For the first time, researchers have employed a gene-editing technique involving low-dose irradiation to repair patient cells, according to a study published in the journal Stem Cells Translational Medicine. This method is 10 times more effective than techniques currently in use.
The irradiation method could prove effective in learning more about diseases such as spinal muscular atrophy, muscular dystrophy and Huntington's disease. Gene editing allows scientists to correct irregular mutations and, theoretically, cure the disease in the petri dish. Additionally, gene-editing technology allows scientists to create disease mutations in normal cells, thus modeling human disease.
When using this form of gene editing, scientists can more efficiently insert reporter genes that glow when a stem cell turns into a specific cell of the body. For example, stem cells would turn green when converted into a heart cell and red when turned into a neuron.
Over the past few years, the field of creating human diseases in the dish using stem cells has expanded rapidly. This work allows scientists to test novel drugs on human cells that carry disease-causing genes.
Based on material originally posted by Cedars-Sinai Medical Center.