CRISPR Advances in the Lab and Human Trials

The first two news summaries below show, once again, that CRISPR gene editing technology is advancing fast in the lab. There are also more and more indications that CRISPR trials in humans are inevitable and are coming soon.

Five Russian couples, who are deaf, want to try the CRISPR gene-editing technique so they can have a biological child who can hear, biologist Denis Rebrikov has told New Scientist. He plans to apply to the relevant Russian authorities for permission. “It is clear and understandable to ordinary people,” said the scientist. “Each new baby for this pair would be deaf without gene mutation editing.”

Rebrikov’s highly controversial plans have been recently covered by Nature and MIT Technology Review. See also Pulse 109, 106, 90, 89.

Nanoparticles precisely deliver CRISPR gene editing payloads to cells. Researchers at Tufts University and the Chinese Academy of Sciences have developed a significantly improved delivery mechanism for the CRISPR/Cas9 gene editing method in the liver. The delivery method, described in a research paper published in Advanced Materials, uses biodegradable synthetic lipid nanoparticles that carry the molecular editing tools into the cell to precisely alter the cells' genetic code with as much as 90 percent efficiency. According to the researchers, the nanoparticles could help overcome technical hurdles to enable gene editing in a broad range of clinical therapeutic applications.

CRISPR-engineered T cells act like natural immune cells. Scientists at Technical University of Munich (TUM) have engineered T cells that are very similar to physiological immune cells, as reported in a study published in Nature Biomedical Engineering. Until now, modified T cells have not been as effective as natural T cells and have only been of limited clinical value. The scientists used CRISPR-Cas9 gene scissors, which can be used to snip out and replace targeted segments of the genome, to generate modified T cells that are very similar to their natural counterparts and could solve some of those problems.

Nanotheranostic system based on harmonic nanoparticles. Chemical engineers at EPFL have developed a novel nanotheranostic system that uses tunable light to activate or image nanoparticles, thus opening a new path for nanotheranostics, a cutting-edge field of medicine that uses nanoparticles to simultaneously diagnose and treat diseases. The new system, described in a study published in ACS Applied Materials and Interfaces, uses "harmonic nanoparticles" (HNPs), a family of metal-oxide nanocrystals, and can be easily activated with near-infrared light and imaged at longer wavelength for both detection and drug release processes.

Common cold viruses target, infect, and destroy cancer cells. Scientists at University of Surrey and Royal Surrey County Hospital have investigated the safety and tolerability of exposure to the oncolytic (cancer-killing) virus coxsackievirus (CVA21), a naturally occurring strain of the common cold, in fifteen patients with non-muscle invasive bladder cancer (NMIBC). In a research paper published in Clinical Cancer Research, the scientists report that the virus is able to target, infect, and destroy cancer cells in patients with bladder cancer. No trace of the cancer was found in one patient following treatment with the virus.

Engineered bacteria deliver cancer immunotherapy, kill cancer cells. Researchers at Columbia University have engineered a strain of non-pathogenic bacteria that can colonize solid tumors in mice and safely deliver potent immunotherapies, acting as a Trojan Horse that treats tumors from within. A study published in Nature Medicine describes how the researchers achieved not only complete tumor regression in a mouse model of lymphoma, but also significant control of distant, uninjected tumor lesions.

Nanoprobe arrays record the electrical activity of cardiac cells and neurons. Scientists at University of Surrey and Harvard University have built scalable nanoprobe arrays small enough to record the inner workings of human cardiac cells and primary neurons. In a paper published in Nature Nanotechnology, the scientists detail how they produced an array of the ultra-small U-shaped nanowire field-effect transistor probes for intracellular recording. The ability to read electrical activity from cells is the foundation of many biomedical procedures, such as brain activity mapping, neural prosthetics, and Brain-Computer interfaces (BCI).

Computer model of cancer cells assists therapy. Researchers at University of Luxembourg have developed a computer model that simulates the metabolism of cancer cells. A study published in EBioMedicine describes how the researchers used the program to investigate how combinations of drugs could be used more effectively to stop tumor growth.

A bacterium seems to reduce cardiovascular risks. Scientists at Université Catholique de Louvain have found that the bacterium Akkermansia limits the increase of several risk factors for cardiovascular diseases, moderates the progression of pre-diabetes, and reduces cholesterol levels in humans. The research results, published in Nature Medicine, are important because one in two individuals is overweight and has cardiovascular risk factors. The scientists are planning larger-scale tests and the commercialization of the bacteria.