IS this the large animal study that muscle regeneration was waiting for

This paper is taking about long term dystrophin production in dystrophic dogs without immunosupression.


Looks to a very strong case and almost as strong as the exon skipping videos of improvement in dogs. Since its taking about dogs and not mice I am all ears if PPMD experts can comment on this.


I sincerly hope the paper does not have a final conclusion that there is lot more work to do






Long-term Engraftment of Multipotent Mesenchymal Stromal Cells That Differentiate to Form Myogenic Cells in Dogs With Duchenne Muscular Dystrophy

Yuko Nitahara-Kasahara, Hiromi Hayashita-Kinoh, Sachiko Ohshima-Hosoyama, Hironori Okada, Michiko Wada-Maeda, Akinori Nakamura, Takashi Okada and Shin'ichi Takeda

Duchenne muscular dystrophy (DMD) is an incurable genetic disease with early mortality. Multipotent mesenchymal stromal cells (MSCs) are of interest because of their ability to differentiate to form myogenic cells in situ. In the present study, methods were developed to expand cultures of MSCs and to promote the myogenic differentiation of these cells, which were then used in a new approach for the treatment of DMD. MSC cultures enriched in CD271+ cells grew better than CD271-depleted cultures. The transduction of CD271+ MSCs with MyoD caused myogenic differentiation in vitro and the formation of myotubes expressing late myogenic markers. CD271+ MSCs in the myogenic cell lineage transplanted into dog leukocyte antigen (DLA)-identical dogs formed clusters of muscle-like tissue. Intra-arterial injection of the CD271+ MSCs resulted in engraftment at the site of the cardiotoxin (CTX)-injured muscle. Dogs affected by X-linked muscular dystrophy in Japan (CXMDJ) treated with an intramuscular injection of CD271+ MSCs similarly developed muscle-like tissue within 8–12 weeks in the absence of immunosuppression. In the newly formed tissues, developmental myosin heavy chain (dMyHC) and dystrophin were upregulated. These findings demonstrate that a cell transplantation strategy using CD271+ MSCs may offer a promising treatment approach for patients with DMD.

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HI Tulika:  We have a report on that stem cell meeting that took place in New Orleans going out in the next e-newsletter (maybe on Monday?).  The Italians were represented at that meeting, but the study you mention, above, is a Brazilian group -- led by Dr. Mayana Zatz.  I am guessing that more will be presented at the World Muscle Society meeting in Perth, this Fall.  I'm not going to that meeting, but I think our Board Chair Bob McDonald may be going.




 A comprehensive view on muscle regeneration would be really great. Hope that the canine studies are as encouraging as the video on exon skipping.

On a similar note not sure if you noticed that Germany has approved a localized muscle regeneration trial. The company plans to market the stem cell product for sports injury but strangely does not mention dystrophy.

Germany Muscle regeneration

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