Editing genes with care and confidence

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By National Human Genome Research Institute (NHGRI) from Bethesda, MD, USA (CRISPR-Cas9 Editing of the Genome) [CC BY 2.0 (https://creativecommons.org/licenses/by/2.0)], via Wikimedia Commons

A pair of scientific papers published recently revealed that editing the genome with the popular molecular tool called CRISPR may cause cancer. CRISPR involves making precise ‘cuts’ in the genetic code, which some cells may perceive as damage with the potential to result in harmful mutations. In its defense, the cell may activate anti-cancer pathways to combat the perceived attack and cause the cell to die – rendering the CRISPR edit useless. The papers published this week demonstrated that certain cells are able to survive editing by CRISPR but lack a functional anti-cancer pathway, leaving them vulnerable.

Some commentators suggest these revelations spell doom for CRISPR and consider whether it should ever be used in human cells. But many news reports exaggerate the implications of these papers, as stated by one contributing author. And the scientists, legislators, farmers, and social activists at this month’s CRISPRcon delivered a clear message: precision gene editing is happening, and it can happen in a socially and ethically responsible way.

I am a 22-year-old, admittedly optimistic, recent college graduate with a degree in molecular biophysics and biochemistry. As part of the generation that will be responsible for continuing medical science’s pursuit of a healthier life for all, I’m confident precision genome editing will be one of our strongest tools in the lab and potentially the clinic. I want to ensure each step is as measured and thoughtful as possible.

CRISPR, short for Clustered Regularly Interspersed Short Palindromic Repeats, makes it possible to change features encoded in an organism’s DNA. Imagine DNA is the blueprint for building a human, and CRISPR is a tool capable of erasing and rewriting those detailed instructions to a reasonable degree. This means scientists can replace problematic regions of DNA, revising areas of vulnerability and potentially curing genetic diseases before they manifest.

The more immediate appeal of CRISPR is its capacity to rapidly increase the pace of discovery. CRISPR has allowed labs across the world to dramatically shorten the length of time required to genetically engineer lab mice and cells – a process that often becomes the rate-limiting step in designing new biological models designed to understand disease.

And while there are valid concerns about the ethics and practicalities of introducing CRISPR into cells destined for the human body, we can address them with the same careful processes that have driven our greatest medical advancements for decades.

We’ve been here before.

When scientists first explored the possibility of in vitro fertilization, concerns regarding the legitimacy of “test-tube” babies consumed the public. Perhaps the near-simultaneous release of Aldous Huxley’s “Brave New World” in 1932 and Harvard scientist Gregory Pincus’ early IVF research with rabbits in 1934 — which suggested IVF could work in humans — contributed to distorting public perception of this new technology. In fact, both Pope Pius XII and Nobel Laureate James Watson strongly opposed the new technology at the time.

It took scientists 44 years to achieve a successful birth with the first IVF baby, Louise Brown, in 1978. But during those 44 years, governments placed moratoriums and held advisory committee gatherings to discuss the practical and ethical possibilities of the technique. After 11 public hearings, the Ethical Advisory Board of the United States approved federal funding of IVF in 1979. The first clinics opened the following year, and by 2004 IVF was considered a mainstream technology for couples seeking infertility treatment.

Many would agree that the procedure still has significant kinks to work out, but consider this: today, approximately six million people on the planet began their lives this way.

This models the proper path forward for CRISPR. Research in the United States follows strict regulations and guidelines — reviewed and updated to ensure adherence to ethics and high quality — imposed and enforced by a multi-layer system of institutional review boards at universities, hospitals, and federal agencies. There are going to be mistakes, but eventually millions of lives will be changed for the better because we persisted despite the rocky path progress usually travels.

So let’s get this right. We must remove the mysterious shroud that obscures an understanding of medical research from much of the population. We cannot afford to have political posturing and misinformation push gene editing away from American institutions and towards more loosely regulated countries. We have an invaluable opportunity to show the world how best to implement and regulate such a mighty, beneficial tool and to model what it looks like to remain confident, but act carefully, when faced with such complicated scientific endeavors.

Haleigh Larson is a fellow with Women’s Health Research at Yale University.


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