Enlarge (credit: Getty | Justin Sullivan)

The US government will pay Moderna $176 million to develop an mRNA vaccine against a pandemic influenza—an award given as the highly pathogenic bird flu virus H5N1 continues to spread widely among US dairy cattle.

The funding flows through BARDA, the Biomedical Advanced Research and Development Authority, as part of a new Rapid Response Partnership Vehicle (RRPV) Consortium. The program is intended to set up partnerships with industry to help the country better prepare for pandemic threats and develop medical countermeasures, the Department of Health and Human Services said in a press announcement Tuesday.

In its own announcement on Tuesday, Moderna noted that it began a Phase 1/2 trial of a pandemic influenza virus vaccine last year, which included versions targeting H5 and H7 varieties of bird flu viruses. The company said it expects to release the results of that trial this year and that those results will direct the design of a Phase 3 trial, anticipated to begin in 2025.

Enlarge / Top row: individual steps in the reaction process. Bottom row: cartoon diagram of the top, showing the position of each DNA and RNA strand. (credit: Hiraizumi, et. al.)

While CRISPR is probably the most prominent gene-editing technology, there are others, some developed before and since. And people have been developing CRISPR variants to perform more specialized functions, like altering specific bases. In all of these cases, researchers are trying to balance a number of competing factors: convenience, flexibility, specificity and precision for the editing, low error rates, and so on.

So, having additional options for editing can be a good thing, enabling new ways of balancing those different needs. On Wednesday, a pair of papers in Nature describe a DNA-based parasite that moves itself around bacterial genomes through a mechanism that hasn't been previously described. It's nowhere near ready for use in humans, but it may have some distinctive features that make it worth further development.

Going mobile

Mobile genetic elements, commonly called transposons, are quite common in many species—they make up nearly half the sequences in the human genome, for example. They are indeed mobile, showing up in new locations throughout the genome, sometimes by cutting themselves out and hopping to new locations, other times by sending a copy out to a new place in the genome. For any of this to work, they need to have an enzyme that cuts DNA and specifically recognizes the right transposon sequence to insert into the cut.

Enlarge / The spliceosome is a large complex of proteins and RNAs. (credit: NCBI)

Almost 1,500 genes have been implicated in intellectual disabilities; yet for most people with such disabilities, genetic causes remain unknown. Perhaps this is in part because geneticists have been focusing on the wrong stretches of DNA when they go searching. To rectify this, Ernest Turro—a biostatistician who focuses on genetics, genomics, and molecular diagnostics—used whole genome sequencing data from the 100,000 Genomes Project to search for areas associated with intellectual disabilities.

His lab found a genetic association that is the most common one yet to be associated with neurodevelopmental abnormality. And the gene they identified doesn’t even make a protein.

Trouble with the spliceosome

Most genes include instructions for how to make proteins. That’s true. And yet human genes are not arranged linearly—or rather, they are arranged linearly, but not contiguously. A gene containing the instructions for which amino acids to string together to make a particular protein—hemoglobin, insulin, albumin, whatever protein you like—is modular. It contains part of the amino acid sequence, then it has a chunk of DNA that is largely irrelevant to that sequence, then a bit more of the protein’s sequence, then another chunk of random DNA, back and forth until the end of the protein. It’s as if each of these prose paragraphs were separated by a string of unrelated letters (but not a meaningful paragraph from a different article).