Vectors derived from the Adeno-Associated Virus (AAV) are highly efficient and broadly used tools for in vitro and in vivo gene delivery. In the past few years, AAV have emerged as one of the most effective vehicles for gene therapy due to their ability to transduce a wide variety of mammalian cell types, and their favorable safety profile. Indeed, AAV is considered non-pathogenic in humans.
The Bertarelli Platform has developed various technologies to improve the yield and the quality of AAV vector production, in order to consistently obtain vector batches at high titer and purity .
In addition, we can produce a broad range of AAV serotypes particularly adapted to applications in the central nervous system (brain and spinal cord) and sensory organs.
Various constructs are available for (1) gene overexpression in a cell type specific manner, (2) gene silencing by RNA interference and (3) gene targeting using CRISPR/Cas9.
We can produce the following serotypes:
Natural serotypes: 1 to 6, other serotypes are available upon request.