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Published on October 11, 2023
Researchers have identified more than 1,000 functional new druggable cancer drug targets using a combination protocol involving gene base editing and chemical proteomics. The research, published in Nature Chemical Biology, focused on editing thousands of bases on more than 1,750 genes known to be correlated with cancer development. The work…
Published on July 5, 2023
Sickle cell disease (SCD) and beta thalassemia could be cured by gene therapy, but which approach would be most effective and safest? A team from St. Jude Children’s Research Hospital and the Broad Institute of MIT and Harvard may have the answer. They compared five gene editing strategies in CD34+ hematopoietic stem…
Published on May 24, 2023
Danish pharmaceutical company Novo Nordisk announced today that it has entered a research and development deal with Life Edit Therapeutics to leverage its next-generation gene editing technologies against a range of therapeutic targets. Under the collaboration agreement, Life Edit will receive an undisclosed upfront payment with the potential to earn…
Published on May 18, 2023
Diagnostics and life sciences company Revvity announced today that it has secured a non-exclusive license with AstraZeneca for the use of technology underlying its Pin-point single strand base editing system to support its gene editing activities. “Our fundamental goal for the Pin-point platform is to translate the technology from pre-clinical…
Published on March 21, 2023
Research led by the University of California, Los Angeles, shows base editing could be used to treat a rare immune disorder known as CD3 delta severe combined immunodeficiency (SCID). Writing in the journal Cell, the authors describe using base editing, a new and more precise version of gene editing than…
Published on January 13, 2022
A team of researchers led by David Liu, professor at Harvard University and the Broad Institute, have engineered DNA-free virus-like particles (eVLPs) that can efficiently deliver base editors, or Cas9 ribonucleoproteins, to multiple organs with minimal off-target effects. While base editing provides a powerful protocol for treating currently untreatable genetic…
Published on July 15, 2021
Scientists say that base editing proved itself efficient in correcting a mutation in patient cells with the monogenic disease Alpha-1 antitrypsin deficiency (AATD). The disorder is a common inherited disease that affects the liver and the lungs. Base editing is different from other forms of editing, including CRISPR, because the…
Published on November 15, 2023
Scientists have engineered a modified CRISPR technology targeting the epigenome that is capable of modulating T cell behavior. In the process, they discovered a master regulator of the genome that reprograms T cells and enhances their cancer cell-killing ability. Since CRISPR technologies were first developed, much of the interest has…
Published on September 14, 2023
A new method of genome manipulation can rapidly engineer RNA viruses through precise cleavage and repair, with potential applications for antiviral therapeutics, vaccines, and screening of mutations. The new recombinant technology enables specific deletions and insertions to the RNA of viruses. This is done through a combination of sequence-specific RNA…
Published on June 28, 2021
A team of researchers based in the U.S., the U.K., and New Zealand from Intellia Therapeutics, Regeneron Pharmaceuticals, and clinical partners showed in a study that the companies’ lead in vivo genome editing candidate NTLA-2001 generated a dose-dependent sustained reduction of protein linked to transthyretin (ATTR) amyloidosis following a single…
Published on March 13, 2024
Researchers at the Massachusetts Institute of Technology (MIT) report that their new approach of using CRISPR-based prime editing provides a faster and more efficient way to screen for cancer mutations in individual genes in their natural setting. This will allow researchers to better understand the role those mutations play in…
Published on January 3, 2024
Gene editing firm Tome Biosciences has acquired startup Replace Therapeutics less than a month after Tome emerged from stealth with $213M in funding. This development adds fuel to an already hot field. In early December, Vertex and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel [exa-cel]) for sickle cell became the first ever…
Published on December 11, 2023
In a landmark moment for sickle cell disease treatment and CRISPR, two gene therapies were approved for the condition by the U.S. FDA. One of them,Vertex and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel [exa-cel]), is the first ever FDA approved CRISPR/Cas9 genome-edited cell therapy. Simultaneously, Bluebird Bio received the FDA OK…
Published on November 29, 2023
Researchers at the Gladstone Institutes have developed a CRISPR-based “genome shredding” technique that shows promise in treating glioblastoma, an incurable brain cancer. Their research was published this week in the journal Cell Reports. Much of the work done to develop the technique was done in the lab of Jennifer Doudna,…
Published on July 28, 2023
An RNA-based gene editing tool that alters red blood cells inside the body could provide a simpler and less toxic route to treating conditions such as sickle cell anemia and beta thalassemia, researchers report. The discovery, outlined in Science, offers an alternative to current gene therapy for these blood disorders,…