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Lipid nanoparticles have been used to encapsulate CRISPR-Cas9 and deliver it to cells in mice, where it was highly effective at knocking down expression of a target protein.
The team used these models to show drug responses and established a CRISPR-screening platform to identify potential therapeutic targets for non-alcoholic fatty liver disease.
US researchers evaluate an experimental gene therapy in the first ever nonhuman primate model for Usher Syndrome.
The scientists say that the engineered CRISPR enzymes could overcome key limitations for eventual use to treat genetic diseases irrespective of a patient’s particular mutation.
US researchers outline the development of a new system for testing and developing CRISPR-based gene drives in the laboratory, and safely converting them into tools for potential real-world applications.
US researchers develop a dual-action cell therapy engineered to eliminate established tumours and train the immune system to eradicate primary tumour and prevent cancer’s recurrence.
The scientists discovered that dual knockout of genes in organoids grown from human tissue can generate a model of a potential therapeutic target for gastroesophageal junction cancer.
Dr Douglas Ross-Thriepland and Dr David Walter, from the Functional Genomics Centre (FGC) – a joint venture between Cancer Research UK’s (CRUK) drug discovery engine, Cancer Research Horizons, and AstraZeneca – speak to Drug Target Review about their work. The centre’s focus is on genetic screening; cancer models; CRISPR reagent…
In this article, Drug Target Review’s Izzy Wood and Ria Kakkad share some of the most ground-breaking moments from drug discovery this year.
The tumour microenvironment can prevent some T cells from carrying out their immunotherapeutic duties. In this Q&A, Dr Brian Shy, Clinical Instructor at the University of California, San Francisco (UCSF) Department of Laboratory Medicine, describes a recent study wherein he and fellow scientists discovered a strategy to strengthen T cells…
Sloan Kettering Institute (SKI) researchers have developed a new machete technique to slice into the cancer genome and study copy number alterations.
This article highlights five of the latest findings revealed using CRISPR that could be used in the development or design of new therapies.
US scientists, using CRISPR technology, removed specific genes in humans to allow the immune system to be more activated against cancer.
US researchers have engineered bacteriophages to deliver CRISPR-Cas payloads for targeted editing of a bacterium.
The researchers used a zebrafish model to test mutations in Mycn, a gene which causes Feingold syndrome.
