Are you looking to move your business into developing Gene Therapies?Â Would knowing the current situation of lentivectors aid you in deciding how to best achieve this goal?
At the recent 2012 World Gene Therapy Congress, part of the 7th annual World Stem Cells and Regenerative Medicine, Dr Michael Antoniou, from the Department of Medical and Molecular Genetics at King’s College London reviewed this very area.
- Exploring the ability of lentiviral vectors (LVs) to efficiently integrate transgenes in stem cells and the potential to provide long-term therapeutic benefit
- Overcoming insertion site position effects and becoming immune to epigenetic-mediated silencing to provide reproducible and stable expression at low vector copies per cell
- The promise to meet these requirements of LVs containing transgenes under control of genetic regulatory elements with a dominant chromatin remodelling capability, locus control regions (LCRs) and especially ubiquitous chromatin opening elements
- Reviewing an encouraging pre-clinical evaluation of LVs containing Î²-globin LCR regulated transgenes suggesting UCOE-based vectors can be used to provide unprecedented stable expression in a very wide range of tissues and disease conditions
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