Transplantation of Human Adipose Mesenchymal Stem Cells in Non-Immunosuppressed GRMD Dogs is a Safe Procedure
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The possibility to treat Duchenne muscular dystrophy (DMD), a lethal X-linked disorder, through cell therapy with mesenchymal stromal cells (MSCs) has been widely investigated in different animal models. However, some crucial questions need to be addressed before starting human therapeutic trials, particularly regarding its use for genetic disorders. How safe is the procedure? Are there any side effects following mesenchymal stem cell transplantation? To address these questions for DMD the best model is the golden retriever muscular dystrophy dog (GRMD), which is the closest model to the human condition displaying a much longer lifespan than other models. Here we report the follow-up of 5 GRMD dogs, which were repeatedly transplanted with human adipose-derived mesenchymal stromal cells (hASC), derived from different donors. Xenogeneic cell transplantation, which was done without immunosuppression, was well tolerated in all animals with no apparent long-term adverse effect. In the present study, we show that repeated heterologous stem-cell injection is a safe procedure, which is fundamental before starting human clinical trials.
KeywordsHuman multipotent mesenchymal stromal cells Xenotransplantation Muscular dystrophy Cell therapy GRMD dogs
The collaboration of the following persons is gratefully acknowledged: Mariane Secco, Eder Zucconi, Mariz Vainzof, Tatiana Jazedje, Oswaldo Keith Okamoto, Carolina G. Pires, Daniel Sanches, Marta Canovas, Munira Tanezi Guilhon e Sá, Constância Gotto, Vanessa Sato, Wagner Falciano, and all the Genocão team. This work was supported by CEPID/FAPESP, INCT, CNPq, ABDIM, AACD, the FID project and the Duchenne Research fund.
Compliance with Ethical Standards
Conflict of Interest
The authors indicate no potential conflict of interest.
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