Lifeboat Foundation

The gene-editing system CRISPR-Cas9 which has revolutionized genetic engineering over the past decade involves cutting DNA strands which is a process that can be quite hard to control and can result in unwanted genetic changes. Now, thanks to researchers at the Massachusetts Institute of Technology and the University of California, San Francisco (UCSF), a new gene-editing technology called CRISPRoff can change that, according to a press release.

“Fast forward four years [from the initial grant], and CRISPRoff finally works as envisioned in a science fiction way,” says co-senior author Luke Gilbert. “It’s exciting to see it work so well in practice.”

Pictured: Illustration of CRISPR-Cas9 editing DNA / iStock, Artur Plawgo

Currently, there are no gene editing–based treatments on the market, but the technology continues its march toward potential FDA approval, with several products in mid-and late-stage trials. As these programs mature, 2023 could be a pivotal year for companies in the space. Here are some highlights to look forward to as the year progresses.

CRISPR Therapeutics/Vertex Pharmaceuticals.

In the last decade, we have witnessed biology bring us some incredible products and technologies: from mushroom-based packaging to animal-free hotdogs and mRNA vaccines that helped curb a global pandemic. The power of synthetic biology to transform our world cannot be overstated: this industry is projected to contribute to as much as a third of the global economic output by 2030, or nearly $30 trillion, and could impact almost every area of our lives, from the food we eat to the medicine we put in our bodies.

The leaders of this unstoppable bio revolution – many of whom you can meet at the SynBioBeta conference in Oakland, CA, on May 23–25 – are bringing the future closer every day through their ambitious vision, long-range strategy, and proactive oversight. These ten powerful women are shaping our world as company leaders, biosecurity experts, policymakers, and philanthropists focused on charting a new course to a more sustainable, equitable, clean, and safe future.

As an early pioneer in the high-throughput synthesis and sequencing of DNA, Emily Leproust has dedicated her life to democratizing gene synthesis to catapult the growth of synthetic biology applications from medicine, food, agriculture, and industrial chemicals to DNA data storage. She was one of the co-founders of Twist Bioscience in 2013 and is still leading the expanding company as CEO. To say that Twist’s silicon platform was a game-changer for the industry is an understatement. And it is no surprise that Leproust was recently honored with the BIO Rosalind Franklin Award for her work in the biobased economy and biotech innovation.

The remarkable physicochemical properties of the natural nucleic acids, DNA and RNA, define modern biology at the molecular level and are widely believed to have been central to life’s origins. However, their ability to form repositories of information as well as functional structures such as ligands (aptamers) and catalysts (ribozymes/DNAzymes) is not unique. A range of nonnatural alternatives, collectively termed xeno nucleic acids (XNAs), are also capable of supporting genetic information storage and propagation as well as evolution. This gives rise to a new field of “synthetic genetics,” which seeks to expand the nucleic acid chemical toolbox for applications in both biotechnology and molecular medicine. In this review, we outline XNA polymerase and reverse transcriptase engineering as a key enabling technology and summarize the application of “synthetic genetics” to the development of aptamers, enzymes, and nanostructures.

Copyright © 2019 Cold Spring Harbor Laboratory Press; all rights reserved.

Individual RNA remains a challenging signal to synthetically transduce into different types of cellular information. Here, we describe Ribozyme-ENabled Detection of RNA (RENDR), a plug-and-play strategy that uses cellular transcripts to template the assembly of split ribozymes, triggering splicing reactions that generate orthogonal protein outputs. To identify split ribozymes that require templating for splicing, we use laboratory evolution to evaluate the activities of different split variants of the Tetrahymena thermophila ribozyme. The best design delivers a 93-fold dynamic range of splicing with RENDR controlling fluorescent protein production in response to an RNA input. We further resolve a thermodynamic model to guide RENDR design, show how input signals can be transduced into diverse outputs, demonstrate portability across different bacteria, and use RENDR to detect antibiotic-resistant bacteria. This work shows how transcriptional signals can be monitored in situ and converted into different types of biochemical information using RNA synthetic biology.

© 2023. The Author(s).

Conflict of interest statement.

Bioethicist Nita Farahany says privacy law hasn’t kept up with science as employers increasingly use neurotechnology in the workplace.

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Lifestyle behaviors such as eating well and exercising can be significant factors in one’s overall health. But the risk of developing cancer is predominantly at the whim of an individual’s genetics.

Our bodies are constantly making copies of our genes to produce new cells. However, there are occasional mistakes in those copies, a phenomenon geneticists call mutation. In some cases, these mistakes can alter proteins, fuse genes and change how much a gene gets copied, ultimately impacting a person’s risk of developing cancer. Scientists can better understand the impact of mutations by developing predictive models for tumor activity.

Christopher Plaisier, an assistant professor of biomedical engineering in the Ira A. Fulton Schools of Engineering at Arizona State University, is developing a software tool called OncoMerge that uses genetic data to improve cancer modeling technology.

Engineers at MIT and the University of Massachusetts Medical School have designed a new type of nanoparticle that can be administered to the lungs, where it can deliver messenger RNA encoding useful proteins.

With further development, these particles could offer an inhalable treatment for cystic fibrosis and other diseases of the lung, the researchers say.

“This is the first demonstration of highly efficient delivery of RNA to the lungs in mice. We are hopeful that it can be used to treat or repair a range of genetic diseases, including cystic fibrosis,” says Daniel Anderson, a professor in MIT’s Department of Chemical Engineering and a member of MIT’s Koch Institute for Integrative Cancer Research and Institute for Medical Engineering and Science (IMES).