Recent monumental advances in gene-editing technology are rapidly turning what was once science fiction into science fact. In March 2017, the first report of gene-edited viable human embryos was published1, and genome-edited human babies could soon be a realistic possibility. While this brings obvious benefits, such as the elimination of inherited genetic diseases and significant advances in gene therapies, there are also important ethical issues that need to be considered.

The gene-editing technique causing all the excitement is called CRISPR, which is short for “Clustered Regularly Interspaced Short Palindromic Repeats”. As recently as 2013, CRISPR technology was named as one of Science magazine’s “Top 10 Breakthroughs” of the year and the amazing simplicity and efficiency of the technology has triggered an explosion of research.

The CRISPR system is derived from a simple immune/defence mechanism first discovered in prokaryotes. The system comprises an enzyme which can cut DNA at a specific predetermined location in the genome, together with an RNA molecule, which precisely guides the enzyme to the exact target DNA sequence. Different enzymes have been described for use in the system, including Cas9 and Cpf1, the latter being a more recent discovery that has the potential for even simpler and more precise gene editing than the former. Once the genome has been cut or “digested” at the target sequence, scientists can manipulate the cell’s DNA repair machinery to alter the genetic sequence at the cleavage site, resulting in a geneedited cell.

Since the first publication of CRISPR in 2012, it is estimated that a billion dollars has been invested in the technology by venture capitalists, pharmaceutical companies, and public stock offerings. Numerous patent applications have been filed by various different academic institutions and corporations all trying to protect their innovations. Unsurprisingly, because of the obvious potential  value of the technology, the first (of probably many) legal proceedings has recently been conducted at the U.S. Patent and Trademark Office (USPTO). The case was brought to determine the entitlement to patent, in the U.S., of the use of CRISPR in eukaryotic cells, including humans.

CRISPR was separately and concurrently developed by independent research groups based in the U.S. The first group led by Jennifer Doudna, of the University of California-Berkeley (UC), and Emmanuelle Charpentier, now at the Helmholtz Center for Infection Research in Germany, were the first to demonstrate the use of the CRISPR system to edit targeted DNA, in a cell-free system. However, the other participant in the race, Feng Zhang of the Broad Institute of MIT and Harvard, was the first to demonstrate that the CRISPR technology can be used to edit DNA in human cells.

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