Archive by category | Research

Alnylam launches era of RNAi drugs

Alnylam launches era of RNAi drugs

On August 10, the US Food and Drug Administration approved the first RNA interference (RNAi) therapeutic, a treatment for polyneuropathy caused by transthyretin (TTR) amyloidosis from Alnylam Therapeutics. The go-ahead for Onpattro (patisiran) sees the RNAi field clear an approval hurdle considered unlikely as recently as six years ago, when pharma exited the RNAi field en masse. The US approval, with Europe expected to follow by early September, is “a major milestone,” says Anastasia Khvorova, an RNAi researcher at the University of Massachusetts in Worcester. Onpattro has an excellent safety record, but there are lingering concerns about potential long-term toxicity from newer, more potent RNAi therapeutics. And the field as a whole still faces investor skepticism in the wake of a decade of clinical trial failures.  Read more

A new approach for DNA synthesis

A new approach for DNA synthesis

Ordering synthetic oligos or genes online is now commonplace and an essential resource to scientists across disciplines. But the phosphoramidite chemistry currently used to synthesize DNA is limited to direct synthesis of about 200 nucleotides, with longer stretches requiring assembly. The capacity to synthesize long stretches of DNA is important for a variety of applications, including DNA storage, DNA origami, and to synthesize DNA containing regions with repeats, which are difficult to put together. In a paper published recently in Nature Biotechnology, Jay Keasling and colleagues report a promising new approach to DNA synthesis. Using a terminal deoxynucleotidyl transferase (TdT) conjugated to a single deoxyribonucleoside triphosphate (dNTP), they tether the primer to TdT after extending it by one nucleotide.  Read more

Hunting connections between cell types and cytokines

Hunting connections between cell types and cytokines

Cytokines are small proteins that mediate signalling among immune and non-immune cells, and they trigger a range of cellular activity, such as proliferation, activation and killing. Over many decades, immunologists have described countless associations between cell types and the cytokines they produce or sense, but many of these findings, although published, are difficult to access. Associations may have been discovered in a particular disease context or cell type, or uncovered as part of a larger study and thus not corroborated or expanded. Work from Shai Shen-Orr and colleagues, published in Nature Biotechnology, aims to unearth these connections and provide a useful resource for enabling new discoveries.  Read more

Will the EU deregulate gene-edited plants?

Will the EU deregulate gene-edited plants?

At the beginning of the year, the advocate general of the Court of Justice of the European Union (CJEU) issued an opinion that plants created using new plant breeding techniques, including gene-editing platforms like CRISPR, TALENs and the like, are eligible for the so-called mutagenesis exemption. This exemption relates to rules the European Union uses to regulate the release and marketing of genetically modified organisms (GMOs), which are outlined in Directive (2001/18/EC), originally drafted in 2001. The exemption covers any plants considered ‘safe’ or produced using techniques that have a history of safety, including plants derived from traditional mutagenesis (hence the mutagenesis exemption).  Read more

Is yellow fever back in Brazil?

Is yellow fever back in Brazil?

The answer to this question is, Not exactly. Yellow fever never left Brazil. I earlier wrote that Oswaldo Cruz eradicated yellow fever in Brazil in the 19th century. In fact the extraordinary work done by Cruz focused on yellow fever in urban areas in Rio de Janeiro, but the illness persisted in the jungles.  Read more

Rumen microbial genomics resource

Robert (Bob) E. Hungate developed methods (the ‘Hungate technique') to culture anaerobic bacteria and archaea. These methods are still used in many labs worldwide.

The Hungate1000 project, named after one of the great microbiologists, Robert E. Hungate (pictured), was launched with the aim of producing a reference set of rumen microbial genome sequences. When this project began there was only a handful of rumen reference microbial genomes available. The first output of the Hungate1000 project, comprising 410 high-quality genome sequences, is reported online today in Nature Biotechnology. Seshadri et al. highlight discovery of degradative enzymes, biosynthetic gene clusters and Crispr sequences. These reference genomes will enable robust interpretation of rumen metagenomes, which should result in a better understanding of rumen functions. Genome-enabled research into feed conversion efficiency, methanogenesis and cellulose degradation will, in turn, assist development of strategies to balance food production with efforts to minimize greenhouse gas emissions. Finally, access to cultivated Hungate Collection strains will provide vital tools for studying carbon flow in the rumen, breakdown of lignocelluloses and methane formation.  Read more

The Developing World Needs GMOs

The Developing World Needs GMOs

The need to feed growing populations in developing countries, especially countries in Africa, must be met by increasing the yields of crops. Also, climate-change related problem such as drought continue to worsen hunger problem and humanitarian crisis in the continent. Genetically modified organisms (GMOs) could greatly help with these issues, yet resistance persists in Europe and Africa both.  Read more

Turning science and technology into a priority in Brazil

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In a previous contribution to this blog, I said that science and technology is not a priority in less developed countries, including Brazil. I recently described why this is in Scientia & Ricerca. Brazil’s government claims it cannot treat science and technology different from other areas. If it cannot double the investments in other areas, it cannot double the investment in science and technology. Since the Gross National Product (GNP) of Brazil cannot double in one year we are stuck with investments in science and technology at 1% of GNP historically.  Read more

Synthetic Vaccines

Traditional and novel forms of vaccines.

In 2010 scientists from the JCVI announced the creation of the first bacterial cell controlled by a chemically synthesized genome1. The ‘synthetic’ cell was mycoplasma, a bacterium with an exceptionally small genome of less than 1 million base pairs and without a cell wall. Carole Lartigue, one of the co-authors of that landmark paper, later returned to the National Research Institute for Agriculture (INRA) in Bordeaux, France, to continue working on Mycoplasma. In fact Mycoplasma is not just a beautiful model organism for synthetic genomics. Their small genomes make them also a great model for systems biology, a work that was spearheaded by Luis Serrano at CRG in Barcelona2, 3, 4, who characterized Mycoplasma in a quantitative manner to apply this knowledge to do a rational engineering for novel applications.  Read more