Your missing links are here (23 October 2020)
By: Karen O'Hanlon Cohrt - Oct. 23, 2020
Top Picks
- Strength in stubbornness: A 2020 Nobel winner reflects on her career. 2019 Interview with Jennifer Doudna with National Geographic for its book ‘Women’.
- The very first World CRISPR Day took place this week. The event was organised by genome engineering company Sythego and featured a key address by recent Nobel Laureate Jennifer Doudna as well as well as in-depth discussion of many areas of basic and clinical CRISPR research. Catch up with our coverage of the event.
- Researchers at University of North Carolina School of Medicine utilise CRISPR-Cas9 to reduce the severity of disease in mouse models of Angelman syndrome, a severe neurodevelopmental disorder associated with intellectual and developmental disabilities, seizures, and problems with speech, balance, movement, and sleep. Research findings published in Nature.
Research
- Researchers from Germany and the US develop compact Cascade–Cas3 system for targeted genome engineering. The system that can make larger genomic deletions than is possible with CRISPR-Cas9, with applications in research and potential for gene insertion and replacement strategies within agriculture and therapy.
- US research team use in vivo delivery of an adenine base editor and a single guide RNA targeting a single mutation to restore sight in adult mice with the inherited retinal disease retinitis pigmentosa. Research findings published in Nature Biomedical Engineering.
- US and Australian researchers use CRISPR to validate genetic targets that may be used in control strategies against Culex moquitoes, a globally widespread vector of human and animal pathogens. Research findings shared on preprint server bioRxiv.
- Computer scientists at University of Oxford report crisprSQL, a novel database platform for CRISPR/Cas off-target cleavage assays. The free resource, found here, is an interactive and bioinformatically enhanced collection of CRISPR/Cas9 off-target cleavage studies that aims to support the fields of cleavage profiling, gene editing safety analysis and transcriptomics. Read the research article here.
Delivery
Researchers in China describe design of a nucleic acid nanogel-based mRNA delivery system that yields robust transgene expression and CRISPR-Cas9 gene editing.
Diagnostics
- CASMEAN is a Cas12a-based reagents-loaded, automated assay that incorportates recombinase-aided amplification (RPA) to detect nucleic acids. Researchers demonstrate detection of Pseudomonas aeruginosa within 1.5 hours.
Industry
- IDEAYA Biosciences, a US oncology-focused precision medicine company has entered into a target and biomarker discovery partnership with the Sellers Laboratory at the Broad Institute of MIT and Harvard. The programme will exploit the Broad’s CRISPR paralogue screening platform to evaluate functionally redundant paralogous genes across ovarian cancer subtypes and to generate novel Synthetic Lethality-based target and biomarker discoveries.
- AavantiBio launches with $107 M Series A financing from Perceptive Advisors, Bain Capital Life Sciences, RA Capital Management and Sarepta Therapeutics. AavantiBio to pursue a diverse pipeline including a lead programme in Friedreich's Ataxia, a rare genetic disease that affects the heart and brain.
- Danish biotech Snipr Biomewelcome new Chief Medical Officer Milan Zdravkovic who will lead the company towards clinical trials early next year for it's CRISPR-based antibacterial platform. You can read more about how Snipr Biome is turning CRISPR into a bacteria killer in our interview with their CEO late last year.
Vision and Opinion
- Gene Editing Sperm and Eggs (not Embryos): Does it Make a Legal or Ethical Difference? Perspectives piece by Professors in Law and Medical Science discusses how Congress-level decisions on genetic editing of human embryos will affect the science, ethics and financing of genome editing for decades to come.
Review
- Review on the potential of CRISPR for research and therapeutics within the Alzheimer's disease (AD) field.
Clinical Trials
- CRISPR TherapeuticsReports Positive Top-Line Results from Its Phase 1 CARBON Trial of CTX110™ in Relapsed or Refractory CD19+ B-cell Malignancies
- Intellia to begin in vivo liver CRISPR knockout phase I clinical trial to cure all forms of severe hereditary liver disease called hATTR-PN (hereditary transthyretin amyloidosis with polyneuropathy).
COVID-19
- CRISPR-engineered human cell lines targeting more than 300 genes may be an important tool to understand host responses to COVID-19 and identifying new avenues to treatment. Collaborative research between genome engineering company Synthego and world-renowned Krogan Lab at University of California, San Francisco.
- Indian labs already approved to use rapid paper-based SARS-CoV-2 test FELUDA do not need to perform further RT-PCR based confirmatory tests, in new advisory from Indian Council of Medical Research (ICMR).
Heh, huh wow
- Norwegian fisheries research institute Nofima will use the gene-editing tool CRISPR-Cas9 to assess why Pacific salmon species are less susceptible to sea lice than Atlantic salmon. A means to make these salmon lice-resistant could be the solution to a problem that currently costs the Norwegian salmon sector the equivalent of 543 M USD annually.
- Argentinian have produced what is believed to be the world’s first genetically-edited horse embryo in an attempt to identify mutations to improve sport performance through precision breeding.
Tags
CLINICAL TRIALS
IND Enabling
Phase I
Phase II
Phase III
IND Enabling
Phase I
Phase II
Phase III
IND Enabling
Phase I
Phase II
Phase III