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CD5 knockout using CRISPR boosts CAR T cell therapy efficacy

The effectiveness of CAR T cell therapy against a variety of cancers, including solid tumors, could be boosted greatly by using CRISPR-Cas9 technology to knock out the gene for CD5, a protein found on the surface of T cells, according to a preclinical study from investigators at the University of Pennsylvania's Perelman School of Medicine and Abramson Cancer Center.

19 Jul 2024

Novel method enables RNA detection with Cas12 nucleases

CRISPR-Cas systems, defense systems in bacteria, have become a plentiful source of technologies for molecular diagnostics.

17 Jul 2024

One-time prime editing targets common genetic cause of cystic fibrosis

Researchers optimized prime editing for the efficient correction of the cystic fibrosis transmembrane conductance regulator F508del mutation in human airway epithelial cells, achieving high correction efficiencies and functional restoration with minimal off-target effects.

15 Jul 2024

Gene editing silences mutant microRNA, restores hearing in adult mice

Researchers have used gene editing to restore hearing in adult mice with a type of inherited hearing loss. They showed that shutting down a damaged copy of a gene called a microRNA (miRNA) enabled the animals to regain hearing. The approach by a research team supported by the National Institutes of Health (NIH), reported in Science Translational Medicine, may eventually lead to potential treatments for inherited hearing loss in people.

13 Jul 2024

Prime editing offers promising path to permanent treatment for cystic fibrosis

Cystic fibrosis is one of the most common genetic disorders, causing thick mucus build-up in the lungs and other parts of the body, breathing problems, and infection.

11 Jul 2024

New gene editing technique offers hope for millions

A team led by Mass Eye and Ear researchers has demonstrated for the first time a successful restoration of hearing through a novel, in vivo genome editing approach in an adult mouse model with a form of inherited deafness caused by mutations in microRNA.

11 Jul 2024

Efficient multi-site genome editing with retron-derived multitrons

Genome editing has become a widely adopted technology to modify DNA in cells, allowing scientists to study diseases in the lab and develop therapies that repair disease-causing mutations.

10 Jul 2024

Scientists identify over 5,000 genetic variants that enable certain cancers to thrive

Researchers performed saturation genome editing of the BAP1, whose dysfunction is related to cancer and impaired neurodevelopment.

10 Jul 2024

Scientists unveil KLF15 transcription factor's role in white fat cells, opening new paths for obesity therapy

Researchers found that KLF15 is crucial for maintaining white adipocyte properties in subcutaneous fat, and its deletion induces beige adipocyte characteristics, suggesting new obesity treatment pathways.

4 Jul 2024

New hope for solid tumors with enhanced CAR-T cells

A promising therapy that treats blood cancers by harnessing the power of the immune system to target and destroy cancer cells could now treat solid tumors more efficiently. Thanks to a recent study published in Molecular Therapy – Methods & Clinical Development from Dan Cappabianca and Krishanu Saha at the Wisconsin Institute for Discovery, Chimeric Antigen Receptor (CAR) T-cell therapy can be improved by altering the conditions the T cells are grown in. And it was all discovered by chance.

3 Jul 2024

CHARMs: A new hope for prion disease therapy

Drug development is typically slow: the pipeline from basic research discoveries that provide the basis for a new drug to clinical trials to production of a widely available medicine can take decades.

27 Jun 2024

CRISPR-based genetic technique eradicates malaria mosquitoes with over 99% efficiency

Scientists at the University of California have developed a precision-guided sterile insect technique to eliminate Anopheles gambiae mosquitoes, achieving over 99.5% male sterility and 99.9% female lethality, effectively suppressing malaria transmission.

26 Jun 2024