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Items: 1 to 20 of 110

1.

Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins.

Mendell JR, Rodino-Klapac LR, Rosales-Quintero X, Kota J, Coley BD, Galloway G, Craenen JM, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Viollet L, Walker CM, Sahenk Z, Clark KR.

Ann Neurol. 2009 Sep;66(3):290-7. doi: 10.1002/ana.21732.

PMID:
19798725
2.

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. doi: 10.1002/ana.22251.

3.

Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D.

Rodino-Klapac LR, Lee JS, Mulligan RC, Clark KR, Mendell JR.

Neurology. 2008 Jul 22;71(4):240-7. doi: 10.1212/01.wnl.0000306309.85301.e2. Epub 2008 Jun 4.

PMID:
18525034
4.

A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C.

Herson S, Hentati F, Rigolet A, Behin A, Romero NB, Leturcq F, Laforêt P, Maisonobe T, Amouri R, Haddad H, Audit M, Montus M, Masurier C, Gjata B, Georger C, Cheraï M, Carlier P, Hogrel JY, Herson A, Allenbach Y, Lemoine FM, Klatzmann D, Sweeney HL, Mulligan RC, Eymard B, Caizergues D, Voït T, Benveniste O.

Brain. 2012 Feb;135(Pt 2):483-92. doi: 10.1093/brain/awr342. Epub 2012 Jan 11.

PMID:
22240777
5.

Recombinant adeno-associated virus type 8-mediated extensive therapeutic gene delivery into skeletal muscle of alpha-sarcoglycan-deficient mice.

Nishiyama A, Ampong BN, Ohshima S, Shin JH, Nakai H, Imamura M, Miyagoe-Suzuki Y, Okada T, Takeda S.

Hum Gene Ther. 2008 Jul;19(7):719-30. doi: 10.1089/hum.2007.184.

PMID:
18578595
6.

Epsilon-sarcoglycan compensates for lack of alpha-sarcoglycan in a mouse model of limb-girdle muscular dystrophy.

Imamura M, Mochizuki Y, Engvall E, Takeda S.

Hum Mol Genet. 2005 Mar 15;14(6):775-83. Epub 2005 Feb 2.

PMID:
15689353
7.

Sarcoglycanopathies: can muscle immunoanalysis predict the genotype?

Klinge L, Dekomien G, Aboumousa A, Charlton R, Epplen JT, Barresi R, Bushby K, Straub V.

Neuromuscul Disord. 2008 Dec;18(12):934-41. doi: 10.1016/j.nmd.2008.08.003. Epub 2008 Nov 7.

PMID:
18996010
8.

β-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice.

Pozsgai ER, Griffin DA, Heller KN, Mendell JR, Rodino-Klapac LR.

Gene Ther. 2016 Jan;23(1):57-66. doi: 10.1038/gt.2015.80. Epub 2015 Aug 20.

PMID:
26214262
9.

Long-term skeletal muscle protection after gene transfer in a mouse model of LGMD-2D.

Pacak CA, Walter GA, Gaidosh G, Bryant N, Lewis MA, Germain S, Mah CS, Campbell KP, Byrne BJ.

Mol Ther. 2007 Oct;15(10):1775-81. Epub 2007 Jul 24.

10.

Overexpression of Galgt2 reduces dystrophic pathology in the skeletal muscles of alpha sarcoglycan-deficient mice.

Xu R, DeVries S, Camboni M, Martin PT.

Am J Pathol. 2009 Jul;175(1):235-47. doi: 10.2353/ajpath.2009.080967. Epub 2009 Jun 4.

11.

The first case of primary alpha-sarcoglycanopathy identified in Albania, in two siblings with homozygous alpha-sarcoglycan mutation.

Babameto-Laku A, Tabaku M, Tashko V, Cikuli M, Mokini V.

Genet Couns. 2011;22(4):377-83.

PMID:
22303798
12.

Sarcolemmal alpha and gamma sarcoglycan protein deficiencies in Turkish siblings with a novel missense mutation in the alpha sarcoglycan gene.

Diniz G, Tosun Yildirim H, Akinci G, Hazan F, Ozturk A, Yararbas K, Tukun A.

Pediatr Neurol. 2014 Jun;50(6):640-7. doi: 10.1016/j.pediatrneurol.2013.12.024. Epub 2014 Jan 25.

PMID:
24742800
13.

Gene therapy for muscular dystrophy: lessons learned and path forward.

Mendell JR, Rodino-Klapac L, Sahenk Z, Malik V, Kaspar BK, Walker CM, Clark KR.

Neurosci Lett. 2012 Oct 11;527(2):90-9. doi: 10.1016/j.neulet.2012.04.078. Epub 2012 May 17. Review.

14.

Phenotypic correction of alpha-sarcoglycan deficiency by intra-arterial injection of a muscle-specific serotype 1 rAAV vector.

Fougerousse F, Bartoli M, Poupiot J, Arandel L, Durand M, Guerchet N, Gicquel E, Danos O, Richard I.

Mol Ther. 2007 Jan;15(1):53-61.

15.

Reengineering a transmembrane protein to treat muscular dystrophy using exon skipping.

Gao QQ, Wyatt E, Goldstein JA, LoPresti P, Castillo LM, Gazda A, Petrossian N, Earley JU, Hadhazy M, Barefield DY, Demonbreun AR, Bönnemann C, Wolf M, McNally EM.

J Clin Invest. 2015 Nov 2;125(11):4186-95. doi: 10.1172/JCI82768. Epub 2015 Oct 12.

16.

Evaluation of sarcoglycans, vinculin-talin-integrin system and filamin2 in alpha- and gamma-sarcoglycanopathy: an immunohistochemical study.

Anastasi G, Cutroneo G, Trimarchi F, Santoro G, Bruschetta D, Bramanti P, Pisani A, Favaloro A.

Int J Mol Med. 2004 Dec;14(6):989-99.

PMID:
15547664
17.

Gene transfer establishes primacy of striated vs. smooth muscle sarcoglycan complex in limb-girdle muscular dystrophy.

Durbeej M, Sawatzki SM, Barresi R, Schmainda KM, Allamand V, Michele DE, Campbell KP.

Proc Natl Acad Sci U S A. 2003 Jul 22;100(15):8910-5. Epub 2003 Jul 8.

18.

Autonomic, locomotor and cardiac abnormalities in a mouse model of muscular dystrophy: targeting the renin-angiotensin system.

Sabharwal R, Chapleau MW.

Exp Physiol. 2014 Apr;99(4):627-31. doi: 10.1113/expphysiol.2013.074336. Epub 2013 Dec 13. Review.

19.

Delivery of alpha- and beta-sarcoglycan by recombinant adeno-associated virus: efficient rescue of muscle, but differential toxicity.

Dressman D, Araishi K, Imamura M, Sasaoka T, Liu LA, Engvall E, Hoffman EP.

Hum Gene Ther. 2002 Sep 1;13(13):1631-46.

PMID:
12228018
20.

A new evidence for the maintenance of the sarcoglycan complex in muscle sarcolemma in spite of the primary absence of delta-SG protein.

Gouveia TL, Kossugue PM, Paim JF, Zatz M, Anderson LV, Nigro V, Vainzof M.

J Mol Med (Berl). 2007 Apr;85(4):415-20. Epub 2007 Jan 30.

PMID:
17265058

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