WP9 – Expanding the breadth of liver-directed gene therapy to include larger patient populations

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:

  1. To expand application of gene therapy to pediatric patients by developing strategies based on stable transgene integration
  2. To develop effective and safe gene therapy strategies for the treatment of hypercholesterolemia.

Tasks:

  • 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

Timeline

WP leader: Angelo Lombardo (FTELE)

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