A new immunodeficient mouse model for human myoblast transplantation

Design of efficient transplantation strategies for myoblast-based gene therapies in humans requires animal models in which xenografts are tolerated for long periods of time. In addition, such recipients should be able to withstand pretransplantation manipulations for enhancement of graft growth. Her...

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Veröffentlicht in:Human gene therapy 2001-05, Vol.12 (7), p.823-831
Hauptverfasser: COOPER, R. N, IRINTCHEV, A, DI SANTO, J. P, ZWEYER, M, MORGAN, J. E, PARTRIDGE, T. A, BUTLER-BROWNE, G. S, MOULY, V, WERNIG, A
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Sprache:eng
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Zusammenfassung:Design of efficient transplantation strategies for myoblast-based gene therapies in humans requires animal models in which xenografts are tolerated for long periods of time. In addition, such recipients should be able to withstand pretransplantation manipulations for enhancement of graft growth. Here we report that a newly developed immunodeficient mouse carrying two known mutations (the recombinase activating gene 2, RAG2, and the common cytokine receptor gamma, gammac) is a candidate fulfilling these requirements. Skeletal muscles from RAG2(-/-)/gammac(-/-) double mutant mice recover normally after myotoxin application or cryolesion, procedures commonly used to induce regeneration and improve transplantation efficiency. Well-differentiated donor-derived muscle tissue could be detected up to 9 weeks after transplantation of human myoblasts into RAG2(-/-)/gammac(-/-) muscles. These results suggest that the RAG2(-/-)/gammac(-/-) mouse model will provide new opportunities for human muscle research.
ISSN:1043-0342
1557-7422
DOI:10.1089/104303401750148784