WP9 will integrate the gene editing and transfer technologies developed by the consortium
to tackle the following liver-associated diseases in mouse models: (i) Mucopolysaccharidosis VI and haemophilia by AAV-mediated delivery of the HITI technology; (ii) hemophilia and hypercholesterolemia by gene replacement with immune stealth and liver-targeted lentiviral vectors; (iii) hypercholesterolemia by viral and non-viral mediated delivery of targeted epigenetic editing components.
Specific WP’s objectives:
- To expand application of gene therapy to pediatric patients by developing strategies based on stable transgene integration
- To develop effective and safe gene therapy strategies for the treatment of hypercholesterolemia.
- Task 9.1: To develop a Homology-Independent Targeted Integration (HITI) approach for the treatment of Mucopolysaccharidosis VI
- Task 9.2: To develop a Homology-Independent Targeted Integration (HITI) approach for the treatment of haemophilia A and B
- Task 9.3: To evaluate LV-mediated gene transfer into liver stem and progenitor cells
- Task 9.4: To treat hypercholesterolemia by forced expression of the LDL-R by LV-mediated liver gene transfer
- Task 9.5: To treat hypercholesterolemia by targeted epigenetic silencing of PCSK9 in the liver
WP leader: Angelo Lombardo (FTELE)
→ Go back to WP list