The Perils and Promises of CRISPR Gene Editing

Six months ago, a Chinese scientist announced (see Pulse 89, 90)  that he had used CRISPR gene editing to alter the genomes of two babies born last year, modifying the CCR5 gene to prevent HIV from invading immune cells.

"Beyond the many ethical issues involved with the CRISPR babies, the fact is that, right now, with current knowledge, it is still very dangerous to try to introduce mutations without knowing the full effect of what those mutations do," says Rasmus Nielsen, a UC Berkeley professor of integrative biology and the senior author of a paper on dangerous side effects of CRISPR gene editing in humans (see below).

"In this case, it is probably not a mutation that most people would want to have,” he said. “You are actually, on average, worse off having it."

It’s worth noting that other researchers have argued that the intervention could also have positive effects, namely increased intelligence (see Pulse 99).

Of course, editing the human genome without fully understanding all possible effects is risky and potentially dangerous. But it can be also argued that preliminary experimentation in humans is a necessary first step toward better understanding. As usual, there are two sides to the coin.

I wish a long, healthy and happy life to the first CRISPR babies!

Scientists warn of dangerous side effects of CRISPR in humans. Scientists at UC Berkeley are suggesting that the CRISPR mutation of the CCR5 gene, achieved by a Chinese scientist in twin babies born last year to help them fend off HIV infection, is also associated with a 21% increase in mortality in later life. In a paper published in Nature Medicine, the scientists suggest that an analysis of 400,000 genomes and associated health records, held in the UK Biobank repository, reveals that people who had two mutated copies of the gene had a significantly higher death rate between ages 41 and 78 than those with one or no copies.

Molecular anti-aging mechanisms stop working in older humans. Researchers at University of Miami have suggested that human disease burden increases so sharply from the sixth decade of life onward because health-protective mechanisms disappear. A research paper published in Aging Cell argues that, in humans, key molecular programs known to promote longevity do not last beyond midlife. The scientists suggest that the complexity of regulation of aging programs may be much greater in humans as compared to other species.

Fighting cancer with nanoparticles stimulated by microwaves. Physicists at University of Texas Arlington have proposed a new concept for treating cancer with microwave-induced radical therapy, or microdynamic therapy: using titanium dioxide (TiO2) nanoparticles stimulated by microwaves to trigger the death of cancer cells without damaging the normal cells around them. A research paper published in Biology and Medicine shows that the nanoparticles can be effectively activated by microwaves for cancer cell destruction, potentially opening new doors to treatment for patients fighting the disease.

New strategy for cancer drug discovery and development. Researchers at Dartmouth College have developed a new strategy for drug discovery and development that can be used to produce targeted therapies against diseases such as cancer and neurodegeneration. The new technique, described in a study published in Nature Communications, uses a novel synthesis approach for a class of organic compounds known as tetracyclic terpenoids, and combines two new chemical reactions that establish bonds between carbon atoms. To demonstrate the value of the technique, the study describes the discovery of a molecule that is selectively toxic to glioblastoma.

Vitamin D could help cancer patients live longer. Physicians at Michigan State University have found that vitamin D, if taken for at least three years, could help cancer patients live longer. The findings, described in a paper published in Journal of Clinical Oncology and presented at the American Society of Clinical Oncology annual meeting on June 3, suggest that the vitamin carries significant benefits other than just contributing to healthy bones. However, the scientists cautioned that the exact amount of the vitamin to take and what levels are needed in the blood are still unknown.

Bioink permits 3D printing therapeutics at precise locations. Researchers at Texas A&M University have developed a way to print therapeutics in 3D for regenerative medicine. A research paper published in Advanced Healthcare Materials describes a bioink consisting of 2D mineral nanoparticles, which permits 3D printing therapeutics at precise locations. The new technology, based on a nanoclay platform, can be used for precise deposition of protein therapeutics.

Expanded Potential Stem Cells. Researchers at University of Hong Kong, Wellcome Sanger Institute, and the Friedrich-Loeffler-Institut have created Expanded Potential Stem Cells (EPSCs) of both pig and human cells. This is the first time scientists have been able to derive stem cells from early pig embryos, which will also be beneficial for animal health and food production. The research, described in a study published in Nature Cell Biology, has potential for facilitating study of human development and regenerative medicine.

Boosting the immune system against antibiotic-resistant infections. Scientists at University of Edinburgh have found that a molecule produced by the body, called LL-37, changes the way cells behave when they are invaded by bacteria. The findings, published in PLOS Pathogens, indicate that the molecule acts like a fire alarm, warning the body's immune system of the infection and the need for urgent action. The scientists are persuaded that boosting the human body's effective natural defenses may prove to be an important part of future treatments for antibiotic-resistant infections.