HOME HELP FEEDBACK SUBSCRIPTIONS ARCHIVE SEARCH TABLE OF CONTENTS		QUICK SEARCH:   [advanced] Author: Keyword(s): Year:  Vol:  Page:

This Article Full Text Full Text (PDF) Supplemental Data All Versions of this Article: 2008-0043v1 26/9/2391    most recent Alert me when this article is cited Alert me if a correction is posted Citation Map Services Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Reprints/Permissions Citing Articles Citing Articles via Google Scholar Google Scholar Articles by Vieira, N. M. Articles by Zatz, M. Search for Related Content PubMed PubMed Citation Articles by Vieira, N. M. Articles by Zatz, M.

TISSUE-SPECIFIC STEM CELLS

SJL Dystrophic Mice Express a Significant Amount of Human Muscle Proteins Following Systemic Delivery of Human Adipose-Derived Stromal Cells Without Immunosuppression

^aHuman Genome Research Center, Biosciences Institute,
^bSchool of Physical Education and Sport, and
^cDepartment of Immunology, Instituto de Ciências Biomédicas, University of São Paulo, São Paulo, Brazil;
^dBiotechnology Department, National Nuclear Energy Commission-IPEN-CNEN, São Paulo, Brazil

Key Words. Human adipose stromal cells • Xenotransplantation • Muscular dystrophy • Therapy

Correspondence: Correspondence: Mayana Zatz, Ph.D., Human Genome Research Center, Department of Genetic and Evolutive Biology, University of São Paulo, Rua do Matão, n.106, Cidade Universitária, São Paulo SP, Brasil-CEP: 05508-090. Telephone: (55) (11) 3091-7966; e-mail: mayazatz{at}usp.br

Received on January 14, 2008; accepted for publication on June 11, 2008.

First published online in STEM CELLS EXPRESS  June 26, 2008.

Limb-girdle muscular dystrophies (LGMDs) are a heterogeneous group of disorders characterized by progressive degeneration of skeletal muscle caused by the absence of or defective muscular proteins. The murine model for limb-girdle muscular dystrophy 2B (LGMD2B), the SJL mice, carries a deletion in the dysferlin gene that causes a reduction in the protein levels to 15% of normal. The mice show muscle weakness that begins at 4–6 weeks and is nearly complete by 8 months of age. The possibility of restoring the defective muscle protein and improving muscular performance by cell therapy is a promising approach for the treatment of LGMDs or other forms of progressive muscular dystrophies. Here we have injected human adipose stromal cells (hASCs) into the SJL mice, without immunosuppression, aiming to assess their ability to engraft into recipient dystrophic muscle after systemic delivery; form chimeric human/mouse muscle fibers; express human muscle proteins in the dystrophic host and improve muscular performance. We show for the first time that hASCs are not rejected after systemic injection even without immunosuppression, are able to fuse with the host muscle, express a significant amount of human muscle proteins, and improve motor ability of injected animals. These results may have important applications for future therapy in patients with different forms of muscular dystrophies.

Disclosure of potential conflicts of interest is found at the end of this article.