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Development of Novel Micro-dystrophins with Enhanced Functionality.

Ramos JN, Hollinger K, Bengtsson NE, Allen JM, Hauschka SD, Chamberlain JS.

Mol Ther. 2019 Mar 6;27(3):623-635. doi: 10.1016/j.ymthe.2019.01.002. Epub 2019 Feb 1.


Muscle-dominant wild-type TDP-43 expression induces myopathological changes featuring tubular aggregates and TDP-43-positive inclusions.

Tawara N, Yamashita S, Kawakami K, Kurashige T, Zhang Z, Tasaki M, Yamamoto Y, Nishikami T, Doki T, Zhang X, Matsuo Y, Kimura E, Tawara A, Maeda Y, Hauschka SD, Maruyama H, Ando Y.

Exp Neurol. 2018 Nov;309:169-180. doi: 10.1016/j.expneurol.2018.08.006. Epub 2018 Aug 18.


Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy.

Amoasii L, Long C, Li H, Mireault AA, Shelton JM, Sanchez-Ortiz E, McAnally JR, Bhattacharyya S, Schmidt F, Grimm D, Hauschka SD, Bassel-Duby R, Olson EN.

Sci Transl Med. 2017 Nov 29;9(418). pii: eaan8081. doi: 10.1126/scitranslmed.aan8081. Erratum in: Sci Transl Med. 2018 Jan 24;10 (425):.


Corrigendum: Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.

Bengtsson NE, Hall JK, Odom GL, Phelps MP, Andrus CR, Hawkins RD, Hauschka SD, Chamberlain JR, Chamberlain JS.

Nat Commun. 2017 Jun 23;8:16007. doi: 10.1038/ncomms16007.


Translation of Cardiac Myosin Activation with 2-deoxy-ATP to Treat Heart Failure via an Experimental Ribonucleotide Reductase-Based Gene Therapy.

Thomson KS, Odom GL, Murry CE, Mahairas GG, Moussavi-Harami F, Teichman SL, Chen X, Hauschka SD, Chamberlain JS, Regnier M.

JACC Basic Transl Sci. 2016 Dec;1(7):666-679. doi: 10.1016/j.jacbts.2016.07.006.


Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.

Bengtsson NE, Hall JK, Odom GL, Phelps MP, Andrus CR, Hawkins RD, Hauschka SD, Chamberlain JR, Chamberlain JS.

Nat Commun. 2017 Feb 14;8:14454. doi: 10.1038/ncomms14454. Erratum in: Nat Commun. 2017 Jun 23;8:16007.


AAV6-mediated Cardiac-specific Overexpression of Ribonucleotide Reductase Enhances Myocardial Contractility.

Kolwicz SC Jr, Odom GL, Nowakowski SG, Moussavi-Harami F, Chen X, Reinecke H, Hauschka SD, Murry CE, Mahairas GG, Regnier M.

Mol Ther. 2016 Feb;24(2):240-250. doi: 10.1038/mt.2015.176. Epub 2015 Sep 21.


Engraftment potential of dermal fibroblasts following in vivo myogenic conversion in immunocompetent dystrophic skeletal muscle.

Muir LA, Nguyen QG, Hauschka SD, Chamberlain JS.

Mol Ther Methods Clin Dev. 2014 Jun 25;1:14025.


Myocyte-mediated arginase expression controls hyperargininemia but not hyperammonemia in arginase-deficient mice.

Hu C, Kasten J, Park H, Bhargava R, Tai DS, Grody WW, Nguyen QG, Hauschka SD, Cederbaum SD, Lipshutz GS.

Mol Ther. 2014 Oct;22(10):1792-802. doi: 10.1038/mt.2014.99. Epub 2014 Jun 3.


Human embryonic stem cell-derived cardiomyocytes migrate in response to gradients of fibronectin and Wnt5a.

Moyes KW, Sip CG, Obenza W, Yang E, Horst C, Welikson RE, Hauschka SD, Folch A, Laflamme MA.

Stem Cells Dev. 2013 Aug 15;22(16):2315-25. doi: 10.1089/scd.2012.0586. Epub 2013 May 8.


Targeted genomic integration of a selectable floxed dual fluorescence reporter in human embryonic stem cells.

Gantz JA, Palpant NJ, Welikson RE, Hauschka SD, Murry CE, Laflamme MA.

PLoS One. 2012;7(10):e46971. doi: 10.1371/journal.pone.0046971. Epub 2012 Oct 10.


Analysis of fiber-type differences in reporter gene expression of ╬▓-gal transgenic muscle.

Tai PW, Smith CL, Angello JC, Hauschka SD.

Methods Mol Biol. 2012;798:445-59. doi: 10.1007/978-1-61779-343-1_26.


