Devangshu Datta: The ethics of editing genes

In theory, CRISPR can be used to edit out diseases such as diabetes

One of the most momentous patents in history was awarded recently and the US Patent and Trademark Office (USPTO) may actually have sided with the wrong scientific team. After a high-profile debate of conflicting claims, the USPTO confirmed a patent for the gene-editing technology, CRISPR (clustered regularly interspaced short palindromic repeats) CAS-9, awarded to Dr Feng Zhang of the Broad Institute, which is jointly owned by MIT and Harvard.  

While USPTO sided with the Broad Institute, the scientific community seems to think the competing team of Dr Jennifer Doudna and Dr Emmanuelle Charpentier at the University of California, Berkeley, have better claims. In early 2012, Dr Doudna and Dr Charpentier, (who is now with the Max Planck Institute, Berlin) published an earth-shaking study. They had developed a new gene-editing technology, CRISPR. CRISPR uses a “biological knife” (the CAS-9 enzyme) to literally cut and paste gene sequences.  

The duo applied for a broad patent in May 2012. In December 2012, Feng Zhang, a Chinese neuroscientist at the Broad Institute, applied for a narrower patent. Doudna-Charpentier claimed a patent to apply across all biological cells. They had started work on simple bacterial cells (prokaryotic cells) and later worked on more complex eukaryotic cells (of mammals and complex organisms). Zhang & Co only filed for patent for eukaryotic cells. 

The Broad Institute had filed seven months later, in December 2012, and Zhang published his key paper in early 2013. But the Broad Institute gamed the patent filing system better. By filing for a simpler patent and asking for accelerated process, Zhang was awarded the patent in May 2014. UC Berkeley disputed the award, which was confirmed this week. Three judges ruled that Zhang’s patent was different from the patent applied for by UC Berkeley. That Berkeley patent is still pending and if accepted, it may yet supersede the Broad Institute patent. UC Berkeley may also appeal. 

In the last five years, Doudna-Charpentier have won many major science prizes, even as the debate worked through the USPTO system. The duo won the $3-million Breakthrough Prize in Life Sciences in 2015, the $500,000 Gruber Genetics Prize in 2015, and the $450,000 Japan Prize in 2017. About the only thing they haven’t won yet is the Nobel and that is very much on the cards. 

CRISPR is one of the most important tools for bio-scientists. Using CRISPR, undesirable genes can be excised and desired genes inserted into a genome. This can be done with enormous accuracy and the traits inserted are inheritable. 

CRISPR already has many current applications. The potential applications are limitless. It has been used to breed mosquitoes that cannot transmit malaria, to edit out cancer cells, and to create disease-resistant plants and pest-resistant plants. It has also been used to edit monkey and beagle genomes, creating embryos carried to term in surrogate mothers. 

It could, in theory, be used to “edit out” diseases such as muscular dystrophy, diabetes or sickle-cell anaemia and research is proceeding along these lines.  In another intriguing experiment, researchers are trying to insert human genes in pigs to grow organs, which could be used for transplants. 

What is troubling is that CRISPR could also be used to create “superbabies” by inserting the right genes and imparting genetic abilities to develop fast twitch muscles or greater lung capacity, etc.  Experimenting on human genomes is illegal in some nations but not in others.  In any case, the legal hurdles will hardly stop the determined. In fact, many governments may throw resources into such research. 

These possibilities have sparked ethical debates.  Dr Doudna recently wrote an emotional open letter outlining her own doubts about the ethics. The US National Academy of Science (NAS) has also released a paper on human genome editing, which looks at research and regulatory guidelines. The paper supports gene editing to control and cure diseases while frowning upon using CRISPR for “enhancement”. But this blurs in practice. Editing out muscular dystrophy, for example, could create more muscular, “enhanced” babies and therefore, the NAS can’t provide definitive answers. 

The scientific papers have been in public domain for years. The technology has been used extensively round the world. Since US patents don’t have extraterritorial jurisdiction, there is nothing to stop researchers using CRISPR elsewhere, without licensing or oversight. Regardless of who owns patents, and regardless of licensing, CRISPR could have a bearing on the records in the 2040 Olympics.