By Julie Nixon
Earlier this month the Broad Institute of MIT and Harvard announced that it had been granted a patent covering the components and methodology for CRISPR, the acronym for Clustered Regularly Interspaced Short Palindromic Repeats. This technique may allow scientists to make specific changes to the genomes of a cell or an organism, and might possibly be a way to treat diseases with a genetic basis. However it is unclear as yet what researchers will and won’t be allowed to do with this revolutionary method for engineering genomes.
The science part…….
Short palindromic repeats of DNA are found in the genomes of bacteria. Until recently they were described as “junk DNA”, that is non-coding DNA with no apparent purpose for the organism. However recently it has been found that the palindromic DNA sequences provide the bacteria with a “memory” of viruses which the bacteria have been exposed to. The bacteria use the CRISPR sequences to recognise the same viruses that subsequently invade the bacteria and the viral target DNA will then be cut up with the bacteria's CRISPR-associated (CAS) enzymes.
The ability to treat genetic defects in humans has long been a goal for scientists. Inherited diseases such as cystic fibrosis, sickle-cell anemia and Huntington's disease are caused by single base pair mutations, and the CRISPR/Cas technology has the potential to correct these errors using “guide RNAS” to target the mutations. There is also the potential for germ-line therapy, such as the prospect of correcting defective genes in IVF embryos prior to implantation.
CRISPR Patent and IP implications
The CRISPR patent has been issued six months after its application was filed. The patent covers a modified version of the CRISPR system found naturally in bacteria, and also covers methods for designing and using CRISPR’s molecular components.
However many people are already using CRISPR and it’s not clear if it will now become harder to access the technology. It will inevitably come down to the MIT and the Broad, and a spokesperson for the Broad has stated “specific details around licensing aren’t available at this time, but the Broad does intend to make this technology broadly available to scientists.”
Whatever the outcome, the debate will remain about whether such revolutionary technology should be patented in the first place. Universities often patent the results of research to provide the incentive to develop the research into a viable therapeutic. Developing medical treatments is an immensely expensive process and having a patent in place can attract investment that might otherwise not happen.
Within Europe there are provisions which would currently exclude patents on correcting gene defects in IVF embryos. While the application of the CRISPR technology to humans is well regulated in Europe and the USA, this is not true for all jurisdictions. The ethical issues surrounding the application of CRISPR in producing “designer babies” is highly controversial, but if there is genuine scope to cure inherited diseases there may be pressure for change. The UK is already moving towards permitting mitochondrial replacement, a variation on IVF that uses a donor egg cell with healthy mitochondria into which the nuclear DNA from a mother with defective mitochondria is inserted. Technically any resulting offspring will have DNA inherited from three individuals, but the child will have no disabilities inherited from defective mitochondria.
Of course we are some way off from finding out if the CRISPR technology fulfils the expectation of providing the “genetic surgery” to cure genetic diseases, but we can be sure there will be further debate on the surrounding ethical and legal issues.