Low-Dose Irradiation to Edit The Genome of Patients’ Stem Cells

In a recent study published in the journal Stem Cells Translational Medicine, a team of researchers from the Cedars-Sinai Board of Governors Regenerative Medicine Institute have employed a gene-editing method that uses low-dose irradiation to edit the genome of patients’ stem cells.

According to the study results, this strategy is 10 times more effective than currently used techniques. “This novel technique allows for far more efficient gene editing of stem cells and will increase the speed of new discoveries in the field,” said co-senior author Clive Svendsen, PhD, director of the Board of Governors Regenerative Medicine Institute.

The irradiation strategy could be successful to increase the understanding of certain conditions including muscular dystrophy, spinal muscular atrophy, and Huntington’s disease.

Genome editing, is a type of genetic engineering in which DNA is inserted, replaced, or removed from a genome allowying researchers to correct mutations causing diseases. The technique also allows researchers to create mutations in normal cells that can then be used to model human malignancies.

In the study titled “Low-Dose Irradiation Enhances Gene Targeting in Human Pluripotent Stem Cells”, researchers demonstrate that this form of gene editing is more efficient in inserting reporter genes that glow when a stem cell turns into a specific cell of the body. “The combination of low-dose irradiation and correct gene copy will accelerate our ability to model human disease using stem cells from patients with many different disorders,” said co-senior author Vaithilingaraja Arumugaswami, MVSc, PhD, director of the Pancreas and Liver Program in the Cedars-Sinai Board of Governors Regenerative Medicine Institute.

Creating human diseases in a petri dish using stem cells is a growing field of expertise. This line of research enables scientist to test new drug agents on human cells carrying genetic mutations that cause diseases.

“Radiation, which is normally considered harmful, has proven beneficial in gene editing,” said Svendsen. This new technique will help us establish far more accurate models and accelerate the discovery process.”