In utero transplantation of myoblasts and adipose-derived mesenchymal stem cells was not successful and may result in fetal death in murine models of Duchenne muscular dystrophy (DMD), researchers reported in a study recently published in the journal Regenerative Therapy. 

Currently, the most widely used treatment for DMD consists of immunosuppressive therapy with corticosteroids. However, several forms of gene therapy have been recently made available and are showing promising results, but they are limited to patients with certain mutations.

Cell therapy, on the other hand, has the advantage of being applicable to all mutations. Some trials in human patients have shown delays in the decrease of muscular and cardiac function with cell therapy.

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One of the main obstacles regarding cell therapy is transplant rejection by immune cells. In utero cell transplantation has the advantage that there is practically no risk of immune rejection due to the naivety of fetal immune cells. Similar procedures have already been performed to treat conditions such as severe combined immune deficiency and osteogenesis imperfecta.

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The authors aimed to perform in utero fetal intraperitoneal transplantation and transplacental transplantation of green fluorescent protein (GFP)-positive myoblasts and adipose-derived mesenchymal stem cells in mice with DMD. Control models received normal saline to murine fetuses.

Myoblast intraperitoneal transplantation was performed in 22 mice, of which 9 were born after cesarean section. Of the 21 mice that received adipose-derived mesenchymal stem cells through intraperitoneal transplantation, only 1 survived until birth. Regarding transplacental transplantation, 7 fetuses received myoblasts, of which 6 survived. However, none of the 8 fetuses that received transplacental adipose-derived mesenchymal stem cells survived.

No GFP-positive cells were found in any of the surviving mice, no matter the transplantation method used.

“However, this does not mean that in utero cell transplantation of myoblasts or adipose-derived stem cells is not promising for DMD, since different route of administration is used in human in utero cell transplantation,” the authors wrote. 


Kihara Y, Tanaka Y, Ikeda M, et al. In utero transplantation of myoblasts and adipose-derived mesenchymal stem cells to murine models of Duchenne muscular dystrophy does not lead to engraftment and frequently results in fetal death. Regen Ther. Published online October 9, 2022. doi:10.1016/j.reth.2022.10.003