Differential Requirement for Utrophin in the Induced Pluripotent Stem Cell Correction of Muscle versus Fat in Muscular Dystrophy Mice
2011

iPSC-Correction of Muscular Dystrophy Mice

Sample size: 14 publication 10 minutes Evidence: moderate

Author Information

Author(s): Beck Amanda J., Vitale Joseph M., Zhao Qingshi, Schneider Joel S., Chang Corey, Altaf Aneela, Michaels Jennifer, Bhaumik Mantu, Grange Robert, Fraidenraich Diego

Primary Institution: University of Medicine and Dentistry of New Jersey

Hypothesis

Can induced pluripotent stem cells (iPSCs) correct muscular dystrophy in mouse models?

Conclusion

The study found that iPSCs can correct muscle pathology in mdx mice but not in mdx∶utrophin mice, while also restoring fat mass in both models.

Supporting Evidence

  • iPSCs were injected into mdx and mdx∶utrophin mutant blastocysts to create chimeric mice.
  • iPSCs supplied dystrophin to the muscle sarcolemma in mdx chimeras.
  • Fat mass was restored in both mdx and mdx∶utrophin chimeras after iPSC injection.
  • Functional tests showed improved muscle performance in WT/mdx mice.
  • Histological analysis revealed improved muscle architecture in WT/mdx but not in WT/mdx∶utrophin mice.

Takeaway

Scientists used special cells called iPSCs to help fix muscles in mice with a disease, but they found that these cells worked better when another protein was present.

Methodology

Mice were injected with WT mouse iPSCs at the blastocyst stage, and the effects on muscle and fat were analyzed.

Potential Biases

Potential bias in interpreting the effects of iPSCs due to the specific genetic backgrounds of the mouse models used.

Limitations

The study primarily focused on mouse models, which may not fully replicate human conditions.

Participant Demographics

Mice used were mdx and mdx∶utrophin mutants.

Statistical Information

P-Value

p<0.05

Statistical Significance

p<0.05

Digital Object Identifier (DOI)

10.1371/journal.pone.0020065

Want to read the original?

Access the complete publication on the publisher's website

View Original Publication