The main mission of the Bertarelli Gene Therapy Platform is to provide support for the development of gene therapies targeting diseases which affect the central nervous system and sensory organs.
The use of vectors of viral origin has emerged in the past few years as a powerful technology to deliver nucleic acids to key cell types in the central nervous system, in order to manipulate their function, or change their fate in pathological conditions. The platform is involved in the design and generation of such molecular tools to genetically modify cells and tissues, both in vitro and in vivo. In particular, adeno-associated viral (AAV) vectors and lentiviral vectors are now broadly used in research, as well as for therapeutic applications.
The platform has expertise in the design and production of vectors for various applications such as:
- Gene overexpression, gene silencing by RNA interference
- CRISPR/Cas9 targeting for gene disruption or gene activation
- Cell-specific expression of various reporters
- Genetic modelling of neurodegenerative diseases including Parkinson’s and Alzheimer’s diseases (alpha-synuclein, tau, amyloid β)
We produce AAV vectors of various serotypes (natural AAV 1 to 6, others on demand). Vectors are purified using density gradient and/or chromatography, and characterized using various methods. The platform also produces VSVG-pseudotyped lentiviral vectors for both in vitro and in vivo applications.
In the context of gene therapy, the Bertarelli platform is particularly active in the field of motoneuron diseases, including genetic forms of Amyotrophic Lateral Sclerosis (ALS). Other research programs aim at the development of vectors for the treatment of blindness as well as genetic forms of deafness.
More detailed information is available on the website of the Bertarelli Foundation Gene Therapy Platform.