Analysis of muscle gene transcription in cultured skeletal muscle cells.

Himeda CL, Tai PW, Hauschka SD.

Methods Mol Biol. 2012;798:425-43. doi: 10.1007/978-1-61779-343-1_25.


Differentiation and fiber type-specific activity of a muscle creatine kinase intronic enhancer.

Tai PW, Fisher-Aylor KI, Himeda CL, Smith CL, Mackenzie AP, Helterline DL, Angello JC, Welikson RE, Wold BJ, Hauschka SD.

Skelet Muscle. 2011 Jul 7;1:25. doi: 10.1186/2044-5040-1-25.


Transcription factor rational design improves directed differentiation of human mesenchymal stem cells into skeletal myocytes.

Gon├žalves MA, Janssen JM, Nguyen QG, Athanasopoulos T, Hauschka SD, Dickson G, de Vries AA.

Mol Ther. 2011 Jul;19(7):1331-41. doi: 10.1038/mt.2010.308. Epub 2011 Jan 25.


Design and testing of regulatory cassettes for optimal activity in skeletal and cardiac muscles.

Himeda CL, Chen X, Hauschka SD.

Methods Mol Biol. 2011;709:3-19. doi: 10.1007/978-1-61737-982-6_1.


KLF3 regulates muscle-specific gene expression and synergizes with serum response factor on KLF binding sites.

Himeda CL, Ranish JA, Pearson RC, Crossley M, Hauschka SD.

Mol Cell Biol. 2010 Jul;30(14):3430-43. doi: 10.1128/MCB.00302-10. Epub 2010 Apr 19.


Interleukin-10 prevents diet-induced insulin resistance by attenuating macrophage and cytokine response in skeletal muscle.

Hong EG, Ko HJ, Cho YR, Kim HJ, Ma Z, Yu TY, Friedline RH, Kurt-Jones E, Finberg R, Fischer MA, Granger EL, Norbury CC, Hauschka SD, Philbrick WM, Lee CG, Elias JA, Kim JK.

Diabetes. 2009 Nov;58(11):2525-35. doi: 10.2337/db08-1261. Epub 2009 Aug 18.


Quantitative proteomic identification of MAZ as a transcriptional regulator of muscle-specific genes in skeletal and cardiac myocytes.

Himeda CL, Ranish JA, Hauschka SD.

Mol Cell Biol. 2008 Oct;28(20):6521-35. doi: 10.1128/MCB.00306-08. Epub 2008 Aug 18.


Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy.

Sun B, Young SP, Li P, Di C, Brown T, Salva MZ, Li S, Bird A, Yan Z, Auten R, Hauschka SD, Koeberl DD.

Mol Ther. 2008 Aug;16(8):1366-71. doi: 10.1038/mt.2008.133. Epub 2008 Jun 17.


Embryonic cardiomyocyte expression of endothelial genes.

Welikson RE, Kaestner S, Evans AM, Hauschka SD.

Dev Dyn. 2007 Sep;236(9):2512-22.


Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle.

Salva MZ, Himeda CL, Tai PW, Nishiuchi E, Gregorevic P, Allen JM, Finn EE, Nguyen QG, Blankinship MJ, Meuse L, Chamberlain JS, Hauschka SD.

Mol Ther. 2007 Feb;15(2):320-9.


BMP induction of cardiogenesis in P19 cells requires prior cell-cell interaction(s).

Angello JC, Kaestner S, Welikson RE, Buskin JN, Hauschka SD.

Dev Dyn. 2006 Aug;235(8):2122-33.


Human umbilical vein endothelial cells fuse with cardiomyocytes but do not activate cardiac gene expression.

Welikson RE, Kaestner S, Reinecke H, Hauschka SD.

J Mol Cell Cardiol. 2006 Apr;40(4):520-8. Epub 2006 Mar 6.


Improvement in survival and muscle function in an mdx/utrn(-/-) double mutant mouse using a human retinal dystrophin transgene.

Gaedigk R, Law DJ, Fitzgerald-Gustafson KM, McNulty SG, Nsumu NN, Modrcin AC, Rinaldi RJ, Pinson D, Fowler SC, Bilgen M, Burns J, Hauschka SD, White RA.

Neuromuscul Disord. 2006 Mar;16(3):192-203. Epub 2006 Feb 17.


Lamin A/C and emerin are critical for skeletal muscle satellite cell differentiation.

Frock RL, Kudlow BA, Evans AM, Jameson SA, Hauschka SD, Kennedy BK.

Genes Dev. 2006 Feb 15;20(4):486-500.


Stable transduction of myogenic cells with lentiviral vectors expressing a minidystrophin.

