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  • Gene Therapy Vehicle for Sickle-Cell Disease Shifts Forward

    In animal models, a new forward-oriented lentiviral vector outperforms the reverse-oriented vectors ordinarily used to deliver gene therapies for sickle-cell disease. The new vectors, which retain the sensitive intron 2 element, can increase vector titers sixfold and increase transduction efficiency four- to tenfold.
    Kevin Ness speaking at the Global Synthetic Biology Summit

    One-Stop-Shop Genome Editing Product Launched by Inscripta

    At the SynBioBeta 2019 conference in San Francisco, Inscripta launched the Onyx—a fully automated benchtop instrument for genome-scale engineering. The CRISPR-mediated, massively parallel platform—which includes software, consumables, instrument, and assays enables researchers to engineer microbial libraries containing the full breadth and scope of possible edit types, in their own labs.
    paper test strip

    CRISPR Dx May Help Nix Disease

    Every inquisitive diagnostician looking to uncover infectious disease or cancer will want to give CRISPR-based tests a close look. Their paper strips and smartphone apps can deliver the inside skinny.
    Data behind outline of man with DNA surrounding him

    In AI We Trust? The Future of Genomic Medicine

    Genomic medicine is borrowing from Greek theatre a device called God in the Machine. In genomic medicine, the device manifests as AI technology, but it can still resolve complicated situations.
    suspension culture in MilliporeSigma lab

    Gene Therapies Are Being Brought to a Wider Audience

    To reach the clinical big time, gene therapy producers need to get their act together, improving transfection, ensuring consistency, enhancing purity and increasing yields.

    Ultracentrifuge Designs Enable Linear Process Scaling

    Sponsored content brought to you by In the gene and cell therapy field, there is a clear need to have cost-effective, scalable processes in the...
    Malvern Panalytical Empyrean Students

    Next-Generation Light Scattering: Determining AAV Titer with Multi-Angle Dynamic Light Scattering

    There is high demand for a method that can rapidly and directly measure AAV particle concentration. With the development of Multi-Angle Dynamic Light Scattering (MADLS?), the Zetasizer Ultra from Malvern Panalytical provides both rapid, accurate viral titer measurements and aggregation profiles in minutes.
    Adeno-associated viruses

    Precise CRISPR-Cas9 Variants Come in Small Packages

    Cas9 from Streptococcus pyogenes (SpCas9) is a precise nuclease, but it can be too large for the payload-limited AAV vector commonly used to deliver gene editing components into cells. The wild-type Cas9 from Staphylococcus aureus (SaCas9) easily fits inside the AAV, but it lacks SpCas9’s precision. Fortunately, rationally engineered SaCas9 variants have been shown to have high genome-wide specificity.

    Beam Therapeutics Files for IPO, Pursuing Treatments Using Base Editing

    Beam Therapeutics has disclosed plans to go public, submitting plans for an initial public offering (IPO) that would raise up to $100 million for the year-old company founded by gene editing pioneers to develop new treatments based on base editing.

    Ultrasound Scanning Monitors Gene Expression in Mammalian Cells

    A genetic program adapted from bacteria for mammalian cells enables the expression acoustic reporter genes. These genes encode proteins that contribute to the assembly of proteinaceous gas vesicles, which may serve as ultrasound contrast agents. This novel live-cell imaging technology could allow gene expression to be monitored in the deep tissues of living animals.

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