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Items: 1 to 20 of 57

1.

Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice.

Lu-Nguyen N, Malerba A, Popplewell L, Schnell F, Hanson G, Dickson G.

Mol Ther Nucleic Acids. 2017 Mar 17;6:15-28. doi: 10.1016/j.omtn.2016.11.009. Epub 2016 Dec 10.

2.

Circulating Biomarkers for Duchenne Muscular Dystrophy.

Aartsma-Rus A, Spitali P.

J Neuromuscul Dis. 2015 Jul 22;2(s2):S49-S58.

3.

Systemic delivery of adeno-associated viral vectors.

Duan D.

Curr Opin Virol. 2016 Dec;21:16-25. doi: 10.1016/j.coviro.2016.07.006. Epub 2016 Jul 25. Review.

PMID:
27459604
4.

Systemic delivery of IL-27 by an adeno-associated viral vector inhibits T cell-mediated colitis and induces multiple inhibitory pathways in T cells.

Zhu X, Liu Z, Liu JQ, Zhu J, Zhang J, Davis JP, Chu J, Yu J, Zhou J, Li MS, Bai XF.

J Leukoc Biol. 2016 Aug;100(2):403-11. doi: 10.1189/jlb.3A1215-540R. Epub 2016 Apr 22.

PMID:
27106672
5.

Enhancing Transgene Expression from Recombinant AAV8 Vectors in Different Tissues Using Woodchuck Hepatitis Virus Post-Transcriptional Regulatory Element.

Wang L, Wang Z, Zhang F, Zhu R, Bi J, Wu J, Zhang H, Wu H, Kong W, Yu B, Yu X.

Int J Med Sci. 2016 Apr 1;13(4):286-91. doi: 10.7150/ijms.14152. eCollection 2016.

6.

GRAF1 deficiency blunts sarcolemmal injury repair and exacerbates cardiac and skeletal muscle pathology in dystrophin-deficient mice.

Lenhart KC, O'Neill TJ 4th, Cheng Z, Dee R, Demonbreun AR, Li J, Xiao X, McNally EM, Mack CP, Taylor JM.

Skelet Muscle. 2015 Aug 21;5:27. doi: 10.1186/s13395-015-0054-6. eCollection 2015.

7.

Muscle hypertrophy induced by myostatin inhibition accelerates degeneration in dysferlinopathy.

Lee YS, Lehar A, Sebald S, Liu M, Swaggart KA, Talbot CC Jr, Pytel P, Barton ER, McNally EM, Lee SJ.

Hum Mol Genet. 2015 Oct 15;24(20):5711-9. doi: 10.1093/hmg/ddv288. Epub 2015 Jul 23.

8.

Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy.

Hathout Y, Brody E, Clemens PR, Cripe L, DeLisle RK, Furlong P, Gordish-Dressman H, Hache L, Henricson E, Hoffman EP, Kobayashi YM, Lorts A, Mah JK, McDonald C, Mehler B, Nelson S, Nikrad M, Singer B, Steele F, Sterling D, Sweeney HL, Williams S, Gold L.

Proc Natl Acad Sci U S A. 2015 Jun 9;112(23):7153-8. doi: 10.1073/pnas.1507719112. Epub 2015 May 26.

9.

Genetic disruption of Smad7 impairs skeletal muscle growth and regeneration.

Cohen TV, Kollias HD, Liu N, Ward CW, Wagner KR.

J Physiol. 2015 Jun 1;593(11):2479-97. doi: 10.1113/JP270201. Epub 2015 May 15.

10.

Overcoming Insulin Insufficiency by Forced Follistatin Expression in β-cells of db/db Mice.

Zhao C, Qiao C, Tang RH, Jiang J, Li J, Martin CB, Bulaklak K, Li J, Wang DW, Xiao X.

Mol Ther. 2015 May;23(5):866-74. doi: 10.1038/mt.2015.29. Epub 2015 Feb 13.

11.

Development of novel activin-targeted therapeutics.

Chen JL, Walton KL, Al-Musawi SL, Kelly EK, Qian H, La M, Lu L, Lovrecz G, Ziemann M, Lazarus R, El-Osta A, Gregorevic P, Harrison CA.

Mol Ther. 2015 Mar;23(3):434-44. doi: 10.1038/mt.2014.221. Epub 2014 Nov 17.

12.

Muscle and heart function restoration in a limb girdle muscular dystrophy 2I (LGMD2I) mouse model by systemic FKRP gene delivery.

Qiao C, Wang CH, Zhao C, Lu P, Awano H, Xiao B, Li J, Yuan Z, Dai Y, Martin CB, Li J, Lu Q, Xiao X.

Mol Ther. 2014 Nov;22(11):1890-9. doi: 10.1038/mt.2014.141. Epub 2014 Jul 22.

13.

GRAF1 promotes ferlin-dependent myoblast fusion.

Lenhart KC, Becherer AL, Li J, Xiao X, McNally EM, Mack CP, Taylor JM.

Dev Biol. 2014 Sep 15;393(2):298-311. doi: 10.1016/j.ydbio.2014.06.025. Epub 2014 Jul 11.

14.

Molecular and cell-based therapies for muscle degenerations: a road under construction.

Berardi E, Annibali D, Cassano M, Crippa S, Sampaolesi M.

Front Physiol. 2014 Apr 8;5:119. doi: 10.3389/fphys.2014.00119. eCollection 2014. Review.

15.

The potential of adeno-associated viral vectors for gene delivery to muscle tissue.

Wang D, Zhong L, Nahid MA, Gao G.

Expert Opin Drug Deliv. 2014 Mar;11(3):345-64. doi: 10.1517/17425247.2014.871258. Epub 2014 Jan 3. Review.

16.

Cytokines and chemokines as regulators of skeletal muscle inflammation: presenting the case of Duchenne muscular dystrophy.

De Paepe B, De Bleecker JL.

Mediators Inflamm. 2013;2013:540370. doi: 10.1155/2013/540370. Epub 2013 Nov 5. Review.

17.

Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy.

Okada T, Takeda S.

Pharmaceuticals (Basel). 2013 Jun 27;6(7):813-36. doi: 10.3390/ph6070813.

18.

AAV-mediated administration of myostatin pro-peptide mutant in adult Ldlr null mice reduces diet-induced hepatosteatosis and arteriosclerosis.

Guo W, Wong S, Bhasin S.

PLoS One. 2013 Aug 1;8(8):e71017. doi: 10.1371/journal.pone.0071017. Print 2013.

19.

Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model.

Rodino-Klapac LR, Janssen PM, Shontz KM, Canan B, Montgomery CL, Griffin D, Heller K, Schmelzer L, Handy C, Clark KR, Sahenk Z, Mendell JR, Kaspar BK.

Hum Mol Genet. 2013 Dec 15;22(24):4929-37. doi: 10.1093/hmg/ddt342. Epub 2013 Jul 17.

20.

Latent myostatin has significant activity and this activity is controlled more efficiently by WFIKKN1 than by WFIKKN2.

Szláma G, Trexler M, Patthy L.

FEBS J. 2013 Aug;280(16):3822-39. doi: 10.1111/febs.12377. Epub 2013 Jul 5.

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