CRISPR, Cures, and the New World of Gene Editing

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On March 7 of this year, NPR shared the story of Victoria Gray and her visit to a scientific summit in London, England.¹ Gray lives with sickle cell disease (SCD), a genetic condition that causes the blood cells responsible for transporting oxygen to become misshapen. Those living with sickle cell can suffer episodes of pain crises, as many as four or more a year, when the sickled cells clog blood flow causing intense pain and organ damage. The accumulation of that damage shortens the lives of those living with SCD, and the pain episodes limit their abilities to enjoy aspects of life many of the rest of us take for granted. Gray was invited to this summit to give a brief presentation because after a lifetime of struggle, she now lives as if she were, for all intent and purposes, cured. That cure is but a small part of the ongoing story of the CRISPR revolution.

Biotechnology known by the acronym CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) took the expensive and complicated world of gene editing and transformed it into a more accessible field. Through observation of the ongoing war between bacteria and viruses, CRISPR scientists discovered a bacterial defense mechanism built around identifying and splicing genetic information in viruses and transformed that bacterial defense into a tool that promises medical miracles, cures, and the ability to edit DNA in areas as diverse as food production and cancer treatment.²

Some worry intellectual and scientific advancement threatens to be divisive if treatment costs and not medical need determines who receives care. A long and expensive process transformed Ms. Gray’s life, so what does that mean for the other 100,000 or so Americans living with SCD, or the millions worldwide?³ Sickle cell disease strikes largely sub-Saharan populations in Africa, Mediterranean populations, and the Black community in the United States. As expensive treatments are created, how do we ensure the communities most in need of treatment enjoy the benefits of the scientific breakthroughs?

The promise of CRISPR, like all medical treatments, comes with built in risks. Copying and editing errors may be a source of mild embarrassment for those who trade in the written word, but so-called off-target edits in our genetic makeup could have devastating consequences. After all, the difference between living with a debilitating chronic condition and living a healthy fulfilling life can sometimes come down to one single base pair within a massive DNA sequence. People identifying themselves as biohackers, like computer hackers but focusing on genetic code, represent a different type of risk, as they look to opensource the advancement of gene editing and gene control — a move that could both rapidly increase the rate of discovery and encourage reckless unscientific practices.

Finally, how do Christians spiritually navigate a world advancing aggressively in the ability to alter the building blocks of life, for good or bad, in areas like CRIPSR research? An apparent lack of consideration for early human life fosters a distrust of the scientific community within many people who identify themselves as Christians, especially Evangelicals. This frustration will not deter advancement. It is incumbent on Christians to reflect deeply on what it means to be a human being in an age when some scientists actively seek to fundamentally alter the human experience or even leave it behind altogether. The Bible tells us that God intended the creation of mankind as a good thing. CRISPR promises to restore many in the human family to natural capacities hindered or lost through disease and genetic adversity. Fellow image bearers of God will be given the opportunity to flourish as God intended. But the pursuit of that flourishing must not lead to commodifying human life. The ability to heal must not create a rubric through which we judge and diminish the value of those human lives still suffering.

CRISPR

In 2012, a research team led by Jennifer Doudna and Emmanuelle Charpentier published an article in the peer-reviewed journal Science, detailing and refining the components of CRISPR to facilitate precise and accessible gene editing. In the ten years prior, scientists from various fields sought a better understanding of a phenomenon initially observed as an odd repeating of palindromic sequences within the genome of an E. coli bacteria. These sequences, ultimately observed in other bacteria, were considered odd since the genetic code of bacteria is not complex enough to account for any wasted portions of sequence. Scientists determined these sequences contributed to a defense system against a class of viruses that attack bacteria in a never-ending war. The bacteria developed CRISPR to identify the virus, cut out a section of its genetic code, and place it within its own DNA as a safeguard against that specific virus.

Researchers realized that if this CRISPR mechanism could be understood, it could be modified to identify selected sequences within the DNA of an organism and excise those segments with previously unachievable precision. It could also be used to replace the code spliced out with a healthier sequence. Doudna and Charpentier’s team mapped out the relationship between two types of RNA and what is known as a CRISPR adjacent enzyme or CAS, CAS-9 being the most popularly used, to identify sequences. As described in a NewScientist article, “When the CRISPR Cas9 protein is added to a cell along with a piece of guide RNA, the Cas9 protein hooks up with the guide RNA and then moves along the strands of DNA until it finds and binds to a 20-DNA-letter long sequence that matches part of the guide RNA sequence. That’s impressive, given that the DNA packed into each of our cells has six billion letters and is two metres long.”⁴ The discovery of “the CRISPR/Cas9 genetic scissors” eventually won Doudna and Charpentier the Nobel Prize in Chemistry.⁵

