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

Mendell JRRodino-Klapac LSahenk ZMalik VKaspar BKWalker CMClark KR.

Center for Gene Therapy, Research Institute at Nationwide Children's Hospital, Department of Pediatrics, The Ohio State University, Columbus, OH 43205, United States. Electronic address: Diese E-Mail-Adresse ist gegen Spambots geschützt! JavaScript muss aktiviert werden, damit sie angezeigt werden kann. .

 


 

 

A phase I trial of adeno-associated virus serotype 1–gammasarcoglycan gene therapy for limb girdle muscular dystrophy type 2C - Brain 2012; doi: 10.1093/brain/awr342, published on January 11, 2012 and available on this


Serge Herson 1, Faycal Hentati 2, Aude Rigolet 1, Anthony Behin 3, Norma B Romero 3, France Leturcq 4, Pascal Laforêt 3, Thierry Maisonobe 3, Rim Amouri 2, Hafedh Haddad 5, Muriel Audit 6, Marie Montus 5, Carole Masurier 5, Bernard Gjata 5, Christophe Georger 5, Mustapha Cheraï 7, Pierre Carlier 3, Jean-Yves Hogrel 3, Ariane Herson 3, Yves Allenbach 1, François M Lemoine 7, David Klatzmann 7, H Lee Sweeney 8, Richard C. Mulligan 9, Bruno Eymard 3, Didier Caizergues 5, Thomas Voit 3, Olivier Benveniste 1

 

Intervista:  Il Dr. Thomas Voit discute la terapia genica per le gamma-sarcoglicanopatie

http://www.nationwidechildrens.org/dr-thomas-voit-discusses-gamma-sarcoglycan-gene-therapy-march-2012

 


 

 

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.


 

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.


 

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. Epub 2008 Jun 4.

 

 


 

 

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.

PMID:  17164775 [PubMed - indexed for MEDLINE]

 
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