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Researchers announce the first patient has been dosed in a trial testing remestemcel-L, a stem cell therapy, in severe COVID-19 patients on ventilators.

grey coronavirus particle interacting with red and purple stem cell

Testing of an experimental COVID-19 stem cell therapy has begun in the US. The therapy has been developed to treat hospitalised COVID-19 patients with moderate to severe acute respiratory distress syndrome (ARDS) who are on ventilators. A total of 300 are expected to be recruited into the randomised, placebo-controlled trial.

ENCODE Project’s third phase offers new insights into the organization and regulation of our genes and genome.

The Encyclopedia of DNA Elements (ENCODE) Project is a worldwide effort to understand how the human genome functions. With the completion of its latest phase, the ENCODE Project has added millions of candidate DNA “switches” from the human and mouse genomes that appear to regulate when and where genes are turned on, and a new registry that assigns a portion of these DNA switches to useful biological categories. The project also offers new visualization tools to assist in the use of ENCODE’s large datasets.

The project’s latest results were published in Nature, accompanied by 13 additional in-depth studies published in other major journals. ENCODE is funded by the National Human Genome Research Institute, part of the National Institutes of Health.

More from 2020 “The Movie”

Chinese government-linked hackers targeted biotech company Moderna Inc, a leading U.S.-based coronavirus vaccine research developer, earlier this year in a bid to steal valuable data, according to a U.S. security official tracking Chinese hacking activity.


WASHINGTON (Reuters) — Chinese government-linked hackers targeted biotech company Moderna Inc, a leading U.S.-based coronavirus vaccine research developer, earlier this year in a bid to steal valuable data, according to a U.S. security official tracking Chinese hacking activity.

CRISPR-Cas9 base editors comprise RNA-guided Cas proteins fused to an enzyme that can deaminate a DNA nucleoside. No natural enzyme deaminates adenine in DNA, and so a breakthrough came when a natural transfer RNA deaminase was fused to Cas9 and evolved to give an adenine base editor (ABE) that works on DNA. Further evolution provided the enzyme ABE8e, which catalyzes deamination more than 1000 times faster than early ABEs. Lapinaite et al. now present a 3.2-angstrom resolution structure of ABE8e bound to DNA in which the target adenine is replaced with an analog designed to trap the catalytic conformation. The structure, together with kinetic data comparing ABE8e to earlier ABEs, explains how ABE8e edits DNA bases and could inform future base-editor design.

Science, this issue p. 566

CRISPR-Cas–guided base editors convert A•T to G•C, or C•G to T•A, in cellular DNA for precision genome editing. To understand the molecular basis for DNA adenosine deamination by adenine base editors (ABEs), we determined a 3.2-angstrom resolution cryo–electron microscopy structure of ABE8e in a substrate-bound state in which the deaminase domain engages DNA exposed within the CRISPR-Cas9 R-loop complex. Kinetic and structural data suggest that ABE8e catalyzes DNA deamination up to ~1100-fold faster than earlier ABEs because of mutations that stabilize DNA substrates in a constrained, transfer RNA–like conformation. Furthermore, ABE8e’s accelerated DNA deamination suggests a previously unobserved transient DNA melting that may occur during double-stranded DNA surveillance by CRISPR-Cas9. These results explain ABE8e-mediated base-editing outcomes and inform the future design of base editors.

Steve Granick, Director of the IBS Center for Soft and Living Matter and Dr. Huan Wang, Senior Research Fellow, report together with 5 interdisciplinary colleagues in the July 31 issue of the journal Science that common chemical reactions accelerate Brownian diffusion by sending long-range ripples into the surrounding solvent.

The findings violate a central dogma of chemistry, that and chemical reaction are unrelated. To observe that molecules are energized by chemical reaction is “new and unknown,” said Granick. “When one substance transforms to another by breaking and forming bonds, this actually makes the molecules move more rapidly. It’s as if the chemical reactions stir themselves naturally.”

“Currently, nature does an excellent job of producing molecular machines but in the natural world scientists have not understood well enough how to design this property,” said Wang. “Beyond curiosity to understand the world, we hope that practically this can become useful in guiding thinking about transducing chemical energy for molecular motion in liquids, for nanorobotics, precision medicine and greener material synthesis.”

Within a mere eight years, CRISPR-Cas9 has become the go-to genome editor for both basic research and gene therapy. But CRISPR-Cas9 also has spawned other potentially powerful DNA manipulation tools that could help fix genetic mutations responsible for hereditary diseases.

Researchers at the University of California, Berkeley, have now obtained the first 3D structure of one of the most promising of these tools: base , which bind to DNA and, instead of cutting, precisely replace one nucleotide with another.

First created four years ago, base editors are already being used in attempts to correct single-nucleotide mutations in the human genome. Base editors now available could address about 60% of all known genetic diseases—potentially more than 15,000 inherited disorders—caused by a mutation in only one nucleotide.