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Published on September 25, 2024
A preclinical mouse model that uses CRISPR reveals how prostate cancer spreads or metastasizes. The model developed by a team of researchers led by Weill Cornell Medicine demonstrates the routes prostate cancer metastatic cells take as they travel through the body, and could lead to more effective treatments. Their findings…
Published on June 20, 2024
A novel technique could potentially eradicate the toxic conditioning process required to effectively transplant hematopoietic stem and progenitor cells (HSPCs), which play a crucial role in treating blood disorders like sickle cell disease and β-thalassemia. Researchers from Stanford University and the University of California, San Francisco (UCSF) utilized CRISPR-Cas9 to…
Published on June 18, 2024
New research has unveiled a key relationship between two proteins that causes a unique subtype of pancreatic ductal adenocarcinoma cancer (PDAC) known as basal-like pancreatic cancer, which affects around 15% of PDAC patients. This subtype exhibits an unusual transformation, causing the pancreatic cells to take on characteristics akin to skin…
Published on June 12, 2024
In the James Bond franchise, a cunning character named Q leads the fictional research and development division of the British Secret Service, which oversees top-secret field technologies. If 007 needed it, Q had already created it. That’s the reputation that KSQ Therapeutics’ CEO, Qasim Rizvi, MD, who also goes by…
Published on March 14, 2024
A new test uses CRISPR to detect Burkholderia pseudomallei, the bacterium that causes melioidosis, in hours rather than the typical three to four days currently needed. It also has 93 percent sensitivity compared to 66.7 percent for current tests. This could be a critical advance, since many patients die of…
Published on January 31, 2024
Researchers in Japan and the United States have developed a new CRISPR-based technology that increases the recognition of cancer cells to the immune system. The findings, published in Proceedings of the National Academy of Sciences, were led by Koichi Kobayashi, professor at Hokkaido University and Texas A&M Health Center, and…
Published on January 17, 2024
The U.S. Food and Drug Administration (FDA) has approved Vertex and CRISPR Therapeutics’ Casgevy (exa-cel), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. This is the latest sign the gene and cell therapy market is maturing. This market is…
Published on January 10, 2024
Nobel laureate Jennifer Doudna will lead a Danaher and Innovative Genomics Institute (IGI) collaborative center that will use CRISPR gene-editing technology to develop treatments for rare and other diseases. The center, called the Danaher-IGI Beacon for CRISPR Cures, will use CRISPR-based gene editing via a unified research, development, and regulatory…
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 November 16, 2023
In a big first, Vertex Pharmaceuticals and CRISPR Therapeutics announced today that the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization for Casgevy (exagamglogene autotemcel [exa-cel]) for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Casgevy is a CRISPR/Cas9 gene editing…
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 July 17, 2023
CRISPR-based therapies for Alzheimer’s are starting to emerge. At least two possible candidates were reported this week at the Alzheimer’s Association International Conference (AAIC) 2023. One aims to reduce the impact of APOE-e4, the strongest known Alzheimer’s risk gene. The other affects the brain’s production of beta amyloid, which is…
Published on June 5, 2023
A little over a decade ago, a paper was published in Science1 that made science fiction a reality. Emmanuel Charpentier, Jennifer Doudna, and colleagues reported that they had identified a means of harnessing an element of a bacterial immune system to carry out genome editing in a way that was…