Cell Reprogramming-based Therapies for Muscular Dystrophies

With a best-in-class technology platform, Creative Bioarray focuses on advances in cell reprogramming and gene editing and is committed to helping our clients develop new therapies based on iPSC for the treatment of muscular dystrophies (MDs). Our efficient technical support and high quality scientific services will greatly accelerate the process of your projects.


MDs are a range of muscle diseases of varying severity caused by multiple genetic defects, such as Becker (BMD), limb-girdle (LGMD), myotonic, and Duchenne Muscular Dystrophy (DMD). These heterogeneous skeletal muscle disorders are usually accompanied by progressive muscle atrophy, formation of adipose and fibrous tissue in the muscle, even leading to death from cardiomyopathy and respiratory failure in severe cases. Unfortunately, there is no effective cure for MDs.

The development of iPSC technology has opened up new avenues for personalized treatment and has facilitated the development of new therapies for several degenerative diseases such as MDs. iPSC technology allows the generation of an unlimited number of specific cell populations for regenerative purposes. In addition, patient-specific iPSCs can theoretically avoid immune rejection. Researchers have now performed cell transplantation in several mouse models of MDs. Experiments have shown that these cells can fuse to host muscle fibers and exhibit good strength. Several recent studies have demonstrated that iPSC offers promising predictions for future clinical trials for the treatment of MDs.

Fig 1. PSC-derived myogenic progenitor cells can be used to treat MD patients.Fig.1 PSC-derived myogenic progenitor cells can be used to treat MD patients. (Selvaraj, 2019)

Development of New Therapies for Muscular Dystrophies based on Cell Reprogramming

Cell Reprogramming-based Therapies for Muscular Dystrophies

Researchers at Creative Bioarray are working to combine iPSC technology with gene-editing techniques to help clients develop effective therapies for muscular dystrophies. To overcome the problem that myogenic cells from these iPSCs may undergo a degenerative process after transplantation, we offer solutions to correct mutations to restore the expression of lost proteins for cell transplantation.

  • Genomic correction of autosomal recessive point mutated genes
  • Genomic correction of autosomal dominant myotonic dystrophy
  • Genomic correction of X-linked BMD/DMD

In addition, we offer our clients the following methods to validate the effectiveness of genomic correction strategies for iPSCs derived from MD patients:

  • Applying exon skipping to reconstruct truncated forms of the dystrophin gene.
  • Using site-specific gene-editing techniques to correct mutations.
  • Seamless allele-specific correction strategies based on a single-stranded oligonucleotide CRISPR/Cas9-mediated approach.
  • Gene correction evaluation in DMD iPSCs and mice models using an updated version of the gene-editing system, CRISPR/Cpf1.


  • Advanced technology platform
  • PhD-level experienced technical staff
  • Flexible solutions
  • Customer-focused services

As a leader in cell reprogramming, Creative Bioarray is well-positioned to provide efficient and professional solutions to our clients in developing new approaches to treat MDs. If you need our scientific help, please contact us for more information.


  1. Selvaraj, S.; et al. Pluripotent stem cell-based therapeutics for muscular dystrophies. Trends in molecular medicine. 2019, 25(9): 803-816.
For Research Use Only. Not For Clinical Use.