WASHINGTON: Scientists at the Johns Hopkins University in the US have shown that a powerful "genome editing" technology known as CRISPR can alter human stem cells precisely and efficiently.
The findings could speed efforts to modify human-induced pluripotent stem cells (iPSCs) for use as treatments or in the development of model systems to study diseases and test drugs.
"Stem cell technology is quickly advancing and we think that the days when we can use iPSCs for human therapy aren't that far away," said Zhaohui Ye from the Johns Hopkins University's School of Medicine.
CRISPR has been used by researchers since 2012 to trim, disrupt, replace or add to sequences of an organism's DNA.
Previous research has shown that CRISPR can generate genomic changes or mutations through these interventions far more efficiently than other gene editing techniques such as TALEN (transcription activator-like effector nuclease).
For the new study, Cheng and the team pitted CRISPR against TALEN in human iPSCs, adult cells re-programmed to act like embryonic stem cells.
Human iPSCs have already shown enormous promise with regard to treating and studying disease.
The comparison found that CRISPR system was significantly more efficient than TALEN.
The researchers found that the CRISPR system has an advantage over TALEN: It can be designed to target only the mutation-containing gene without affecting the healthy gene in patients.
"CRISPR-mediated genome editing opens the door to many genetic applications in biologically relevant cells that can lead to better understanding of and potential cures for human diseases," added Linzhao Cheng, professor at the Johns Hopkins University.
The report appeared in the journal Molecular Therapy.