News

CMN Weekly (13 August 2021)

Some of the best links we picked up around the internet

By: Karen O'Hanlon Cohrt - Aug. 13, 2021

Top picks

  • Research led by Jennifer Doudna at UC Berkeley has elucidated how the smallest known CRISPR-Cas system, CasΦ, recognises and cleaves double-stranded DNA. The researchers used cryo-electron microscopy to determine the structure of CasΦ in pre- and post-DNA-binding states. The findings were published in Nature Structural and Molecular Biology earlier this week. Last year, we interviewed two of the researchers involved in discovering the so-called tiny but mighty CasΦ. You can read that interview here.
  • A team of researchers led by Hans Clevers at Hubrecht Institute and collaborators in Holland have used prime editing to correct the underlying mutations in cystic fibrosis in human intestinal organoids (or mini organs), by replacing the mutated sequence with the healthy sequence. The study, published in Life Science Alliance this week, shows that prime editing is safer than editing with CRISPR-Cas9 with respect to off-target edits, and lab results so far indicate restoration of function in gene-edited organoids. We interviewed Hans Clevers last year after his group published work showing that base editing could be used to correct cystic fibrosis-causing mutations in gut organoids. You can read that interview here.

Industry

Industry quarter 2 financial updates

This week saw several additional quarter 2 finanical and business updates from companies working in the gene-editing space. You will find the highlights at the links below:

Research

  • CRISPR-associated transposase (CAST) systems, which are harnessed from cyanobacteria, use transposons to precisely and efficiently insert large DNA sequences into a genome. Researchers at Cornell University have now used cryo-electron microscopy to unravel a previously unexplained feature of CAST systems – that is, how they direct insertions in a single orientation at a precise distance from the programmed target sequence. The findings were published in Science today.
  • To explore the effect of gene-editing on cardiac disease associated with Duchenne muscular dystrophy (DMD), researchers led by Eric Olson (University of Texas Southwestern) used CRISPR-Cas9 to target exon 45 of the DMD gene in order to generate corrected DMD in patient-derived cells, wherein the DMD open reading frame was restored via reframing (RF) or exon skipping (ES). They found that it was possible to correct dysfunctional DMD cardiomycyes (CMs) by adenoviral delivery of Cas9-gRNA and that correction of DMD CMs post-differentiation reduced their arrhythmogenic potential. They also showed that transcriptional dysregulation in CMs and fibroblasts in DMD mice could be restored to similar levels as wildtype mice following CRISPR-Cas9 targeting of exon 45. The findings were published in Circulation Research earlier this week. You can read more about how CRISPR is used to develop new therapies for DMD in our earlier article.

Pioneers and patents

Conferences

Reviews

  • Gene drives gaining speed. Gene drives are selfish genetic elements that are passed down to progeny at super-Mendelian (>50%) frequencies. CRISPR-Cas9-based gene-drive systems have shown great potential for controlling the prevalence of vector-borne diseases, crop pests and non-native invasive species, but concerns reamin about their long-term effects in nature. This review summarises progress within CRISPR-based gene drives.

Huh, Heh, Wow

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News: CMN Weekly (13 August 2021)
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