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Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice.
Cell Stem Cell. 2007 Dec 13; 1(6):646-57.CS

Abstract

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.

Authors+Show Affiliations

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.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

18371406

Citation

Benchaouir, Rachid, et al. "Restoration of Human Dystrophin Following Transplantation of Exon-skipping-engineered DMD Patient Stem Cells Into Dystrophic Mice." Cell Stem Cell, vol. 1, no. 6, 2007, pp. 646-57.
Benchaouir R, Meregalli M, Farini A, et al. Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice. Cell Stem Cell. 2007;1(6):646-57.
Benchaouir, R., Meregalli, M., Farini, A., D'Antona, G., Belicchi, M., Goyenvalle, A., Battistelli, M., Bresolin, N., Bottinelli, R., Garcia, L., & Torrente, Y. (2007). Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice. Cell Stem Cell, 1(6), 646-57. https://doi.org/10.1016/j.stem.2007.09.016
Benchaouir R, et al. Restoration of Human Dystrophin Following Transplantation of Exon-skipping-engineered DMD Patient Stem Cells Into Dystrophic Mice. Cell Stem Cell. 2007 Dec 13;1(6):646-57. PubMed PMID: 18371406.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice. AU - Benchaouir,Rachid, AU - Meregalli,Mirella, AU - Farini,Andrea, AU - D'Antona,Giuseppe, AU - Belicchi,Marzia, AU - Goyenvalle,Aurélie, AU - Battistelli,Maurizio, AU - Bresolin,Nereo, AU - Bottinelli,Roberto, AU - Garcia,Luis, AU - Torrente,Yvan, PY - 2007/05/04/received PY - 2007/07/28/revised PY - 2007/09/24/accepted PY - 2008/3/29/pubmed PY - 2008/8/19/medline PY - 2008/3/29/entrez SP - 646 EP - 57 JF - Cell stem cell JO - Cell Stem Cell VL - 1 IS - 6 N2 - 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. SN - 1875-9777 UR - https://www.unboundmedicine.com/medline/citation/18371406/Restoration_of_human_dystrophin_following_transplantation_of_exon_skipping_engineered_DMD_patient_stem_cells_into_dystrophic_mice_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S1934-5909(07)00186-5 DB - PRIME DP - Unbound Medicine ER -