Genome Editing

Myotonic dystrophy type I (DM1) is the most common type of adult-onset muscular dystrophy. People with DM1 inherit repeated DNA segments that lead to the toxic buildup of repetitive RNA. Now researchers from the University of California, San Diego, School of Medicine report they have reversed symptoms of DM1 in a mouse model by using their CRISPR-mediated RNA targeting method.

Cytiva—which employs more than 7,000 people worldwide—will carry out its expansion across its global product manufacturing and distribution network, which encompasses 13 sites across the U.S. and the rest of the Americas, as well as Asia and Europe.

Sponsor: TecanJoin us for Episode 4 of GEN Live, sponsored by Tecan. In this live zoom event we discuss the state of vaccine development for COVID-19. We evaluate the front-runners, explore the potential of RNA vaccines, ponder the challenges of manufacturing, and tackle questions about herd immunity.

A blue-ribbon commission convened by the National Academy of Sciences (NAS) and the UK’s Royal Society has released a much-anticipated 200-page report on Heritable Human Genome Editing (HHGE). The report charts a course for limited use of HHGE to help couples have a healthy, biologically related child, once several important criteria have been met.

A study is setting new standards for how yeast can be recruited for the production of complex plant natural products. The learnings from this study will enable a far wider application of yeast-based production of complex plant natural products that can be used as pharmaceuticals and nutraceuticals.

Researchers have now developed an approach—targeted individual DNA molecule sequencing (IDMseq)—that can accurately detect a single mutation in a pool of 10,000 cells. This tool is the first quantitative evidence of large structural variants and an increase in single-nucleotide variants at the on-target locus following CRISPR-Cas9 genome editing in human embryonic stem cells.

CRISPR isn’t just a technology, it’s an industry, too. Relationships among suppliers and customers are enabling diverse CRISPR applications—in vivo/ex vivo, allogeneic/autologous, and viral/nonviral.

Lipid- and polymer-based nanoparticles are less likely to arouse immune resistance or wreak collateral damage by targeting the wrong cells or genomic loci. But what about their delivery rates?

Safer and more practical alternatives to viruses are being sought by innovative companies. For example, these companies are developing nonviral gene delivery systems that incorporate nanoparticle formulations, ultrasound, and electric fields. These systems can slip bits of genetic material into cells efficiently and cost-effectively in a range of applications.

GEN asked six outstanding CRISPR researchers about the use of CRISPR for knockout (CRISPR KO), interference, and activation, ramping up to editing primary cells from immortalized cell lines, and how they approach off-target effects. And we asked them to look ahead and share what they think the future holds.