Gene Editing

“Even if an agreement is settled in the future, some believe an ethical consensus will be overridden by economic issues.”

by Cheryl Mai

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[dropcap]W[/dropcap]hat if you could buy designer genes for your children? With a gene editing technique called CRISPR, this concept may become a reality in the not-so-distant future. Introduced in 2012, CRISPR (clustered regularly-interspaced short palindromic repeats) technology has completely transformed the field of biology, surpassing older gene editing techniques in efficiency, accessibility and potential.

The boundaries of progress are becoming less defined and the finish line fuzzier. Just last April, Chinese researchers claimed to have used Cas9, a CRISPR-associated DNA-cutting enzyme, to modify a gene in non-viable human embryos. Published in Protein & Cell, the studies yielded cautionary results: after researchers injected CRISPR/Cas9 complexes into embryos to edit a gene encoding a human protein, only a small fraction of the embryos had their genes successfully edited. The rest did not survive or were mutated incorrectly. This study has helped rekindle a long-simmering ethical debate on tampering with the human genome.

To address these growing concerns, the U.S. National Academies of Sciences and Medicine, the Chinese Academy of Sciences, and the U.K.’s Royal Society co-hosted the International Summit on Human Gene Editing in Washington, D.C., in December 2015 on “recent scientific developments in human gene editing and the range of ethical and governance issues associated with these advances.” The conference brought together nearly 500 scientists, philosophers, bioethicists, and legal experts from over 20 countries to discuss any limitations on further research that were appropriate to take. Germline editing – in which embryos are genetically modified – was at the forefront of the meeting.

Jennifer Doudna of UC Berkeley and Emmanuelle Charpentier of the Max Planck Institute discovered the CRISPR technique. The precise targeting mechanism relies on a synthetic piece of genetic material that guides Cas9 to the complementary sequence of DNA that is to be cut or swapped out. Now the CRISPR/Cas9 system can deliver any gene into any site within an organism’s genome.    

CRISPR/Cas9 research is advancing at an astounding speed. In 2012, 126 papers were published on CRISPR. In 2015, 1265. From gene-editing biotech company startups worth billions to controversial patent disputes, scientists from around the world are racing to make the next major breakthrough with this new technology. Edita, a startup backed by Bill Gates, plans on using CRISPR on humans by 2017 to treat a rare eye disorder.    

Yet the summit statement released at the end of the three-day conference shows that scientists and ethicists alike are still far from reaching a universal agreement on the uses and boundaries of human gene-editing. Without explicitly banning the process, the statement declares the clinical use of germline editing – for now – to be “irresponsible.” The wide range of ethical beliefs among scientists of differing cultural backgrounds, in part, contributes to the agreement’s ambiguity.

In the US, federal funding is unavailable for research in which embryos are created or destroyed. Meanwhile, in China, there are no such restrictions. In the US, abortion and human rights before birth are hotly debated topics. “What spooks people out is that you are playing God,” said Shelly Kagan, a professor of philosophy at Yale. In China, there is little to discuss.

With such differing cultural mindsets between just two countries, is it possible for scientists to reach an ethical consensus across over twenty countries? And even if an agreement is settled in the future, some believe an ethical consensus will be overridden by economic issues. “What seemed like a moral or technical issue in the past is – in this society – very likely to become a consumer question of who can afford it,” said Daniel Kevles, a speaker at the summit.

The commercial potential of CRISPR is so compelling that it becomes likely that a ban on embryonic research will not bar the richest of the rich from buying genetic perks. Ironically, this in itself is a complex ethical concern. “The technology changes the method but not the issues,” said Kagan.

Cheryl Mai is a freshman Molecular Biophysics and Biochemistry major in Davenport College. Contact her at cheryl.mai@yale.edu.