CRISPR is an innovation in genetic engineering expected to accelerate cures and therapies for gene-related problems.  Along the way, CRISPR will rekindle ethical controversies.  How the US handles these controversies will be key to harvesting the benefits of genetic engineering.

CRISPR allows scientists to precisely cut out and replace DNA in genes.  It has proven to be a faster and more precise approach for genetic modification in animals and crops.  The CRISPR acronym stands for “clustered regularly interspaced short palindromic repeats” describing a natural defense system that bacteria use to fend off harmful infections.

Researchers target the segment of a genome that is to be cut out or replaced by using a strip of RNA and Cas9, an enzyme.  The RNA guide positions the Cas9 which then cuts the DNA to deactivate the unwanted section.  The success rate of these gene-level surgeries is continually being improved and those improvements will result in new patents.

CRISPR-Cas9 patents held by the MIT/Harvard Broad Institute were awarded on a “first to invent” basis. Those patents are disputed by the University of California on grounds that it was “first to file.” “First to invent” was the earlier standard for patent award and “first to file“ is the current standard.

The research at each university was funded by public grants and private donations, but since the parties are unwilling to negotiate a compromise we can expect massive litigation fees and delays to fritter away the first movers’ advantages.

Genetic engineering will continue at a faster pace because of CRISPR and its successors. New crop and food animal variants with better parasite, insect and disease resistance will be developed. New treatments for hereditary human diseases are expected from genetic engineering supported by CRISPR. While many of us welcome this progress, some are not so sure.

Only some of the human genes on our chromosomes are identified and there are vast stretches of chromosomes that are underexplored. CRISPR may accelerate the massive work needed to map our genome segments to humans’ physical characteristics.

We can count on the anti-GMO stalwarts to raise a loud ruckus about fish, cattle, chickens, grains and garden vegetable improvements that were genetically engineered. They will want to ban anything improved by GMO techniques or at least have them labelled as vaguely satanic.

Genetic engineering to prevent human and animal disease may get a pass, but we can expect vigorous debate on the ethics of genetic engineering, if the goal is to produce humans with superior attributes – e.g. intelligence, disease resistance, muscle or lifespan. Opposition may be styled as preventing lethal genes from being released into the wild, but the underlying motive for many will be religious objections to tinkering with God’s work.

In Italy, all human embryo research is banned. In the UK, gene editing was permitted to study embryo development. The US has banned federal funding for human embryo editing, but it does not regulate research done with private money. In 1990, the FDA approved genetic screening of embryos to weed out the mutation that causes cystic fibrosis. That opened the door to other screenings. Now, doctors can screen for anything, from predisposition to cancer to a baby’s gender. Some countries will excel in genetic research precisely because their government does not ban it.

The cost of genetic health fixes for human patients is yet unknown, but we can assume it will be large. The US health care burden is already in the $3 trillion range, so pragmatism dictates that we cannot afford “nice to have” treatments. On the other hand, genetic engineered treatments may provide an overall lower cost for some diseases or conditions than would conventional treatment.

Provided the FDA and other regulators practice oversight tempered with humility, we can expect the practice of genetic engineering to progress quickly. That should offer new hope to many patients.