The Future of Gene Editing

Saira Munshani ’20 Senior Op/Ed Editor

Gene editing and therapy, like Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR), are revolutionizing the way we view and treat disease.

There are many factors to consider such as the larger effects of seemingly small changes on an individual’s genome and the overarching ethics. The use of CRISPR spans disciplines - science, technology, politics - which makes it harder to find a single answer regarding regulation. The search for a universal protocol and limit becomes more complex with every scientific advancement.

Two years ago, Dr. He Jiankui of the Southern University of Science and Technology in Shenzhen, China, modified human embryos with CRISPR. The father was HIV-positive, so Dr. He and his team used a CRISPR system to prevent his children from contracting the disease as well. Scientists have long been searching for a way to prevent HIV-enabling pathways in order to eradicate the disease. With the use of modern laboratory techniques and knowledge of CRISPR, Dr. He and his team used CRISPR to knock out the CCR5 gene, which allows HIV to enter immune cells and infect them, in embryos that were a day old. He did this with sixteen embryos and implanting them in wombs, he achieved a viable pregnancy.

In November 2018, as a result of Dr. He’s work, twin girls were born healthy. This sparked a myriad of questions from scientists all over the world. Would the changes to the genome cause issues for the twins later in life? Is it ethical to impact the DNA that will be passed down for generations? Who gets access to CRISPR related technologies? Almost immediately after the incident, scientists called a moratorium “on all clinical uses of human germline editing — that is, changing heritable DNA (in sperm, eggs or embryos) to make genetically modified children,” as cited from an article published in Nature in March of 2019. The goal of the moratorium was not to completely halt all CRISPR related research. Instead, scientists hoped to establish limits on the use of clinical genetic editing in order to investigate the ethics surrounding altering the human genome. Authorities later revealed that Dr. He forged ethical documents and misled doctors into implanting the edited embryos. There is still little known about his exact activities; however, Dr. He was sentenced to three years in jail for his actions this past December.

CRISPR research is still ongoing and studies that eliminate the risk of heritable changes have been successful. For instance, Dr. Haydar Frangoul in Tennessee has been able to modify bone marrow cells inside a sickle cell disease patient’s body so that they create a protein which helps to alleviate the effects of the disease. These advancements are happening all over the world - the same type of modified sickle cells are helping another patient in Germany. In contrast to Dr. He’s work, bioethicists are now heavily involved every step of the way, and work with the scientists, doctors, and patients.

Determining the definitive and ethical uses of CRISPR is a persistent process. Currently, many studies involve systems in vivo (live animal experiments) and in vitro (experiments on isolated cells), and these will help provide insight into how CRISPR can be most effectively used. Whether it be to create “knockout” versions of a gene for transcriptional repression/activation, or for patient treatment, this technology has the power to change our world forever. CRISPR is unlike other scientific advancements because of its multifaceted impact on society. It is up to both scientists and citizens to find a balance between globally spanning advancements of CRISPR and the ethics of implementing it.

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The Future of Gene Editing