International researchers have developed a lentivirus-based gene therapy which they have tested in a mouse model of Pompe disease:
- the lentiviral vector is used to incorporate the drug gene into the host genome, in this case the mouse model of Pompe disease,
- The target tissue was haematopoietic stem cells,
- the efficacy in producing the enzyme missing in Pompe disease was remarkable, both in muscle and in the central nervous system,
- and the oncological risk associated with integration of the transgene appears to be minimal.
This new approach could provide a highly attractive therapeutic alternative to conventional enzyme replacement therapy and adeno-associated virus-mediated gene therapy.
