Method of cell fusion to incorporate corrective factors into any cell
Novel gene used in conjunction with another annotated gene fusing two cells types. Use for gene delivery to correct mutation and disease process.
Dr. Millay has discovered a novel gene which has been named myomerger. Used in conjunction with another annotated gene, myomaker, fusion of any two cell types results. Fibroblasts expressing the two genes were shown to have fused. This is the first time two mammalian genes have been shown to confer fusogenic activity to cells that normally do not fuse. These two genes (myomaker and myomerger) could be utilized in vivo to fuse any two cell types to correct diseases. The possibilities include fusing iPSC-derived cardiomyocytes into an injured heart, enhancing engraftment of cells into muscle, liver, neurons, or essentially any other tissue. Alternatively, this technology could be used for tissue repair in vivo, such as with iPSCs or ES cells, bypassing many of the problems that normally afflict these cells in standard cell therapy based treatments. This is currently being tested to fuse muscle cells to result in dystrophin expression to treat Duchenne's muscular dystrophy.
- Correcting mutations or disease processes
- In vivo tissue repair
- Fusing cardiomycytes into an injured heart
- Enhancing engraftment of cells into muscle, liver, neurons and other tissues
There are more than 30 forms of muscular dystrophy, Duchenne is just one of them. Duchenne muscular dystrophy occurs in 1 in 3,500 to 5,000 males born worldwide. The time from initial symptoms to diagnosis is about two and a half years, the average age of diagnosis being 5 years of age. More than 90% of children are in wheelchairs by age 15.
Douglas Millay, PhD - Assistant Professor, UC Department of Pediatrics