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Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion

Scritto il . Pubblicato in Terapia genica.

Hum Gene Ther. 2019 Jul;30(7):794-801. doi: 10.1089/hum.2019.006. Epub 2019 Apr 19.

Jerry R Mendell 1 2 3, Louis G Chicoine 1 2, Samiah A Al-Zaidy 2, Zarife Sahenk 1 2 3, Kelly Lehman 1, Linda Lowes 1 2
3, Natalie Miller 1, Lindsay Alfano 1 3, Beverly Galliers 1, Sarah Lewis 1, Darren Murrey 1, Ellyn Peterson 1, Danielle A
Griffin 1, Kathleen Church 1, Sharon Cheatham 4, John Cheatham 4, Mark J Hogan 5, Louise R Rodino-Klapac 1 2
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PMID: 30838895 PMCID: PMC6648191 DOI: 10.1089/hum.2019.006
Free PMC article


https://pubmed.ncbi.nlm.nih.gov/30838895/

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

Scritto il . Pubblicato in Terapia genica.

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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

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