WP6 will optimise gene editing application in HSC using the improved technologies developed in WP 1-3 and develop novel strategies to select edited HSC, expand them ex vivo and induce preferential in vivo engraftment. The outcomes of these studies will significantly broaden application of targeted genome editing in HSC to potentially include most inherited diseases that affects this cell compartment, as well as acquired diseases.
Specific WP’s objectives:
- Improve the efficiency of targeted gene editing in HSC and the yield of edited cells.
- Identify novel targets and devising new non-toxic strategies for enhancing self-renewal and in vivo engraftment of edited HSC (enhanced gene edited, EGE, cells).
- Develop strategies to select edited HSC that are clinically compliant and couple them to emerging ex vivo HSC expansion protocols.
- Match enhancement strategy to the choice of biological conditioning regimen so that EGE HSC can effectively outcompete resident HSC by the transiently expressed gain-of-function
- Task 6.1. Improve the efficiency of targeted gene editing in HSC
- Task 6.2. Enhancing self-renewal and engraftment of edited HSC
- Task 6.3. Selection of the edited cells by means of ETA
- Task 6.4. Match enhancement to biological conditioning
WP leader: Luigi Naldini (FTELE)
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