Your missing links are here (18 December 2020)

Top Picks

• Can CRISPR-Based Covid-19 Testing Using Smartphones Slow The Pandemic? Discussion on recent advances in CRISPR-based approaches to COVID-19 testing that incorporate smartphone technology for rapid and high capacity screening.
• The first patients to receive CRISPR therapy CTX001 for sickle cell disease appear to be cured and continue to thrive. Vertex Pharmaceuticals and CRISPR Therapeutics, who are co-developing CTX001 as a one-off curative therapy for sickle cell disease, shared positive clinical data about CTX001 last week.

Research

• Earlier this month, researchers in the US reported a one-step CRISPR-Cas9-based method that can produce mice with genetically engineered human B cell receptors in just a few weeks. This removes a serious bottleneck in applications that rely on mice to produce human antibodies, e.g., within vaccine development and research to understand human immune responses.
• Researchers use CRISPR–Cas13 to screen for functional circular RNAs. These poorly understood but widely expressed RNAs are covalently closed loops of single-stranded RNA that are produced by back-spliced exons. A lack of methods to discriminate between circular RNAs and mRNAs has hampered research in this area but they have recently been linked to cancer. The new findings were published in Nature Methods.
• CRISPR-Cas9-mediated disruption of the GAD1 gene in rats leads to complex behavioral changes that may have implications for schizophrenia and may warrant the use of a rat model for this disease. GAD1 encodes the GABA synthetic enzyme glutamate decarboxylase 67-kDa isoform, which has been linked to schizophrenia, although a causal relationship has not yet been established.
• A CRISPR interference (CRISPRi) platform is developed for large-scale perturbation of functional long non-coding RNA loci in human cells, providing clues about their function and cell-type specificity.

Industry

• Californian gene-editing company Graphite Bio obtain IND clearance to initiate a Phase 1/2 trial for next-generation gene-editing therapy GPH101 in sickle cell disease. Enrolment for the clinical trial called CEDAR is set to start in in early 2021, and GPH101 represents the first experimental treatment that aims to correct the underlying mutation in sickle cell disease.
• CRISPR Therapeutics receives grant from Bill & Melinda Gates Foundation to explore in vivo gene-editing therapies for the treatment of HIV.
• Metagenomi, a next generation gene-editing spinout from UC Berkeley (2018) comes out of stealth mode following a USD 65 million Series A financing round. The company, with locations in California and Germany, is combining the power of big data and metagenomics to screen thousands of genomes from microorganisms around the world with which to build a proprietary suite of CRISPR-based gene-editing systems for novel gene therapies.
• Allogene Therapeuticsand Overland Pharmaceuticals join forces in Greater China to develop and commercialise AlloCAR T™ cell therapies for haemological and solid cancers. The venture includes the formation of Allogene Overland Biopharm (Allogene Overland), which will have an exclusive license to develop, manufacture and commercialise specific Allogene CAR T-Cell candidates in the licensed territories.

COVID-19

• Sherlock Biosciencesreceives a $5 million grant from the Bill & Melinda Gates Foundation to advance INSPECTR™, its instrument-free, synthetic biology-based molecular diagnostics platform for COVID-19 self-testing. This grant is just a fraction of the foundation’s $250 million commitment to the COVID-19 response.
• US and Chinese researchers describe a new portable, highly sensitive CRISPR-based saliva test for SARS-CoV-2 with a 15 minute turnaround time. The assay, which neither requires RNA isolation nor laboratory equipment, relies on Cas12a activity to enhance the signal generated from an amplified viral RNA target, which is then stimulated by laser diode integrated into a smartphone-based fluorescence microscope readout device. The findings were published in Science Advances.

Reviews and Roundups

• Gene Editing and Genotoxicity: Targeting the Off-Targets. New peer-reviewed survey of the currently available techniques to predict expected and unexpected modifications that occur during gene editing. Benefits and limitations, as well as applicability to clinical settings are discussed.
• CRISPR Will Change the World. Here’s What’s Already In the Works. A sampling from UC Berkeley’s Alumni Association of how gene-editing is being used to rewrite the world’s DNA.

Opinion

• CRISPR Therapeutics Deserves Attention After Positive CRISPR/Cas9 Patient Data. Financial diagnosis of CRISPR Therapeutics following recent positive Phase 1 data for CTX001, which is being co-developed with Vertex Pharmaceuticals.

Quality Control

• US National Institute of Standards and Technology (NIST) launch first genome editing lexicon. According to NIST: This lexicon was developed to provide a unified reference set of terms and technical definitions that standardizes their use and meaning to serve the needs of the biotechnology community.’ Find the lexicon here.

Interviews, Podcasts and Webinars

• Watch Out Bluebird Bio, CRISPR Is on Your Heels. Video discussion and transcript following recent American Society of Hematology Meeting, about the differences between CRISPR Therapeutics and Vertex Pharmaceuticals co-developed CTX001 CRISPR therapy and Bluebird Bio’s approved lenteviral-based gene therapy for beta-thalassemia.
• Podcast: How do COVID vaccines work? CRISPR kills cancer; Danish study debunks mask mandates? In the latest episode of Science Facts and Fallacies, Geneticist Kevin Folta and Genetic Literacy Project editor Cameron English break down some of the recent news stories to hit mainstream media. They discuss the recent breakthrough made by Dan Peer’s lab at Tel Aviv Univesity, which demonstrated that CRISPR could chop up tumour DNA to kill cancer cells, which may be a game-changer in cancer treatment. We recently interviewed researchers from Dan Peer's lab about this work.
• On the 14^th January 2021, a group of 10 scientists from Wisconsin will disusss CRISPR in a live webinar hosted by the Wisconsin Technology Council. The panel will focus on where CRISPR is leading within cell and genetic therapies as well as antibiotic resistance.

Heh, huh, wow

• CRISPR squids made the list of Top Science Photos From 2020. The incredible photos were taken by researchers, who in July reported the first use of CRISPR editing in cephalopods, a class of marine invertebrates.