Li S, Kimura E, Fall BM, Reyes M, Angello JC, Welikson R, Hauschka SD, Chamberlain JS.

Gene Ther. 2005 Jul;12(14):1099-108.


Hematopoietic stem cell transplantation does not restore dystrophin expression in Duchenne muscular dystrophy dogs.

Dell'Agnola C, Wang Z, Storb R, Tapscott SJ, Kuhr CS, Hauschka SD, Lee RS, Sale GE, Zellmer E, Gisburne S, Bogan J, Kornegay JN, Cooper BJ, Gooley TA, Little MT.

Blood. 2004 Dec 15;104(13):4311-8. Epub 2004 Aug 24.


Quantitative proteomic identification of six4 as the trex-binding factor in the muscle creatine kinase enhancer.

Himeda CL, Ranish JA, Angello JC, Maire P, Aebersold R, Hauschka SD.

Mol Cell Biol. 2004 Mar;24(5):2132-43.


Tissue engineering of skeletal muscle using polymer fiber arrays.

Neumann T, Hauschka SD, Sanders JE.

Tissue Eng. 2003 Oct;9(5):995-1003.


Differences in the function of three conserved E-boxes of the muscle creatine kinase gene in cultured myocytes and in transgenic mouse skeletal and cardiac muscle.

Nguyen QG, Buskin JN, Himeda CL, Shield MA, Hauschka SD.

J Biol Chem. 2003 Nov 21;278(47):46494-505. Epub 2003 Sep 10.


Spatially organized layers of cardiomyocytes on biodegradable polyurethane films for myocardial repair.

McDevitt TC, Woodhouse KA, Hauschka SD, Murry CE, Stayton PS.

J Biomed Mater Res A. 2003 Sep 1;66(3):586-95.


Viral vectors for gene transfer of micro-, mini-, or full-length dystrophin.

Scott JM, Li S, Harper SQ, Welikson R, Bourque D, DelloRusso C, Hauschka SD, Chamberlain JS.

Neuromuscul Disord. 2002 Oct;12 Suppl 1:S23-9. Review.


In vitro generation of differentiated cardiac myofibers on micropatterned laminin surfaces.

McDevitt TC, Angello JC, Whitney ML, Reinecke H, Hauschka SD, Murry CE, Stayton PS.

J Biomed Mater Res. 2002 Jun 5;60(3):472-9.


A novel leucine zipper targets AKAP15 and cyclic AMP-dependent protein kinase to the C terminus of the skeletal muscle Ca2+ channel and modulates its function.

Hulme JT, Ahn M, Hauschka SD, Scheuer T, Catterall WA.

J Biol Chem. 2002 Feb 8;277(6):4079-87. Epub 2001 Nov 30.


IGFs, insulin, Shh, bFGF, and TGF-beta1 interact synergistically to promote somite myogenesis in vitro.

Pirskanen A, Kiefer JC, Hauschka SD.

Dev Biol. 2000 Aug 15;224(2):189-203.


Analysis of muscle creatine kinase regulatory elements in recombinant adenoviral vectors.

Hauser MA, Robinson A, Hartigan-O'Connor D, Williams-Gregory DA, Buskin JN, Apone S, Kirk CJ, Hardy S, Hauschka SD, Chamberlain JS.

Mol Ther. 2000 Jul;2(1):16-25.


Electromechanical coupling between skeletal and cardiac muscle. Implications for infarct repair.

Reinecke H, MacDonald GH, Hauschka SD, Murry CE.

J Cell Biol. 2000 May 1;149(3):731-40.


Ex vivo gene transfer using adenovirus-mediated full-length dystrophin delivery to dystrophic muscles.

Floyd SS Jr, Clemens PR, Ontell MR, Kochanek S, Day CS, Yang J, Hauschka SD, Balkir L, Morgan J, Moreland MS, Feero GW, Epperly M, Huard J.

Gene Ther. 1998 Jan;5(1):19-30.


Improved adenoviral vectors for gene therapy of Duchenne muscular dystrophy.

Hauser MA, Amalfitano A, Kumar-Singh R, Hauschka SD, Chamberlain JS.

Neuromuscul Disord. 1997 Jul;7(5):277-83.


Skeletal myoblast transplantation for repair of myocardial necrosis.

Murry CE, Wiseman RW, Schwartz SM, Hauschka SD.

J Clin Invest. 1996 Dec 1;98(11):2512-23.


Muscle differentiation during repair of myocardial necrosis in rats via gene transfer with MyoD.

Murry CE, Kay MA, Bartosek T, Hauschka SD, Schwartz SM.

J Clin Invest. 1996 Nov 15;98(10):2209-17.

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