor kidney response to high dose morpholino oligonucleotides in the mdx mouse.

Zhang A, Uaesoontrachoon K, Shaughnessy C, Das JR, Rayavarapu S, Brown KJ, Ray PE, Nagaraju K, van den Anker JN, Hoffman EP, Hathout Y.

Toxicol Rep. 2015;2:838-849.


Muscular dystrophy in the mdx mouse is a severe myopathy compounded by hypotrophy, hypertrophy and hyperplasia.

Duddy W, Duguez S, Johnston H, Cohen TV, Phadke A, Gordish-Dressman H, Nagaraju K, Gnocchi V, Low S, Partridge T.

Skelet Muscle. 2015 May 1;5:16. doi: 10.1186/s13395-015-0041-y. eCollection 2015.


Best Practices and Standard Protocols as a Tool to Enhance Translation for Neuromuscular Disorders.

Willmann R, Luca A, Nagaraju K, Rüegg MA.

J Neuromuscul Dis. 2015 May 14;2(2):113-117.


Non-invasive MRI and spectroscopy of mdx mice reveal temporal changes in dystrophic muscle imaging and in energy deficits.

Heier CR, Guerron AD, Korotcov A, Lin S, Gordish-Dressman H, Fricke S, Sze RW, Hoffman EP, Wang P, Nagaraju K.

PLoS One. 2014 Nov 12;9(11):e112477. doi: 10.1371/journal.pone.0112477. eCollection 2014.


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.

Hum Mol Genet. 2014 Dec 15;23(24):6458-69. doi: 10.1093/hmg/ddu366. Epub 2014 Jul 15.


Pharmacologic management of Duchenne muscular dystrophy: target identification and preclinical trials.

Kornegay JN, Spurney CF, Nghiem PP, Brinkmeyer-Langford CL, Hoffman EP, Nagaraju K.

ILAR J. 2014;55(1):119-49. doi: 10.1093/ilar/ilu011. Review.


Long-term treatment with naproxcinod significantly improves skeletal and cardiac disease phenotype in the mdx mouse model of dystrophy.

Uaesoontrachoon K, Quinn JL, Tatem KS, Van Der Meulen JH, Yu Q, Phadke A, Miller BK, Gordish-Dressman H, Ongini E, Miglietta D, Nagaraju K.

Hum Mol Genet. 2014 Jun 15;23(12):3239-49. doi: 10.1093/hmg/ddu033. Epub 2014 Jan 23.


Role of Toll-like receptors in the pathogenesis of dystrophin-deficient skeletal and heart muscle.

Henriques-Pons A, Yu Q, Rayavarapu S, Cohen TV, Ampong B, Cha HJ, Jahnke V, Van der Meulen J, Wang D, Jiang W, Kandimalla ER, Agrawal S, Spurney CF, Nagaraju K.

Hum Mol Genet. 2014 May 15;23(10):2604-17. doi: 10.1093/hmg/ddt656. Epub 2013 Dec 23.


Effects of Dantrolene Therapy on Disease Phenotype in Dystrophin Deficient mdx Mice.

Quinn JL, Huynh T, Uaesoontrachoon K, Tatem K, Phadke A, Van der Meulen JH, Yu Q, Nagaraju K.

PLoS Curr. 2013 Nov 8;5. pii: doi: 10.1371/


VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects.

Heier CR, Damsker JM, Yu Q, Dillingham BC, Huynh T, Van der Meulen JH, Sali A, Miller BK, Phadke A, Scheffer L, Quinn J, Tatem K, Jordan S, Dadgar S, Rodriguez OC, Albanese C, Calhoun M, Gordish-Dressman H, Jaiswal JK, Connor EM, McCall JM, Hoffman EP, Reeves EK, Nagaraju K.

EMBO Mol Med. 2013 Oct;5(10):1569-85. doi: 10.1002/emmm.201302621. Epub 2013 Sep 9.

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