In a review at last year’s NASPGHAN meeting, John Barnard gave a basic science review (Basic Science Year in Review -#NASPGHAN 2014 | gutsandgrowth) that touched on CRISPR-Cas9 for genome engineering (Cell 2014; 157: 1262-78). Reading through a recent editorial (Lander ES. NEJM 2015; 373: 5-7), it seems that the potential for genome editing is not that far from landing into clinical use.
Genome editing holds great therapeutic promise…
- “physicians might edit a patient’s immune cells to delete the CCR5 gene, conferring the resistance to HIV carried by the 1% of the U.S. population.”
- “Editing blood stem cells might cure sickle cell anemia and hemophilia.”
- Eliminate genes which increase the risk for Alzheimer’s, Huntingdon’s disease and heart attacks
- “Genetically modified humans” and true “designer babies”
- Technical issues to perform editing with precision.
- Unanticipated effects with various edits. “We remain terrible at predicting the consequences of even simple genetic modification.”
- Who decides? Future generations cannot consent to their modification.
- Is it morally right? “Would the ‘best’ genomes go to the most privileged?”
In the U.S., genome editing would not garner approval from FDA or NIH in the near future. But, given the advancing technical capabilities, it is not too early to begin the discussion about genome editing. At the very least, this technology should spurn a couple great sci-fi movies.
Take-home point: “Authorizing scientists to make permanent changes to the DNA of our species is a decision that should require broad societal understanding and consent…We should exercise great caution before we rewrite” the human genome.