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Dystrophin expression in muscles of duchenne muscular dystrophy patients after high-density injections of normal myogenic cells.
J Neuropathol Exp Neurol. 2006 Apr; 65(4):371-86.JN

Abstract

A clinical trial was conducted to test a new protocol of normal muscle precursor cell (MPC) allotransplantation in skeletal muscles of patients with Duchenne muscular dystrophy (DMD). Cultured MPCs obtained from one of the patient's parents were implanted in 0.25 or 1 cm of a Tibialis anterior in 9 patients with DMD. MPC injections were placed 1 to 2 mm from each other, and a similar pattern of saline injections was done in the contralateral muscle. The patients were immunosuppressed with tacrolimus. Muscle biopsies were performed at the injected sites 4 weeks later. In the biopsies of the cell-grafted sites, there were myofibers expressing donor's dystrophin in 8 patients. The percentage of myofibers expressing donor's dystrophin varied from 3.5% to 26%. Evidence of small myofiber neoformation was observed in some patients. Donor-derived dystrophin transcripts were detected by reverse transcriptase-polymerase chain reaction in the cell-grafted sites in all patients. The protocol of immunosuppression was sufficient to obtain these results, although it is not certain whether acute rejection was efficiently controlled in all the cases. In conclusion, intramuscular allotransplantation of normal MPCs can induce the expression of donor-derived dystrophin in skeletal muscles of patients with DMD, although this expression is restricted to the sites of MPC injection.

Authors+Show Affiliations

Human Genetic Research Unit, Laval University Hospital Center, Quebec, Canada.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 availableNo affiliation info available

Pub Type(s)

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

Language

eng

PubMed ID

16691118

Citation

Skuk, Daniel, et al. "Dystrophin Expression in Muscles of Duchenne Muscular Dystrophy Patients After High-density Injections of Normal Myogenic Cells." Journal of Neuropathology and Experimental Neurology, vol. 65, no. 4, 2006, pp. 371-86.
Skuk D, Goulet M, Roy B, et al. Dystrophin expression in muscles of duchenne muscular dystrophy patients after high-density injections of normal myogenic cells. J Neuropathol Exp Neurol. 2006;65(4):371-86.
Skuk, D., Goulet, M., Roy, B., Chapdelaine, P., Bouchard, J. P., Roy, R., Dugré, F. J., Sylvain, M., Lachance, J. G., Deschênes, L., Senay, H., & Tremblay, J. P. (2006). Dystrophin expression in muscles of duchenne muscular dystrophy patients after high-density injections of normal myogenic cells. Journal of Neuropathology and Experimental Neurology, 65(4), 371-86.
Skuk D, et al. Dystrophin Expression in Muscles of Duchenne Muscular Dystrophy Patients After High-density Injections of Normal Myogenic Cells. J Neuropathol Exp Neurol. 2006;65(4):371-86. PubMed PMID: 16691118.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Dystrophin expression in muscles of duchenne muscular dystrophy patients after high-density injections of normal myogenic cells. AU - Skuk,Daniel, AU - Goulet,Marlyne, AU - Roy,Brigitte, AU - Chapdelaine,Pierre, AU - Bouchard,Jean-Pierre, AU - Roy,Raynald, AU - Dugré,Francine J, AU - Sylvain,Michel, AU - Lachance,Jean-Guy, AU - Deschênes,Louise, AU - Senay,Hélène, AU - Tremblay,Jacques P, PY - 2006/5/13/pubmed PY - 2006/6/10/medline PY - 2006/5/13/entrez SP - 371 EP - 86 JF - Journal of neuropathology and experimental neurology JO - J. Neuropathol. Exp. Neurol. VL - 65 IS - 4 N2 - A clinical trial was conducted to test a new protocol of normal muscle precursor cell (MPC) allotransplantation in skeletal muscles of patients with Duchenne muscular dystrophy (DMD). Cultured MPCs obtained from one of the patient's parents were implanted in 0.25 or 1 cm of a Tibialis anterior in 9 patients with DMD. MPC injections were placed 1 to 2 mm from each other, and a similar pattern of saline injections was done in the contralateral muscle. The patients were immunosuppressed with tacrolimus. Muscle biopsies were performed at the injected sites 4 weeks later. In the biopsies of the cell-grafted sites, there were myofibers expressing donor's dystrophin in 8 patients. The percentage of myofibers expressing donor's dystrophin varied from 3.5% to 26%. Evidence of small myofiber neoformation was observed in some patients. Donor-derived dystrophin transcripts were detected by reverse transcriptase-polymerase chain reaction in the cell-grafted sites in all patients. The protocol of immunosuppression was sufficient to obtain these results, although it is not certain whether acute rejection was efficiently controlled in all the cases. In conclusion, intramuscular allotransplantation of normal MPCs can induce the expression of donor-derived dystrophin in skeletal muscles of patients with DMD, although this expression is restricted to the sites of MPC injection. SN - 0022-3069 UR - https://www.unboundmedicine.com/medline/citation/16691118/Dystrophin_expression_in_muscles_of_duchenne_muscular_dystrophy_patients_after_high_density_injections_of_normal_myogenic_cells_ L2 - https://academic.oup.com/jnen/article-lookup/doi/10.1097/01.jnen.0000218443.45782.81 DB - PRIME DP - Unbound Medicine ER -