西亚试剂：Restoration of Human Dystrophin Following Transplantation o

Restoration of Human Dystrophin Following Transplantation of Exon-Skipping-Engineered DMD Patient Stem Cells into Dystrophic Mice

Rachid Benchaouir,1 Mirella Meregalli,1 Andrea Farini,1 Giuseppe D'Antona,3 Marzia Belicchi,1 Aurélie Goyenvalle,4 Maurizio Battistelli,1 Nereo Bresolin,1 Roberto Bottinelli,3 Luis Garcia,4,5, and Yvan Torrente1,2,

1 Stem Cell Laboratory, Department of Neurological Sciences, Fondazione IRCCS Ospedale Maggiore Policlinico, Centro Dino Ferrari, University of Milan, via F. Sforza 35, 20122 Milan, Italy
2 UNISTEM, Centro Interdipartimentale di Ricerca sulle Cellule Staminali, University of Milan, via Balzaretti 9, 20133 Milan, Italy
3 Department of Experimental Medicine, Human Physiology Unit, University of Pavia, via Forlanini 6, 27100 Pavia, Italy
4 Genethon-CNRS UMR 8115, Groupe Maladie de Duchenne, 1 bis Rue de l'Internatonale, 91000 Evry, France

Corresponding author
Yvan Torrente

Duchenne muscular dystrophy (DMD) is a hereditary disease caused by mutations that disrupt the dystrophin mRNA reading frame. In some cases, forced exclusion (skipping) of a single exon can restore the reading frame, giving rise to a shorter, but still functional, protein. In this study, we constructed lentiviral vectors expressing antisense oligonucleotides in order to induce an efficient exon skipping and to correct the initial frameshift caused by the DMD deletion of CD133+ stem cells. The intramuscular and intra-arterial delivery of genetically corrected CD133 expressing myogenic progenitors isolated from the blood and muscle of DMD patients results in a significant recovery of muscle morphology, function, and dystrophin expression in scid/mdx mice. These data demonstrate that autologous engrafting of blood or muscle-derived CD133+ cells, previously genetically modified to reexpress a functional dystrophin, represents a promising approach for DMD

• 以上资料由西亚试剂：http://www.xiyashiji.com/ 提供此产品的详细信息如密度,含量,分子式,分子量等均可在西亚官网查询   
• 相关产品如汞乙酸汞氯化汞氧化汞碘化汞硫酸汞硝酸汞溴化汞硝酸亚汞氯化亚汞乙酸苯汞碘化汞钾硫氰酸汞氯化氨基汞三氯生三氯氧磷三氯乙烯水合氯醛三氯化磷三氯化钌三氯化钛三氯化铱三氯化铑三氯硫磷三氯乙烷三氯甲烷三氯卡班TCC1,3,5-三氯苯1,2,4-三氯苯1,2,3-三氯苯无水氯化铝三氯乙酸酐三氯乙酸钠碘甲烷二碘甲烷三碘甲烷 三氟碘甲烷硫酸二甲酯氯磺酸苯硫酚苯硫酚钠3-氨基苯硫酚2,6-二氯苯硫酚2,4-二氯苯硫酚2,5-二氯苯硫酚2-甲氧基苯硫酚2-氯乙醇 等均有销售.欢迎订购