WP1 aims at developing an expanded toolbox of improved technologies instrumental to accomplish the ambitious therapeutic goals of WP7-WP9. CRISPR/Cas9 and orthologous DNA targeting systems will be further perfected to develop genome and base editors, tailored nucleases, and novel epigenetic editing systems. Also, a new set of user designed recombinases will be developed and combined with programmable vectors developed in WP2 to address large edits.
Specific WP’s objectives:
- Develop shorter and more precise CRISPR/Cas systems
- Develop strategies to enhance the efficiency of genome editing
- Improve epigenome editing tools towards in vivo applications
- Develop platform for advanced generation of programmable site-specific recombinases
Tasks:
- Task 1.1: Turning inactive CRISPR/Cas systems into effective and precise tools to edit the mammalian genome
- Task 1.2: Development of novel high-fidelity Cas9 proteins or orthologs variants
- Task 1.3: Identification of cellular factors that increase efficiency of targeted genome editing
- Task 1.4: Optimization of targeted epigenetic editing towards in vivo application in the liver
- Task 1.5: Optimization of targeted epigenetic editing towards in vivo application in striated muscle
- Task 1.6: Speeding up designer-recombinase evolution by the generation of “ISOR”-libraries
- Task 1.7: Nanopore sequencing of recombinase libraries
- Task 1.8: Optimization and characterization of “ISOR”-recombinases
Timeline:
WP leader: Anna Cereseto (UNITN)
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