Your Missing Links Are Here (12 March 2021)

Top picks

• CRISPR could be the end of opioid abuse – a story that has hit many outlets this week after researchers in the U.S. show that CRISPR-based repression of NaV1.7, a sodium channel protein associated with pain perception led to analgesic (pain-relieving) effects in mice. The research, published in Science Translational Medicine, describes how targeted epigenetic repression of NaV1.7 by CRISPR-Cas9 technology and zinc finger proteins - a methodology that the scientists have called LATER - might have potential for long-lasting pain relief as a much needed alterative to opioids, which are highly addictive and currently the gold standard for chronic pain management.
• This is the year that CRISPR moves from lab to clinic: a brief status update from Nobel Laureate Jennifer Doudna.

Research

• Researchers in Japan show that optimised nano-micelles can induce efficient genome editing in the mouse brain. Nano-micelles were used to co-deliver Cas9-encoding mRNA and gRNA and the researchers could monitor gene editing in the mouse brain via expression of a red fluorescent reporter protein. The work was published in Journal of Controlled Release.
• Researchers in the U.S. investigated how Cas12a accesses and cleaves human DNA in nucleosomes, and found that nucleosome unwrapping regulates accessibility of DNA to Cas nucleases. The team proposes ways to improve DNA targeting in eukaryotic cells by relaxing DNA wrapping within nucleosomes. The findings were published in Science Advances this week.
• Researchers in China have developed a dissolvable microneedle patch that can mediate transdermal co-delivery of CRISPR-Cas9 ribonucleoproteins targeting an inflammatory protein NLRP3 and glucocorticoids for the effective treatment of inflammatory skin disorders. The patch dissolves quickly to deliver its cargo locally and mediate anti-inflammatory effects at the site of inflammation, with promising results in in vivo mouse models for atopic dermatisis and psoriasis. The findings were published in Science Advances this week.
• Research led by Jennifer Doudna reveals the mechanisms by which Cas9 precisley trims 30-base pair DNA molecules from foreign DNA bodies to generate immunological memory in prokaryotes. The findings were published in Nucleic Acids Research this week.

Industry

• Earlier this week, Intellia Therapeutics presented preclinical proof of concept for non-viral CRISPR-Cas9-based in vivo genome editing of bone marrow and haematopoietic stem cells in mice at the virtual Keystone eSymposium. The data supports the potential for in vivo CRISPR therapy for a range of blood disorders.
• Sherlock Biosciences named to Fast Company’s (2021) annual list of the World’s most innovative companies. This prestigious list honours the businesses that have demonstrated resilience in the past year and have transformed challenges into impact-making processes. Last year, Sherlock was the first company to receive FDA approval for a CRISPR-based tool for its Cas13-based COVID-19 test. Read our interview with the CEO here.
• Copenhagen-based Cas12a gene-editing company Twelve Bio has just secured investment from London-based global venture capital company Arix Bioscience. Twelve Bio is developing novel engineered Cas12a nucleases for therapeutic gene editing, and Arix is now the sole investor with a 49% ownership stake. Read our interview with Twelve Bio last year to learn more about their unique approach to CRISPR-based therapies.
• CRISPR Therapeutics announced that it will present a poster at the American Association for Cancer Research 2021 Annual Meeting on the 10^th April (virtual). The posted is entitled ‘CD70 knockout: A novel approach to augment CAR-T cell function’.
• ERS Genomics and Setsuro Tech sign CRISPR-Cas9 license agreement. ERS Genomics holds the intellectual property of CRISPR Nobel Laureate Emmanuelle Charpentier and the agreement will see Setsuro, a biotech start-up using genome-editing techniques to develop and supply cell and animal models, gain access to ERS Genomics’ CRISPR-Cas9 patent portfolio for Japan.
• The U.S. National Institute of Allergy and Infectious Diseases (NIAID) has awarded $3 million to Boston-based PhagePro to target antibiotic resistance genes in Vibrio cholerae using a phage-encoded CRISPR-Cas system to improve efficacy of phage prophylaxis.
• Last month, we shared news that Bluebird Bio had halted its clinical studies for a gene therapy for sickle cell disease (SCD) after two patients developed leukaemia-like cancer. This week, the company shared a press release revealing that the lentiviral vector used in the new therapy was unlikely to have caused leukaemia in the clinical study of LentiGlobin for SCD.

Vision and opinion

• Book review in Science journal. Review of Walter Isaacon’s “The Code Breaker” by George J. Annas.
• CRISPR Gene Therapy Gives Me Hope for a Future Without Hemophilia: A piece about CRISPR written by a father whose two sons have haemophilia.
• A biographer and a bioethicist take on the CRISPR revolution. Nature journal book review on "CRISPR People: The Science and Ethics of Editing Humans" by Henry T. Greely (MIT Press, 2021) and "The Code Breaker: Jennifer Doudna, Gene Editing, and the Future of the Human Race" by Walter Isaacson (Simon & Schuster, 2021).

Conferences and events

• Registration and abstract submission are open for the 14th annual CRISPR conference. This year's conference will be held virtually from the 1st-10th June and will be hosted by Dupont. Read more about the event, sign up and view the invited speaker list at the conference website here.
• Genome Editing 2021 virtual conference program now available here. The event, which takes place on the 30th March, will cover functional genomics technologies for translational research and therapies.
• CRISPR in Drug Discovery: From Targets to Therapeutics: a virtual event running from 23rd to 24th March 2021 that will focus on the application of genome editing to identify drug targets, develop better disease models and advance genome-editing therapies.

Podcasts and Interviews

• Master wordsmith Walter Isaacson takes the media by storm with his latest book "The Code Breaker". Hear him discuss CRISPR in the short (link), the intermediate (link) and the long interview (link).