On March 21, 1919, the novelties were published on the “in vitro” works in patient cells (fibroblasts), using the “pseudo” exon skipping technique. Among the authors, there are the most important experts on Collagen VI worldwide, such as Dr. Bönnemann and Dr. Muntoni, leaders of this research.
In this article, they demonstrate a high efficiency in the corregimiento of the Protein of Collagen VI mutated and consequently, corregimiento also of the extracellular matrix that should form Collagen VI. This was achieved by administration of antisense oligonucleotides and also by means of CRISPR.
This publication is a sample of the advances that are being made and the good results in the pre-clinical phase. No doubt this technique continues to maintain a high potential to become a real therapy. Much still remains, but we are on the right track.