Once the sequence is identified, CRIPSR can excise the sequence — effectively turning off the genetic expression — replace the sequence, replace a specific single letter in a genetic code, or simply switch a specific function on or off. Gene editing existed prior to CRISPR, but it was expensive and difficult. CRISPR changed all that and opened a new world of possibility.⁶

CRISPR Therapies

How did CRISPR transform the life of Victoria Gray? Sickle cell disease is the most common genetic disease in the world, affecting somewhere around 100,000 U.S. citizens alone. Bone marrow transplants offered help, but there is often little hope of finding a suitable donor. Without a close genetic match, the patient faces a difficult and expensive procedure with risk of their body rejecting foreign biological material. Because the nature of the genetic defect in sickle cell disease is understood, SCD became an early test case for the potential of CRISPR treatments.

In brief, as long as the body produces fetal blood cells, the effects of the genetic disorder appear held at bay.⁷ The pain crisis events begin when the adult hemoglobin cells become the predominate cells in the patient. Scientists noticed that patients who continued to produce fetal blood cells as well as adult cells suffered fewer of the negative side effects of SCD. If they could remove bone marrow stem cells, edit them to reproduce as fetal cells, and reintroduce them, perhaps the patient’s body would produce healthy blood cells, enabling them to live a normal life.

The CRISPR treatment process is expensive and difficult; the cost was estimated at $1 million per patient in trials and requires each patient to undergo chemotherapy to kill the existing pathological cells in the bone marrow. It does enjoy the merit of offering a solution to a life of pain without the risk of donor rejection because the treatment uses the patient’s own genetic material. Victoria Gray spent her time in London in museums and riding the Eye of London, living her life as anyone else who doesn’t live with SCD. However difficult and expensive the process was, she feels the results are miraculous.

The Cost of Cures

Walter Isaacson’s book The Code Breaker details the race to discover and control the potential of CRISPR.⁸ Brilliant minds worked long hours, funded and supported by prestigious research universities and firms. The drive for academic credit and patents wasn’t entirely altruistic in nature. Curing disease and genetically modifying consumable products leads to massive profits.

Innovation comes at a cost. The drive to create draws the best minds because the payoff for winning this competition is immense, but compensation cannot always be provided by those in need of the treatments.⁹ The treatment protocol that benefited Victoria Gray is one of many being pursued to help those with SCD. If this treatment, or any of the others, were to become a reliable and relatively safe cure for SCD, then how do we proceed? Senators Cory Booker and Tim Scott reintroduced a bill intended to help Americans get access to effective treatments¹⁰, but the U.S. government cannot possibly commit to paying for every SCD treatment protocol at the current price point. Any attempt to compel fixed prices threatens to create a disincentive to further research. We are faced with the twin concerns of protecting an environment that inspires the best scientific minds to aggressively pursue medical advancement without cultivating a society where physical health is inextricably tied to financial wealth.

CRISPR Risks

Risks accompany any medical and scientific advancement. The risks can be broadly placed into two categories: (1) the new advancement fails to work as intended or (2) it works so well it is used in irresponsible ways.

There have already been concerns raised based on off-target effects. Some claim that the CRISPR mechanisms linger too long in the body and make off-target edits, erasing, activating, or changing genetic information not specifically targeted by the RNA delivery system. The most serious claims have been quashed, but there appear to be genuine concerns about CRISPR treatments inadvertently removing small portions of off-target genetic code.¹¹ Thus far, the off-target corrections appear to impact inactive sequences of code in controlled research environments, but it raises a concern. As our ability to read and manipulate genetic information accelerates, controlling off-target effects must be prioritized to prevent unintended damage to functioning systems.

The rapid success and accessibility of CRIPSR biotechnology creates a far greater and immediate risk: a complete lack of control of the rush to advancement. The capacity to reshape the genetic future of humanity threatens to remove safeguards from research and further commodify nascent human life as a laboratory resource to exploit.

