Muscles from mice treated with modified human stem cells show
human dystrophin - producing muscle fibers (yellow) integrated among mouse muscle fibers (red).
Skeletal muscle cells isolated using the ERBB3 and NGFR surface markers (right) restore
human dystrophin (green) after transplantation significantly greater than previous methods (left).
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.
12 guide RNAs developed to find mutation «hotspots» along the
dystrophin gene helped rescue cardiac function to near - normal levels in
human heart muscle tissue.
Scientists say the new strategy enhances the accuracy for surgical - like editing of the
human genome, correcting mistakes in the DNA sequence that cause devastating diseases like DMD, a deadly condition caused by defects in the
dystrophin gene.
Using the natural
human development process as a guide, the researchers developed ways to mature muscle cells in the laboratory to create muscle fibers that restore
dystrophin, the protein that is missing in the muscles of boys with Duchenne.
«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.»
Researchers at the Center, co-directed by Drs. Olson and Mammen, have successfully edited mutations in the X-linked DMD gene that encodes the protein
dystrophin in
human cells.
It is essential the findings from the study in the
dystrophin - deficient mice are confirmed in
human DMD patients before its potential applications in these patients can be considered.