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Items: 1 to 20 of 845

1.

A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy.

Rodino-Klapac LR, Janssen PM, Montgomery CL, Coley BD, Chicoine LG, Clark KR, Mendell JR.

J Transl Med. 2007 Sep 24;5:45.

2.

[Gene therapy for muscular dystrophy].

Takeda S.

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

PMID:
15031985
3.

Differential effects of dystrophin and utrophin gene transfer in immunocompetent muscular dystrophy (mdx) mice.

Ebihara S, Guibinga GH, Gilbert R, Nalbantoglu J, Massie B, Karpati G, Petrof BJ.

Physiol Genomics. 2000 Sep 8;3(3):133-44.

PMID:
11015608
4.

[Current status and perspective of gene therapy on dystrophic animal model].

Takeda S.

Rinsho Shinkeigaku. 2004 Nov;44(11):911-3. Review. Japanese.

PMID:
15651329
5.

Stable micro-dystrophin gene transfer using an integrating adeno-retroviral hybrid vector ameliorates the dystrophic pathology in mdx mouse muscle.

Roberts ML, Wells DJ, Graham IR, Fabb SA, Hill VJ, Duisit G, Yuasa K, Takeda S, Cosset FL, Dickson G.

Hum Mol Genet. 2002 Jul 15;11(15):1719-30.

6.

Mdx mice inducibly expressing dystrophin provide insights into the potential of gene therapy for duchenne muscular dystrophy.

Ahmad A, Brinson M, Hodges BL, Chamberlain JS, Amalfitano A.

Hum Mol Genet. 2000 Oct 12;9(17):2507-15.

7.

Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product.

Yuasa K, Sakamoto M, Miyagoe-Suzuki Y, Tanouchi A, Yamamoto H, Li J, Chamberlain JS, Xiao X, Takeda S.

Gene Ther. 2002 Dec;9(23):1576-88.

8.

Full-length dystrophin gene transfer to the mdx mouse in utero.

Reay DP, Bilbao R, Koppanati BM, Cai L, O'Day TL, Jiang Z, Zheng H, Watchko JF, Clemens PR.

Gene Ther. 2008 Apr;15(7):531-6. doi: 10.1038/gt.2008.8. Epub 2008 Feb 14.

PMID:
18273052
9.

Restoration of dystrophin expression in mdx mice by intravascular injection of naked DNA containing full-length dystrophin cDNA.

Liang KW, Nishikawa M, Liu F, Sun B, Ye Q, Huang L.

Gene Ther. 2004 Jun;11(11):901-8.

PMID:
14985786
10.

AAV-mediated overexpression of human α7 integrin leads to histological and functional improvement in dystrophic mice.

Heller KN, Montgomery CL, Janssen PM, Clark KR, Mendell JR, Rodino-Klapac LR.

Mol Ther. 2013 Mar;21(3):520-5. doi: 10.1038/mt.2012.281. Epub 2013 Jan 15.

11.

Autotransplantation in mdx mice of mdx myoblasts genetically corrected by an HSV-1 amplicon vector.

Bujold M, Caron N, Camiran G, Mukherjee S, Allen PD, Tremblay JP, Wang Y.

Cell Transplant. 2002;11(8):759-67.

PMID:
12588108
12.
13.

Immune response to full-length dystrophin delivered to Dmd muscle by a high-capacity adenoviral vector.

Gilchrist SC, Ontell MP, Kochanek S, Clemens PR.

Mol Ther. 2002 Sep;6(3):359-68.

PMID:
12231172
14.

Genetic correction of dystrophin deficiency and skeletal muscle remodeling in adult MDX mouse via transplantation of retroviral producer cells.

Fassati A, Wells DJ, Sgro Serpente PA, Walsh FS, Brown SC, Strong PN, Dickson G.

J Clin Invest. 1997 Aug 1;100(3):620-8.

15.

Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy.

Harper SQ, Hauser MA, DelloRusso C, Duan D, Crawford RW, Phelps SF, Harper HA, Robinson AS, Engelhardt JF, Brooks SV, Chamberlain JS.

Nat Med. 2002 Mar;8(3):253-61.

PMID:
11875496
17.

Electroporation of corrective nucleic acids (CNA) in vivo to promote gene correction in dystrophic muscle.

Kapsa RM, Wong SH, Quigley AF.

Methods Mol Biol. 2008;423:405-19. doi: 10.1007/978-1-59745-194-9_32.

PMID:
18370218
18.

Enhanced effect of microdystrophin gene transfection by HSV-VP22 mediated intercellular protein transport.

Xiong F, Xiao S, Yu M, Li W, Zheng H, Shang Y, Peng F, Zhao C, Zhou W, Chen H, Fang L, Chamberlain JS, Zhang C.

BMC Neurosci. 2007 Jul 8;8:50.

19.

Efficient and fast functional screening of microdystrophin constructs in vivo and in vitro for therapy of duchenne muscular dystrophy.

Jørgensen LH, Larochelle N, Orlopp K, Dunant P, Dudley RW, Stucka R, Thirion C, Walter MC, Laval SH, Lochmüller H.

Hum Gene Ther. 2009 Jun;20(6):641-50. doi: 10.1089/hum.2008.162.

PMID:
19239382
20.

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse.

Lu QL, Mann CJ, Lou F, Bou-Gharios G, Morris GE, Xue SA, Fletcher S, Partridge TA, Wilton SD.

Nat Med. 2003 Aug;9(8):1009-14. Epub 2003 Jul 6.

PMID:
12847521
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