Improvement on the accuracy of gene editing in the treatment for Epidermolysis bullosa

24 May, 2021

  • Epidermolysis bullosa or Butterfly Skin is characterised by the extreme fragility of the skin and carries a high risk of aggressive skin cancers for patients.
  • Scientists from CIEMAT, UCSM and CIBERER in collaboration with the institute for research at the Jimenez Dias Foundation and the University of Stanford in the U.S have managed to increase the levels of accuracy in gene editing technology for the treatment of this disease.
  • Spanish scientists have increased the levels of accuracy and precision in the technology involved in gene editing for the treatment of dystrophic recessive Epidermolysis bullosa, which opens the door to further future clinical trials.

 

Dystrophic Recessive EB is characterised by the extreme fragility of the skin and mucous membranes as well as a high risk of aggressive skin cancer.  This hereditary condition is caused by mutations in the collagen gene, where the skin is lacking the essential protein that binds the skin together.

In a series of previous investigations, the Spanish research group have already achieved notable scientific advances in the efficacy of gene editing: reaching a point of being able to establish the correct levels of collagen production necessary for realistic clinical trials in adult stem cells.

Precision correction of the gene that causes the disease

In this ground-breaking work published in the Molecular Therapy magazine, they describe the new advances in the technology of gene editing for the correction of the mutations responsible for dystrophic recessive Epidermolysis bullosa. These developments are now patent pending.

The promising results of the gene editing research now pave the way for further clinical trials in this area.

 

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