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Center for Gene Therapy
Recent Publications

To view a list of all publications for a given individual faculty member, please access his/her biography

Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD gene.
Flanigan KM, Dunn DM, von Niederhausern A, Soltanzadeh P, Howard MT, Sampson JB, Swoboda KJ, Bromberg MB, Mendell JR, Taylor L, Anderson CB, Pestronk A, Florence J, Connolly AM, Mathews KD, Wong B, Finkel RS, Bonnemann CG, Day JW, McDonald C, Weiss RB; the United Dystrophinopathy Project ConsortiumOther investigators and members of the consortium are listed in the Acknowledgments. Hum Mutat. 2010 Dec 16. [Epub ahead of print] PMID: 21165918

BMP9-Induced muscle heterotopic ossification requires changes to the skeletal muscle microenvironment.
Leblanc E, Trensz F, Haroun S, Drouin G, Bergeron E, Penton CM, Montanaro F, Roux S, Faucheux N, Grenier G. J Bone Miner Res. 2010 Dec 16. [Epub ahead of print] PMID: 21165905

Down syndrome patients with pulmonary hypertension have elevated plasma levels of asymmetric dimethylarginine.
Cua CL, Rogers LK, Chicoine LG, Augustine M, Jin Y, Nash PL, Nelin LD. Eur J Pediatr. 2010 Dec 1. [Epub ahead of print ] PMID: 21120524

Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.
Mendell JR, Rodino-Klapac LR, Rosales XQ, Coley BD, Galloway G, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Taylor LE, Flanigan KM, Gastier-Foster JM, Astbury C, Kota J, Sahenk Z, Walker CM, Clark KR. Ann Neurol. 2010 Nov;68(5):629-38.PMID: 21031578

A humanized Smn gene containing the SMN2 nucleotide alteration in exon 7 mimics SMN2 splicing and the SMA disease phenotype.
Gladman JT, Bebee TW, Edwards C, Wang X, Sahenk Z, Rich MM, Chandler DS. Hum Mol Genet. 2010 Nov 1;19(21):4239-52. Epub 2010 Aug 12.PMID: 20705738

Dystrophin immunity in Duchenne's muscular dystrophy.
Mendell JR, Campbell K, Rodino-Klapac L, Sahenk Z, Shilling C, Lewis S, Bowles D, Gray S, Li C, Galloway G, Malik V, Coley B, Clark KR, Li J, Xiao X, Samulski J, McPhee SW, Samulski RJ, Walker CM. N Engl J Med. 2010 Oct 7;363(15):1429-37.PMID: 20925545

Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material.
Lock M, McGorray S, Auricchio A, Ayuso E, Beecham EJ, Blouin-Tavel V, Bosch F, Bose M, Byrne BJ, Caton T, Chiorini JA, Chtarto A, Clark KR, Conlon T, Darmon C, Doria M, Douar A, Flotte TR, Francis JD, Francois A, Giacca M, Korn MT, Korytov I, Leon X, Leuchs B, Lux G, Melas C, Mizukami H, Moullier P, Müller M, Ozawa K, Philipsberg T, Poulard K, Raupp C, Rivière C, Roosendaal SD, Samulski RJ, Soltys SM, Surosky R, Tenenbaum L, Thomas DL, van Montfort B, Veres G, Wright JF, Xu Y, Zelenaia O, Zentilin L, Snyder RO. Hum Gene Ther. 2010 Oct;21(10):1273-85.PMID: 20486768  

DUX4, a candidate gene for facioscapulohumeral muscular dystrophy, causes p53-dependent myopathy in vivo.
Wallace LM, Garwick SE, Mei W, Belayew A, Coppee F, Ladner KJ, Guttridge D, Yang J, Harper SQ. Ann Neurol. 2010 Oct 28. [Epub ahead of print] PMID: 21154413  

Genetic defects in muscular dystrophy.
Chandrasekharan K, Martin PT. Methods Enzymol. 2010;479:291-322.PMID: 20816173

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