Work Packages

1 Novel strategies to increase precision and efficacy of gene and epigenetic editing
2 Retargeting gene/vector integration
3 Genome-wide off-target profiling of gene-editing reagents
4 Viral and non-viral mediated delivery of genes and genome engineering components
5 Increase immune evasion of genome engineering components
6 Advanced genetic engineering of hematopoiesis
7 Novel strategies to target skeletal and cardiac muscle wasting in aging and disease conditions
8 Retinal degeneration
9 Expanding the breadth of liver-directed gene therapy to include larger patient populations
10 Management and Scientific coordination
11 Communication, dissemination, stakeholders engagement and exploitation
12 Ethics requirements