Grants and Contributions

There are about 7,000 different hereditary diseases each due to the mutation of a gene. These genes are expressed in different cells. The treatment of these diseases requires the delivery of a normal gene or of gene modifying agents to the appropriate cells, i.e. those involved in the disease.

The CRISPR/Cas system has revolutionized the analysis of gene function in mammalian cells. Cas cuts DNA at specific genomic region by associating with a single guide RNA (sgRNA) molecule whose sequence is complimentary to the genomic DNA to be targeted.

The CRISPR/Cas system has revolutionized the analysis of gene function in mammalian cells. Cas enzymes cut DNA at a specific genomic site as targeted by a single guide RNA (sgRNA) complimentary to the desired modification site.

This project aims to develop an analytical method to measure transduction efficiency at the single-cell level in a gene-modified, viral-vector based immunotherapy model. The method will employ digital droplet PCR (ddPCR) implemented in polymer microfluidic devices to encapsulate engineered cells with appropriate reagents (lysis and PCR master mix), including vector specific primers and hydrolysis probes, as well primers and probes for the reference gene. The encapsulated droplets will then be subjected to thermal cycling and subsequently arrayed in a monolayer format for fluorescence imaging.

The goal of this project is to explore different non-viral technologies for precision targeting of gene therapies that could be deployed for different molecular targets and diseases.

More recently, NK cells, modified with chimeric antigen receptor (CAR-NK cells), are being explored as potential off-the-shelf therapy and these have shown better efficacy and lower toxicity than equivalent CAR-T cells in pre-clinical animal models.

The UKRI will hold a call for proposals aiming to solicit collaborations between UK researchers and the National Research Council of Canada’s Cell and Gene Therapy (CGT) Challenge Program, on research projects aiming to innovate therapeutic viral vector biomanufacturing processes.

The project will enable the graduate to learn the complexities of nanomedicine development, characterization and testing as well as manufacturing of personalised gene therapies to address multiple age-related diseases.

The proposed project will involve repeat dose testing in a Huntington's Disease model to demonstrate the ability to perform titratable genome editing. Additionally the project will involve proof of concept testing in other organ models of genetic disease.

Through the application of national and local priorities, the CSJ program seeks to provide youth, particularly those who face barriers to employment with access to work opportunities. Funded employers must demonstrate that they are providing quality work experiences for youth that provide opportunities to develop and improve their skills.