The CRIPSR experimental community immediately set guidelines for evaluating potential germline changes.¹² Somatic genetic edits occur on mature cells. Scientists remove cells, edit the DNA to address a specific pathology, and reintroduce the cells into the patient’s body. None of these somatic genetic changes survive past the life of the patient. However, germline edits occur at the outset of a new life before cellular specialization. These edits exist in every cell of the nascent human life and can be passed on to the next generation. Germline changes hold incredible promise — debilitating and deadly genetic diseases can be removed from the human family forever. They also risk at least two unintended consequences: (1) we may not know the full impact of edits until after they have been passed to future generations and (2) germline-editing technology may lead to dehumanizing early human life under the pretense of choosing a better future for humanity. The first concern remains hypothetical; the second is an observation of current research practices.

CRISPR Babies

He Jianku, a Chinese CRISPR researcher, illustrated the uncontrollable nature of this field. Impassioned by the desire to protect future generations from HIV, an illness that ravaged the area of China in which he grew up, and the less altruistic desire to bring glory to his name and China by being the Neil Armstrong of genetic editing, the first man to create CRISPR edited human babies — Jianku created a line of embryos genetically altered to be incapable of contracting HIV.¹³ After several failed trials, wasting multiple embryonic human lives, Jianku successfully implanted twins into their biological mother; they then matured and were ultimately born.¹⁴ The scientific community responded almost universally with criticism and condemnation. HIV is already treatable, other therapies are being developed that do not require germline changes, the edits may leave the children and their children more vulnerable to other illnesses, and, finally, the move wasn’t necessary — failing to meet the standards established by the scientific community for risking germline changes. A Chinese court convicted Jianku of illegal practices, sentenced him to pay fines, three years in prison, and banned him from future work in the reproductive sciences.¹⁵

Not everyone shared the negative reaction. Josiah Zayner, a self-described biohacker, celebrated Jianku’s bold step.¹⁶ People like Zayner believe that rapid advancement and equitable distribution of the rewards of CRISPR can be attained only through open-source efforts, like the contribution hackers made to the advancement of computers. Let the crowd see the science and pursue it wherever they can, limited only by their own ingenuity.

Zayner lives with bipolar disorder and argues that germline efforts hold the key to eliminating unnecessary suffering and moving humanity toward a superior genetic existence.¹⁷ Why limit advancement to somatic and therapeutic changes? Why not have the tallest, smartest, most athletic, and most beautiful child possible through gene editing? Zayner argues that it isn’t fair that blind chance plays such a powerful role in the genetic destiny of individuals, and CRISPR makes it possible to solve that inequity. Like the opening quote from the old TV show The Six Million Dollar Man (1973–1978), we have the technology to make the next generation better — better…faster…stronger. Biohackers will pursue those goals without consideration to worldview concerns that see such efforts as treating our humanity with a recklessly cavalier attitude in effecting unnecessary changes. For biohackers, the rigor of the institutional scientific process only slows down advancement.

Germline changes are going to occur. They cannot be stopped. There is little evidence the practice can even be controlled. That is an area of legitimate concern, as not all agents operate from pure motives.

What Place for the Image of God?

The 2001 film, The Lord of the Rings: The Fellowship of the Ring, opens in a whispered quote in Elvish tongue quickly translated into English for the audience, “The world has changed.”¹⁸ The real world has changed as well. All that we thought we knew about what it means to be human stands challenged daily in the world around us. The scientific community promises cures, therapies, and modern-day miracles, while often treating early human life as a resource through experimentation. That disregard for the value of early human life moves those who hold an inclusive view of human life to carefully sort through the ethics of their participation in the current scientific revolution.

Victoria Gray sees no conflict between science and faith — only cooperation.¹⁹Her medical team participated in a miracle that released her from a lifetime of pain and limits. She believes God worked through that community to heal her and others like her. In raising concerns, we should not begrudge her this feeling. Many years ago, I heard a criticism leveled at the Christian community by an unbelieving scholar. He argued that Christians and the public wait too long to respond to the ethical dilemmas raised through new advances. We are not forward thinking, he argued, but reactionary. We wait till the genie is all the way out of the bottle then wring our hands that releasing the genie may not have been a good idea. It is too late, and the world we wish to reach with our appeals already moved down the road working on the next development to which the faith community will inevitably respond too late.

As CRISPR research advances, Christians should give thought to how the world around us has changed and give voice to our views. We can celebrate the cures while condemning some of the practices. We can watch with interest, seeking clarity, without allowing a natural distrust to exclude us from dialogue by our own design. CRISPR was born out of the natural defenses of life against attack, it is fitting that we discovered something so powerful in existing life and not in an unnatural laboratory process. It reminds us to be good stewards of God’s creation because it holds the keys to our future health and healing.

