Your missing links are here (27 November 2020)
By: Gorm Palmgren - Nov. 27, 2020
- A study from the US-based National Human Genome Research Institute concludes that clinical trials of genome editing to treat sickle cell anaemia must be designed with input from patients and their parents and physicians. The study finds that this group of people make a tough decision when deciding whether to participate in a CRISPR-based therapy. If not adequately informed, seriously ill patients could overestimate the benefits of early-phase studies while underestimating the risks.
- Precision BioSciences' proprietary gene-editing system ARCUS - using the naturally occurring homing endonuclease I-CreI - has been licensed to the American pharmaceutical company Lilly. The license includes a gene target for Duchenne Muscular Dystrophy and two other undisclosed targets as well as another three optional targets. Precision will receive $100 million cash upfront and an equity investment by Lilly of $35 million as well as potential milestone payments and royalties.
- Studying the crystal structure of Cas10d from the archaea Sulfolobus islandicus, researchers from Aarhus University, Denmark have elucidated how viral anti-CRISPR proteins work. The new results give scientists insights into the arms race that is continually taking place in nature and suggests how it might evolve in the future.
- Expression of DLGAP2 - a gene required in the synapses of neurons involved in learning and memory - have been discovered to be linked to Alzheimer's dementia in humans. CRISPR vectors controlling the expression of DLGAP2 are already commercially available and might play a future role in gene-editing therapy of dementia.
- CRISPR-Cas9 HDR has been used to introduce the light chain of an HIV broadly neutralising antibody into an IgH gene in B-cells. A new study shows, that when such engineered B cells are reintroduced to the body, they can mature into memory cells and plasma cells that produce high levels of protective antibodies for long periods in the body.
Industry & Clinical
- American geneticist George Church has launched 64x Bio, a new startup company. The startup will discover and design enhanced mammalian cell lines to increase the efficiency of viral vector production and reduce the cost of gene therapy manufacturing. The startup uses the VectorSelect platform that leverages leading-edge DNA library synthesis, next-generation sequencing, and a proprietary genetic barcoding method.
- Mammoth Biosciences has signed agreements with MilliporeSigma and Hamilton Company targeting commercialization of Mammoth's DETECTR BOOST™ platform, a high-throughput CRISPR-based SARS CoV-2 test. You can read more about the DETECTR diagnostic kit here.
- Researchers at the University of Connecticut, USA have developed a so-called digital warm-start CRISPR (WS-CRISPR) assay for sensitive, quantitative detection of SARS-CoV-2 in clinical samples. The method combines combines a low-temperature reverse transcription dual-priming mediated isothermal amplification (RT-DAMP) and CRISPR-Cas12a-based detection in one-pot.
- Another digital one-pot CRISPR/Cas assay for rapid and sensitive detection of SARS‐CoV-2 has been developed by researchers at the Johns Hopkins University, USA. The so-called deCOViD assay is realized through tuning and discretizing a one‐step, fluorescence‐based, CRISPR/Cas12a‐assisted reverse transcription recombinase polymerase amplification assay into sub‐nanoliter reaction wells within commercially available microfluidic digital chips.
- Packing CRISPR reagents into metal-organic frameworks (MOFs) enhanced with a green tea phytochemical coating, researchers from RMIT University in Melbourne, Australia have achieved high cellular uptake in prostate cancer cells. MOFs can be manufactured at industrial scale quantities and thereby potentially reduce the cost of effective CRISPR delivery systems.
- Researchers from the University of Minnesota Medical School gives an update on emerging liver targeted gene therapies in a review in Drug Discovery Today: Technologies. Advances in CRISPR and similar technologies like base editing and prime editing are discussed as well as other forms of gene therapy.
- In a review in Frontiers in Genome Editing, researchers from University College London, UK look at new technologies to evaluate the safety and genotoxicity of gene-editing technologies like CRISPR/Cas, TALEN and ZFN. The review surveys the current techniques, highlighting benefits and limitations, and discusses approaches that may achieve sufficient accuracy and predictability for application in clinical settings.
- New advances in the CRISPR–FokI dead Cas9 (fdCas9) strategy is discussed in a review in Cells. fdCas9 has been reported to have almost complete elimination of any off-target effects.
Vision and Opinion
- In an easy-to-read piece in US News, science writer Arlene Weintraub tells the story of how CRISPR has become an often essential part of CAR-T and other therapies in the fight against blood disorders.
Heh, huh wow
- In an opinion requested by the European Commission, the European Food Safety Authority (EFSA) Panel on Genetically Modified Organisms concludes that the current strict rules on GM crops are only partially applicable to CRISPR plants. Risk assessment of gene-edited plants that don't contain exogenous DNA should not be subject to requirements that are linked to the presence of exogenous DNA.
- Chinese researchers have used CRISPR/Cas9 to induces exon skipping in the OsBADH2 gene and thereby facilitated the development of fragrant rice, which is is one of the most important rice quality traits.