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Claimed Birth of CRISPR-Engineered Babies

3 December 2018
Giulio Prisco

Baby CRISPR Genetic Engineering

The claimed birth of the world’s first designer babies, two Chinese twin girls with CRISPR-edited DNA, as reported in the previous issue of Pulse, continues to make waves. Most reactions are strongly negative.

Research leader He Jiankui is now facing an investigation from China’s National Health Commission on the experiment, with a top official at the Ministry of Science and Technology telling state media that He’s work was “extremely abominable,” Time reports.

A spokeswoman for China’s Southern University of Science and Technology said that media reports of He’s detention were inaccurate but added that the university would be unable to answer any further questions regarding the matter, Hong Kong’s South China Morning Post reports.

Time emphasizes that the rumored development was met with a huge backlash from the wider scientific community.

However, some top scientists are praising this development. “I feel an obligation to be balanced,” says geneticist George Church, whose Harvard University lab played a pioneering role in developing CRISPR, in an interview with Science. “[He Jiankui]’s just an acquaintance. But it seems like a bullying situation to me … [He Jiankui]’s not doing it the way I’d do it, but I’m hoping it doesn’t work out badly. As long as these are normal, healthy kids it’s going to be fine for the field and the family.”

I wish to add my voice to Church’s. He Jiankui used CRISPR to try to make the babies resistant to HIV by crippling a receptor, CCR5, that the virus uses to infect white blood cells. This can’t be considered as a bad thing, and real medical needs should take priority over abstract “ethical” considerations. “The unmet medical need is there is no cure or vaccine for HIV,” says Church.

Fine-tuning CRISPR gene editing and avoiding undesired effects. In a new study published in Cell, researchers from the Novo Nordisk Foundation Center for Protein Research have described how one of the CRISPR gene editing technologies, the so-called Cas12a, works - all the way down to the molecular level. This makes it possible to fine-tune the gene-editing process to only achieve the desired effects.

Simple 3D bioprinting technique creates lifelike tissues. Bioengineers at UC San Diego have developed a 3D bioprinting technique that works with natural materials and is easy to use, allowing researchers of varying levels of technical expertise to produce lifelike organ tissue models. A study published in Advanced Healthcare Materials describes how, as a proof of concept, the scientists created blood vessel networks capable of keeping a breast cancer tumor alive outside the body. They also created a model of a vascularized human gut. The new technique is expected to facilitate the development of human organ models that can be studied outside the body or used to test new drugs ex vivo.

Blue Brain Project releases digital 3D brain cell atlas for the whole mouse brain. The Blue Brain Project at EPFL has produced the first digital 3D atlas of every cell in the mouse brain. The atlas provides neuroscientists with previously unavailable information on major cell types, numbers, and positions in all 737 brain regions, which will potentially accelerate progress in brain science massively. The Blue Brain Cell Atlas, published in Frontiers in Neuroinformatics, integrates data from thousands of whole brain tissue stains into a comprehensive, interactive, and dynamic online resource that can be continuously updated with new findings.

Minimally invasive endoscopy for deep brain studies. Researchers at the Leibniz Institute of Photonic Technology in Jena and the University of Edinburgh have used a hair-thin fibre endoscope to gain insights into hardly-accessible brain structures. A research paper published in Light: Science & Applications describes the probe -- the most minimally invasive endoscopic probe reported so far -- and its ability to achieve high-resolution observations of neuronal structures inside deep brain areas of living mice. The scientists are persuaded that this could be a major step toward a better understanding of the functions of deeply hidden brain compartments, such as the formation of memories, as well as related dysfunctions, including Alzheimer's disease.

Toward usable and effective polio vaccines. Researchers at University of Southern California have developed a polio vaccine that doesn't require refrigeration, meaning it could someday be used all over the world to eliminate polio. A temperature-stable vaccine is needed for use in developing countries where refrigeration may be unavailable. A study published in mBio shows that the new injectable vaccine, freeze-dried into a powder, kept at room temperature for four weeks and then rehydrated, offered full protection against the polio virus when tested in mice.

New strategy for precision cancer therapy. Scientists at UC San Francisco have figured out why some lung cancers become drug-resistant after initially responding to targeted therapies. A research paper published in Nature Medicine reports that, in laboratory studies with drug-resistant cancers, drugs targeting a protein called Aurora Kinase A, when combined with osimertinib or rociletinib, killed the cancer cells once and for all. The researchers also identified a promising biomarker that could permit identifying cancers that would respond to the combined therapy. According to the researchers, the study “holds tremendous promise” for the future of precision medicine.

Toward effective cancer vaccines. Researchers at the Biodesign Institute, Arizona State University, have developed a method for pinpointing tumor-specific factors in blood that can elicit a protective immune response in the body and may one day be harnessed to produce an effective vaccine against the disease. A study published in Scientific Reports outlines a means for rapidly identifying peptides produced by tumor-associated mutations, then screening these peptides to find those exhibiting a strong immune response. According to the scientists, this research could lead to potent new weapons against cancer, leveraging the body's own immune defenses to stop this leading killer in its tracks.

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