Stem Cell Reviews and Reports

, Volume 12, Issue 4, pp 448–453 | Cite as

Transplantation of Human Adipose Mesenchymal Stem Cells in Non-Immunosuppressed GRMD Dogs is a Safe Procedure

  • M. V. Pelatti
  • J. P. A. Gomes
  • N. M. S. Vieira
  • E. Cangussu
  • V. Landini
  • T. Andrade
  • M. Sartori
  • L. Petrus
  • Mayana ZatzEmail author


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.


Human 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.

Supplementary material

12015_2016_9659_MOESM1_ESM.pdf (516 kb)
Supplementary figure 1 Smart Ultrasound evaluation 3 months after treatment. (PDF 516 kb)
12015_2016_9659_MOESM2_ESM.pdf (554 kb)
Supplementary figure 2 Tutu Ultrasound evaluation 3 months after treatment. (PDF 554 kb)
12015_2016_9659_MOESM3_ESM.docx (1 mb)
Supplementary figure 3 Smart x-ray evaluation 3 months after treatment. (DOCX 1044 kb)
12015_2016_9659_MOESM4_ESM.docx (1 mb)
Supplementary figure 4 Tutu x-ray evaluation 3 months after treatment. (DOCX 1038 kb)
12015_2016_9659_MOESM5_ESM.pdf (505 kb)
Supplementary figure 5 Yuan Ultrasound evaluation 61/2 years after treatment. (PDF 504 kb)
12015_2016_9659_MOESM6_ESM.docx (867 kb)
Supplementary figure 6 Yuan x-ray evaluation 61/2 years after treatment. (DOCX 866 kb)
12015_2016_9659_MOESM7_ESM.pdf (527 kb)
Supplementary figure 7 Rum Ultrasound evaluation 1 year after treatment. (PDF 526 kb)
12015_2016_9659_MOESM8_ESM.docx (1.2 mb)
Supplementary figure 8 Rum x-ray evaluation. (DOCX 1220 kb)
12015_2016_9659_MOESM9_ESM.pdf (441 kb)
Supplementary table 1 Cardiac evaluations of Sushi (normal dog), Smart and Tutu before and after treatment. Cardiac data include electrocardiography, mean arterial pressure and echocardiography. (PDF 441 kb)
12015_2016_9659_MOESM10_ESM.pdf (291 kb)
Supplementary table 2 Complete blood test, including blood count and biochemical blood test of Sushi (normal dog), Smart and Tutu. Blood was collected before and 48 h after each stem cell injection. Presented data show minimum and maximum values observed in all 18 blood tests. (PDF 290 kb)
12015_2016_9659_MOESM11_ESM.pdf (201 kb)
Supplementary table 3 Last complete blood test of Sushi (normal dog), Smart, Tutu, Yuan and Rum. Evaluation was performed 6 month after Tutu and Smart treatment; 10 months after Rum treatment; and 61/2year after Yuan treatment. (PDF 200 kb)


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Copyright information

© Springer Science+Business Media New York 2016

Authors and Affiliations

  • M. V. Pelatti
    • 1
  • J. P. A. Gomes
    • 1
  • N. M. S. Vieira
    • 1
  • E. Cangussu
    • 1
  • V. Landini
    • 1
  • T. Andrade
    • 1
  • M. Sartori
    • 1
  • L. Petrus
    • 1
  • Mayana Zatz
    • 1
    Email author
  1. 1.Human Genome and Stem-cell Research Center, Institute of BiosciencesUniversity of São PauloSão PauloBrasil

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