Johns Hopkins stem cell team tests out CRISPR tech, with encouraging results

CRISPR technology's potential for gene editing has helped inspire the launch of a trio of closely watched biotech startups with their sights set on some cutting-edge approaches to new therapeutics. And now a team at Johns Hopkins has done some experiments to demonstrate its promise in engineering human stem cell therapies.

Zhaohui Ye, Linzhao Cheng and colleagues were particularly interested in seeing if CRISPR tech would trigger some unwanted effects on cancer stem cell lines similar to what's been seen in off-target genes in other editing research efforts. And they compared the editing techniques involved with CRISPR against TALEN--transcription activator-like effector nuclease--on induced pluripotent stem cells (iPSCs), adult cells reprogrammed to act like embryonic stem cells, by surgically removing and replacing pieces of known genes.

They focused on three genes: JAK2, SERPINA1 and AAVS1, a "safe harbor" used to stitch in foreign genes.

Their conclusion: CRISPR was far better at making the surgical cuts that were needed to remove segments of genes, but CRISPR and TALEN were similar when it comes to replacing the segments with new bits of genes. And CRISPR was better than TALEN when it comes to zeroing in on a specific gene mutation when only one copy of the gene is involved. Healthy, off-target genes were left unmolested.

"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," says Cheng in a statement. And the work is also relevant to stem cell projects that can be used in drug testing.

"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," says Ye, an instructor of medicine at the Johns Hopkins University School of Medicine. "This is one of the first studies to detail the use of CRISPR in human iPSCs, showcasing its potential in these cells."

- here's the release