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News: CMN Weekly (17 December 2021) - Your Weekly CRISPR Medicine News

Some of the best links we picked up around the internet

By: Karen O'Hanlon Cohrt - Dec. 17, 2021

Top Picks

  • Copenhagen-based SNIPR BIOME announced earlier this week that the European Patent Office has granted the company with a fundamental patent in the field of CRISPR-based targeting in microbiomes. According to a company press release, the broad protection afforded by this patent recognises pioneering work by SNIPR scientists as innovators of CRISPR-Cas modulation in microbiomes.

Clinical

Industry

  • Canadian biotech company Defence Therapeutics, which focuses on the development of vaccines and therapeutics for cancer and infectious diseases, has announced in a press release that its AccumTM platform can potently enhance Cas9 delivery in target cells. The company reported that the accumulation of AccumTM-linked Cas9 inside the nucleus of mammalian cells was at least 9-fold higher than free Cas9 following a single dose in in vitro experiments. The mechanism of action of AccumTM has not been disclosed.
  • In a press release this week, Allogene Therapeutics reported that it will expand its headquarters in South San Francisco to support company growth and pipeline innovation. The company currently has 10 gene-edited cell therapy programmes in its pipeline (all of which are being developed for cancer treatment), 6 of which are at clinical stage (Phase 1).

Research & Reviews

  • In an article published in Nature Communications last week, scientists in Japan report that low immunogenicity of lipid nanoparticles allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice. These findings present a possible solution to the immunogenicity associated with adeno-associated viral vectors, which are the most widely-explored vehicle for in vivo delivery, and may be useful in the development of new therapies to treat skeletal muscle disorders.
  • Researchers in the US describe the detailed methodology behind their recently developed "universal" platform that can incorporate multiple vaccine targets into the same nanoparticle scaffold by CRISPR engineering of bacteriophage T4. The authors report that a T4-COVID vaccine designed with this technology elicited broad immunogenicity and complete protection against virus challenge in a mouse model. The findings were published recently in Methods in Molecular Biology.
  • Researchers in the UK demonstrate the application of dFnCas12a-VPR for multiplex gene regulation in mammalian cells. dFnCas12a-VPR is a nuclease dead form of Cas12a from the bacteria Francisella novicida that possesses a shorter PAM sequence than Acidaminococcus sp. (As) or Lachnospiraceae bacterium (Lb) Cas12a variants, enabling denser targeting of genomic loci without loss of targeting efficiency. The researchers observed that dFnCas12a-VPR could be used as a synthetic transcription factor that offers a flexible tool for transcriptional modulation, and that it further expands our understanding of the design capabilities and limitations related to multiplexed targeting. The findings were published in Nucleic Acids Research this week.
  • Scientists in Japan show that CRISPR-Cas9-mediated base editing enables a chain reaction through sequential repair of sgRNA scaffold mutations. The findings were published in Science Reports this week.
  • Towards CRISPR powered electrochemical sensing for smart diagnostics. An opinionated review that examines the rapidly evolving applications for CRISPR-Cas integrated electrochemical biosensor detection systems and the future scope associated with such diagnostics.
  • CRISPR/Cas9 in Gastrointestinal Malignancies. This review from scientists in Germany discusses recent applications for CRISPR-Cas9-based genome editing in gastrointestinal cancer research including colorectal cancer, gastric cancer, oesophageal cancer, pancreatic duct adenocarcinoma, and hepatocellular cancer. These applications include functional studies of candidate genes in cancer cell lines or organoids in vitro as well as in murine cancer models in vivo, library screening for the identification of previously unknown driver mutations and even gene therapy of gastrointestinal cancers.

Highlights from 63rd American Society for Hematology (ASH) Annual Meeting

The ASH meeting look place from December 11th-14th virtually and in Atlanta. Here are the highlights from companies working in the therapeuic gene-editing space:

Webinars

Huh, Heh, Wow

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