We also must champion the inclusive view of human value. Future therapies for some human lives cannot, as a matter of course, be built on the lives of embryonic humans sacrificed to the cause. Health cannot be a perk of material wealth to be fully enjoyed only by those who can afford cures. The potential ability to address genetic disorders through germline changes cannot allow us to see those living with affliction as living lesser lives. The ability to impact humanity and reprogram the human gene cannot cloud our view that all human beings are the image bearers of God and worthy of our respect.

Jay Watts is founder and president of Merely Human Ministries, an organization defending intrinsic human dignity.

NOTES

1. Rob Stein, “Why Genetic Engineering Experts Are Putting a Spotlight on Victoria Gray’s Case,” NPR, March 7, 2023, https://www.npr.org/2023/03/07/1161570067/why-genetic-engineering-experts-are-putting-a-spotlight-on-victoria-grays-case.
2. Walter Isaacson, The Code Breaker: Jennifer Doudna, Gene Editing, and the Future of the Human Race (New York: Simon & Schuster, 2021), 71–77; Michael LePage, “There’s a New Kind of Superfood, and It’s Not What You Think,” NewScientist, May 23, 2018, https://www.newscientist.com/article/mg23831790-300-theres-a-new-kind-of-superfood-and-its-not-what-you-think/; Heidi Ledford, “CRISPR Cancer Trial Success Paves the Way for Personalized Treatments,” Nature, November 10, 2022, https://www.nature.com/articles/d41586-022-03676-7.
3. Megan Molteni, “With CRISPR Cures on Horizon, Sickle Cell Patients Ask Hard Questions about Who Can Access Them,” STAT, March 7, 2023, https://www.statnews.com/2023/03/07/crispr-sickle-cell-access/.
4. Michael Le Page, “CRISPR: A Technology That Can Be Used to Edit Genes,” NewScientists, https://www.newscientist.com/definition/what-is-crispr/.
5. “Jennifer Doudna and Emmanuelle Charpentier Win 2020 Nobel Prize in Chemistry,” UNESCO, October 7, 2020, https://www.unesco.org/en/articles/jennifer-doudna-and-emmanuelle-charpentier-win-2020-nobel-prize-chemistry#.
6. Heidi Ledford, “CRISPR, the Disruptor,” Nature, June 3, 2015, https://www.nature.com/articles/522020a.
7. Michael Le Page, “Children to get CRISPR Treatment for Sickle Cell Disease in Trial,” NewScientist, June 16, 2022, https://www.newscientist.com/article/2324518-children-to-get-crispr-treatment-for-sickle-cell-disease-in-trial/; Fintan Burke, “CRISPR Provides Hope of Sickle Cell Cure,” Horizon, December 12, 2019, https://ec.europa.eu/research-and-innovation/en/horizon-magazine/crispr-provides-hope-sickle-cell-cure.
8. See bibliographical information in endnote 2.
9. Molteni, “CRISPR Cures on Horizon,” https://www.statnews.com/2023/03/07/crispr-sickle-cell-access/.
10. “Booker, Scott, Davis, Burgess Reintroduce Legislation Addressing Legislation Addressing Sickle Cell Disease,” Press Release from the Office of Cory Booker, March 21, 2023, https://www.booker.senate.gov/news/press/booker-scott-davis-burgess-reintroduce-legislation-addressing-sickle-cell-disease.
11. Gaetan Burgio, “Should We Be Worried about CRISPR/Cas9 Off Target Effects?,” Medium, June 3, 2017, https://medium.com/@GaetanBurgio/should-we-be-worried-about-crispr-cas9-off-target-effects-57dafaf0bd53; Michael Le Page, “CRISPR Gene Editing Is Not Quite as Precise and as Safe as Thought,” New Scientist, July 16, 2018, https://www.newscientist.com/article/2174149-crispr-gene-editing-is-not-quite-as-precise-and-as-safe-as-thought/.
12. Isaacson, The Code Breaker, 286–89.
13. Isaacson, The Code Breaker, 304–05.
14. Isaacson, The Code Breaker, 304–06.
15. Isaacson, The Code Breaker, 332.
16. Isaacson, The Code Breaker, 252–57
17. Isaacson, The Code Breaker, 327–28.
18. The Lord of the Rings: The Fellowship of the Ring, directed by Peter Jackson, screenplay by Fran Walsh, Philippa Boyens, and Peter Jackson (Los Angeles: New Line Cinema, 2001).
19. Stein, “Genetic Engineering Experts,” https://www.npr.org/2023/03/07/1161570067/why-genetic-engineering-experts-are-putting-a-spotlight-on-victoria-grays-case.