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Gene therapy for mucopolysaccharidosis - adeno-associated virus and lentivirus as vectors for muscle, liver, central nervous system and bone marrow


Research objectives and content
The goal of this project is to permanently correct beta-glucuronidase deficiency in the mucopolysaccharidosis type VII (MPS) mouse model. For this purpose, we will perform gene transfer experiments in several tissues affected by this lysosomal storage disease such as liver, skeletal muscle, central nervous system and bone marrow cells using adeno-associated virus and lentivirus as gene transfer vectors. These vectors have been proven to be efficient for gene transfer in muscle (adeno-associated virus), central nervous system and bone marrow cells (lentivirus). An alternative approach that will be tested is a cell-encapsuladon method where the cells, permanently transfected with AAVs or retroviruses, will be protected against the host immune system by a permselective membrane and then, implanted into the target tissue. The efficiency of these gene transfer systems in the MPS mouse model will be analyzed by specific enzyme staining as well as phenotypic correction of storage lesions at short and long term. Training content (objective, benefit and expected impact) The training objective of this project is to test alternative gene transfer strategies to improve gene therapy for mucopolysaccharidosis. The applicant will be able to learn new gene transfer techniques and the impact of this research will be very significant for treating MPS patients in the future.
Links with industry / industrial relevance (22)

Call for proposal

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Institut Pasteur
EU contribution
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28,Rue du Docteur Roux
75724 Paris

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