Genetic Therapy
A platform to facilitate the preparation of AAV vectors for the genetic therapy for a range of neurological disorders
Available now
Location: Queen Square House, Room 924
Full service to open at Grays Inn Road in 2028
The Genetic Therapy Vector core facility uses cutting-edge technologies to create and produce a variety of high quality adeno-associated viral vectors (AAV) to provide researchers with the most suitable and efficient gene transfer vectors for their research applications.
The platform is lead by the Genetic Therapy Accelerator Centre
The facility will provide a catalogue of reporter gene AAV vectors, available to order; a Capsid packaging service, a custom gene therapy design and production and training.
AAV production for DNA, RNA and editing, developing AAV technology with capsid, promoter, and protein engineering to reach Orphan Drug Designation. Refeyn Samux MP for Empty: Full AAV capsid characterisation and dedicated HPLC and ultracentrifuge for purification
Services offered
- Scientific expertise AAV expression cassette design
- Scientific expertise in experimental design in capsid selection, prep size, vector dosing for application
- Comprehensive AAV vector production, titration and characterisation service
- AAV purification service (if AAV tissue culture production is performed elsewhere)
- Vector Characterisation service
Costs
Please contact us for a quote
The platform will provide AAV vectors to Institute of Neurology and Institute of Child Health (GOSH) in the first instance on a cost recovery basis. Until a full service is operational at Grays Inn Road, availability to other groups working in UCL at other institutes and faculties may be limited.
Our Impact
Recent breakthroughs in medicine now allow scientists to treat previously untreatable diseases by fixing faulty genes. UCL is a global leader in developing and testing these genetic therapies, especially for neurological conditions like Parkinson’s, dementia, epilepsy, and motor neuron disease. The genetic therapy platform allows researchers to use advanced tools to test if their therapies work in cells—an essential step toward creating future treatments and cures through clinical trials.
A dedicated team
Dedicated team of experts to support custom-design and produce high quality AAV vectors for research needs.
Consistent and reliable
Dedicated team of experts to support custom-design and produce high quality AAV vectors for research needs.
Best practice methods
High level of AAV vector characterisation and quality control available
How to register to use Infinity, the research technology platform booking system
1. Visit Infinity website (link below)
2. Click on SSO icon to log on using your UCL ID
2. Complete registration wizard, including your lab and the facilities that you want to access.
3. Your application will now be sent to the facility manager to be approved. This can take up to 3 days.
4. Once your registration has been approved, you will be assigned a funding code by your departmental administrator. This will appear in the system.
5. Equipment and services are now bookable in the system.
