Scientists led by the Children's National Hospital faculty found that a novel drug derived from scorpion venom reversed motor deficits in pre-clinical models. This finding could offer hope to an estimated 119,000 children born with fetal alcohol spectrum disorder (FASD) worldwide each year.
Sponsor: Hera BioLabsIn this GEN webinar, sponsored by Hera BioLabs, we will hear from scientific experts about the design and implementation considerations for Cas-CLOVER, as well as validation data for the use of this gene-editing system in human and CHO cells, as well as yeast and plants.
Arginine residues in the Cas9 bridge helix influence guide RNA and the specificity of target DNA binding and cleavage. By swapping out selected arginine residues, which contact the phosphate backbone of guide RNA, the formation of a stable R-loop is favored, researchers generated Cas9 variants that produced fewer off-target effects. One variant, R63A/Q768A, showed increased specificity in human cells.
Scientists headed by a team at the Donnelly Centre for Cellular and Biomolecular Research have developed a CRISPR-based system, called CHyMerA, that can edit multiple target sites in the genome at the same time. The new system, combines both Cas9 and Cas12a CRISPR-associated enzymes, with machine learning-optimized hybrid Cas9-Cas12a guide RNAs.
Microbiologists from two NC State colleges teamed with startup company Locus Biosciences to test the effectiveness of using a bacteriophage to carry a programmable CRISPR to specifically target and eliminate C. difficile bacteria.
Ori’s platform reportedly enables therapy developers and contract manufacturers to achieve automated CGT manufacturing in a closed platform.
A former GlaxoSmithKline campus in Upper Merion Township, PA and an adjacent property are being transformed into a $1.2 billion co-working campus focused on cell and gene therapy development.
Researchers at the Northwestern University developed a system, ribosome synthesis and evolution (RISE), which is an important step toward using ribosomes beyond their natural capabilities. The result could be new ways to synthesize materials, like nylon, or therapies like new antibiotics that could address rising antibiotic resistance.
Besides developing platforms that elaborate on good old Cas9 in various ways, researchers are exploring alternative Cas enzymes, such as Cas3, Cas12, and Cas13, as well as CRISPR-based transposon systems.
Sponsor: Wyatt TechnologyIn this GEN webinar, sponsored by Wyatt Technology, we will discuss how dynamic light scattering (DLS) and multi-angle light scattering combined with size exclusion chromatography (SEC-MALS) or field-flow fractionation (FFF-MALS) characterize AAV, LV, and other gene delivery vectors.
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