Sentences with phrase «functional dystrophin»

The technique partially restored not only functional dystrophin expression, but also dystrophic muscle function.

In a subsequent study, they used CRISPR with two gRNAs to delete exon 51 or exons 45 - 55 in patient myoblasts; when injected into DMD mice, these cells expressed functional dystrophin.

Gersbach and his team first delivered the therapy directly to a leg muscle in an adult mouse, resulting in the restoration of functional dystrophin and an increase in muscle strength.

Not exact matches

The result was the largest deletion ever observed in the dystrophin gene using CRISPR / Cas9, and the study was the first to create corrected human iPS cells that could directly restore functional muscle tissue affected by Duchenne.

In the current study, Dr. Rudnicki and his team discovered that muscle stem cells also express the dystrophin protein, and without this protein, they produce ten-fold fewer muscle precursor cells, which in - turn generate fewer functional muscle fibres.

«This is the first study to demonstrate that functional muscle cells can be created in a laboratory and restore dystrophin in animal models of Duchenne using the human development process as a guide.»

This approach has proved successful in disrupting multiple genes simultaneously in neurons and in editing the Dmdmdx mutation in muscle, thereby restoring dystrophin protein expression and partially alleviating the dystrophic muscles» functional deficiencies.

Within the mutational hotspot for dystrophin, exons 45 - 55, there are multiple common deletions that maintain the protein's reading frame, leading to the production of a smaller, but at least partially functional protein.