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#apaperaday: Exploring the Therapeutic Potential of Ectoine in Duchenne Muscular Dystrophy

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Exploring the Therapeutic Potential of Ectoine in Duchenne Muscular Dystrophy: Comparison with Taurine, a Supplement with Known Beneficial Effects in the mdx Mouse

Today’s pick is from the international journal of molecular sciences by Merckx et al on the comparison of taurine and ectoine in mdx mice. I’m a bit confused by the findings and study set up. Doi 10.3390/ijms23179567

Duchenne is associated with chronic loss of muscle tissue due to loss of dystrophin resulting in inflammation, fibrosis formation and impaired regeneration. Taurine is an amino acid involved in stabilizing muscle fibers and also has anti inflammatory properties.

Treatment of mdx showed reduction in oxidative stress but also body weight. Ectoine is a compound that is similar to taurine in function. Authors here wanted to establish whether it was a better alternative than taurine.

First they tested this in vitro showing (their words) less cell death and inflammatory response after cytokine addition to their cell cultures when cells were pretreated with taurine or ectoine. I am confused because I see an increase in production of inflammatory cytokines…

by qPCR and for the cell death experiment the variability between untreated is large (lane 1 and lane 7), so I dare not draw any conclusions. Authors treated mdx mice with ectoine in drinking water or IP injections, compared to taurine in drinking water and untreated.

No wild type controls…also each group is a litter…Ectoine treated mice had lowest body weight which was unexpected. Authors discuss it could be a litter effect as these groups were from largest litters. It is known that large litter sizes (9 & 11 here) lead to lower body weights.

And this dear followers is why you randomize litters over groups…because now you cannot draw a conclusion. If you have a major treatment impact you will still find it. However, for minor things – like expected here – you now are left wondering: litter or treatment effect?