The Gene Therapy Laboratory was created in June 1996, and the vector core was set up in 1997.
The vector core facility started to provide the scientific community with viral vectors derived from adenovirus, lentivirus and adeno-associated virus. The idea was to make easier and to accelerate research work in the field of viral vectors-mediated gene transfer. The Core is funded by Association Française contre les Myopathies (AFM), Institut National de la Santé et de la Recherche Médicale (INSERM) and the University Hospital (CHU) of Nantes.

Today our laboratory is a joint research unit, INSERM UMR1089, organized in three groups:
  • A Research group, which develops in vivo gene transfer protocols for retina and neuromuscular diseases (e.g. spinal amyotrophy and Duchenne muscular distrophy). In each model disease, our focus is to evaluate the efficiency of gene transfer according to the administration rout, to study the possible associated immune response, and to examine the molecular fate of the vector following administration.

  • A Development group, which develops innovative tools for AAV vectors production, production and purification processes for manufacturing of clinical-grade vectors, and assays for quality control of pre-clinical and clinical vector lots. Our aim is to transfer these developments to our cGMP facility (Atlantic Bio GMP) or to our collaborators at Genethon.

The services provided by the vector core include:
1. Production of recombinant viral vectors carrying your gene of interest

Adeno-associated virus (AAV) derived vectors pseudotyped with capsid from AAV serotypes -1, -2, -4, -5, -6, -7, -8, -9 and -rh10. For each serotype, the vector can be produced as ssAAV (single-stranded) or scAAV (self-complementary, i.e. double-stranded).

First generation (E1/E3 deleted) adenovirus type 5 derived vectors.

Third generation (self inactivating) HIV derived lentiviral vectors.

The vector core takes care of custom vector production, from construction of the recombinant viral genome to vector purification and quality control. We also provide investigators with standard reporter gene vectors (carrying GFP or LacZ). 

2. Primary cells immortalization

Cell immortalization is achieved through serial infections of the cells with an MLV-based retroviral vector expressing SV40 T antigen and neomycin resistance. A cell bank is then generated from either the selected cell population or a single cell clone.

3. Construction of plasmids intended to recombinant viral vectors production.

4. Provision of biological materials: viral vectors samples, cell lines, plasmids, ...

4. Technical and scientific advice for optimal design and use of the viral vectors