Finding cures for children's genetic diseases


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  • AAV

Translational Vectorology Group

The Translational Vectorology Group (TVG), Headed by Dr Leszek Lisowski was established at the end of 2015 and is the newest group to be formed at CMRI. We like to think of ourselves as “translational tool makers” as we develop and improve vector-based tools that can be used in wide range of basic and preclinical studies, as well as in clinical applications.

Our research interests are related to two main areas: 1) development of new gene therapy vectors based on Adeno-Associated Virus (AAV) as tools for tissue-specific gene delivery; and 2) development of new, safer genome-editing tools not dependant on endonuclease activity. We are developing these tools to target a broad array of tissue types and thus address a range of diseases. Although our main interests are in paediatric genetic and acquired liver disorders, we are also actively developing novel therapeutic tools for such applications as gene therapy in the CNS, eye and haematopoietic system. 

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