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Myostatin propeptide gene delivery by adeno-associated virus serotype 8 vectors enhances muscle growth and ameliorates dystrophic phenotypes in mdx mice.

Qiao C, Li J, Jiang J, Zhu X, Wang B, Li J, Xiao X.

Hum Gene Ther. 2008 Mar;19(3):241-54. doi: 10.1089/hum.2007.159.


Hydrodynamic limb vein injection of adeno-associated virus serotype 8 vector carrying canine myostatin propeptide gene into normal dogs enhances muscle growth.

Qiao C, Li J, Zheng H, Bogan J, Li J, Yuan Z, Zhang C, Bogan D, Kornegay J, Xiao X.

Hum Gene Ther. 2009 Jan;20(1):1-10. doi: 10.1089/hum.2008.135.


Systemic myostatin inhibition via liver-targeted gene transfer in normal and dystrophic mice.

Morine KJ, Bish LT, Pendrak K, Sleeper MM, Barton ER, Sweeney HL.

PLoS One. 2010 Feb 11;5(2):e9176. doi: 10.1371/journal.pone.0009176.


Combined effect of AAV-U7-induced dystrophin exon skipping and soluble activin Type IIB receptor in mdx mice.

Hoogaars WM, Mouisel E, Pasternack A, Hulmi JJ, Relizani K, Schuelke M, Schirwis E, Garcia L, Ritvos O, Ferry A, 't Hoen PA, Amthor H.

Hum Gene Ther. 2012 Dec;23(12):1269-79. doi: 10.1089/hum.2012.056.


Functional improvement of dystrophic muscle by myostatin blockade.

Bogdanovich S, Krag TO, Barton ER, Morris LD, Whittemore LA, Ahima RS, Khurana TS.

Nature. 2002 Nov 28;420(6914):418-21.


Myostatin propeptide-mediated amelioration of dystrophic pathophysiology.

Bogdanovich S, Perkins KJ, Krag TO, Whittemore LA, Khurana TS.

FASEB J. 2005 Apr;19(6):543-9.


Transgenic expression of a myostatin inhibitor derived from follistatin increases skeletal muscle mass and ameliorates dystrophic pathology in mdx mice.

Nakatani M, Takehara Y, Sugino H, Matsumoto M, Hashimoto O, Hasegawa Y, Murakami T, Uezumi A, Takeda S, Noji S, Sunada Y, Tsuchida K.

FASEB J. 2008 Feb;22(2):477-87.


Novel adeno-associated viral vector delivering the utrophin gene regulator jazz counteracts dystrophic pathology in mdx mice.

Strimpakos G, Corbi N, Pisani C, Di Certo MG, Onori A, Luvisetto S, Severini C, Gabanella F, Monaco L, Mattei E, Passananti C.

J Cell Physiol. 2014 Sep;229(9):1283-91. doi: 10.1002/jcp.24567.


AAV-mediated delivery of a mutated myostatin propeptide ameliorates calpain 3 but not alpha-sarcoglycan deficiency.

Bartoli M, Poupiot J, Vulin A, Fougerousse F, Arandel L, Daniele N, Roudaut C, Noulet F, Garcia L, Danos O, Richard I.

Gene Ther. 2007 May;14(9):733-40.


Muscle-bone interactions in dystrophin-deficient and myostatin-deficient mice.

Montgomery E, Pennington C, Isales CM, Hamrick MW.

Anat Rec A Discov Mol Cell Evol Biol. 2005 Sep;286(1):814-22.


Adeno-associated virus serotype-9 microdystrophin gene therapy ameliorates electrocardiographic abnormalities in mdx mice.

Bostick B, Yue Y, Lai Y, Long C, Li D, Duan D.

Hum Gene Ther. 2008 Aug;19(8):851-6. doi: 10.1089/hum.2008.058.


AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype.

Yoshimura M, Sakamoto M, Ikemoto M, Mochizuki Y, Yuasa K, Miyagoe-Suzuki Y, Takeda S.

Mol Ther. 2004 Nov;10(5):821-8.


Inhibition of the IKK/NF-κB pathway by AAV gene transfer improves muscle regeneration in older mdx mice.

Tang Y, Reay DP, Salay MN, Mi MY, Clemens PR, Guttridge DC, Robbins PD, Huard J, Wang B.

Gene Ther. 2010 Dec;17(12):1476-83. doi: 10.1038/gt.2010.110.


Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophy.

Zhang Y, Yue Y, Li L, Hakim CH, Zhang K, Thomas GD, Duan D.

Hum Mol Genet. 2013 Sep 15;22(18):3720-9. doi: 10.1093/hmg/ddt224.


Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice.

Koo T, Popplewell L, Athanasopoulos T, Dickson G.

Hum Gene Ther. 2014 Feb;25(2):98-108. doi: 10.1089/hum.2013.164.


[Gene therapy for muscular dystrophy].

Takeda S.

No To Hattatsu. 2004 Mar;36(2):117-23. Review. Japanese.


Delivery of AAV2/9-microdystrophin genes incorporating helix 1 of the coiled-coil motif in the C-terminal domain of dystrophin improves muscle pathology and restores the level of α1-syntrophin and α-dystrobrevin in skeletal muscles of mdx mice.

Koo T, Malerba A, Athanasopoulos T, Trollet C, Boldrin L, Ferry A, Popplewell L, Foster H, Foster K, Dickson G.

Hum Gene Ther. 2011 Nov;22(11):1379-88. doi: 10.1089/hum.2011.020.


Improved success of myoblast transplantation in mdx mice by blocking the myostatin signal.

Benabdallah BF, Bouchentouf M, Tremblay JP.

Transplantation. 2005 Jun 27;79(12):1696-702.


A canine minidystrophin is functional and therapeutic in mdx mice.

Wang B, Li J, Qiao C, Chen C, Hu P, Zhu X, Zhou L, Bogan J, Kornegay J, Xiao X.

Gene Ther. 2008 Aug;15(15):1099-106. doi: 10.1038/gt.2008.70.

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