Based at the School of Pharmacy, the UCL GeneTxNeuro Vector Core Facility can help with the production of research-grade viral vector batches.
The UCL GeneTxNeuro Vector Core Facility is funded by the NIHR GOSH BRC.
The scope of the Facility is to provide access to a state-of-the-art vector production laboratory for research-grade viral vector batches. The UCL team has extensive expertise developing gene therapy products and will provide expertise and support for new and established researchers wishing to produce and use these tools.
Users will be guided and advised through all the steps of process, from construct design to in vivo applications. This platform provides the opportunity to learn how to produce customised viral vectors to researchers who do not have experience with gene therapy or do not have access to specialised equipment.
Reasons for use
Viral vectors can be engineered to deliver a gene to cells or a model organism for:
- Gene replacement/augmentation therapy – delivery of the functional version of the gene to compensate for an inherited disease-causing copy in monogenic diseases;
- Reporter gene expression in specific cell types;
- Gene silencing – delivery of siRNA or shRNA to reduce gene expression;
- Genome editing – delivery of CRISPR-Cas construct to delete or insert specific nucleotides within the GOI.