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Quality of life - Health - Food
Integrated genetic approaches in therapeutic discovery for rare diseases (INTEGRARE)
Research unit
INTEGRARE (Integrated Genetic Approaches and New Therapies for Rare Diseases, UMR_S951) is a research unit specializing in translational research and gene therapy of rare genetic diseases. The unit is mainly interested in myopathies, metabolic diseases, blood diseases or immune deficiency. Using recombinant viral vectors (AAV and lentiviral vectors) or CRISPR technologies, researchers design novel therapeutic approaches, defining the pharmacological parameters and the safety of the approaches, and study how to overcome the roadblocks notably to avoid immune responses to gene therapy.
Promising therapeutic candidates are developed towards the clinic through Genethon's therapeutic programs. Several clinical trials of gene therapy are currently underway.
INTEGRARE is a partnership between Généthon, (a private R et D organization created by AFM / Téléthon), Inserm and the Universities (University of Evry / Paris Saclay University, EPHE).
Innovation themes
1 bis, rue de l'Internationale
Généthon
91002 ÉVRY
Expertises
-genetics, genomic, bioinformatics
-study of myopathies and muscular dystrophy
-gene transfer into liver and metabolism
-immunology, immune responses, immune deficits
-biotherapies, preclinical development
Applications sectors
- Biotechnology
- Health / wellness
Total number of employees
Number of researchers : 30
Number of doctoral students : 14
Equipment(s) open to collaboration
-imaging and cytometry platform
Keywords
Valuation offer
- imaging and cytometry platform
Projects examples
-liver gene transfer and immune tolerance induction.
-gene therapy of progressive muscular dystrophies
-new approaches for the gene therapy of neuromuscular disorders
Industrial and scientific relations
Private collaborations
Genosafe
Yposkesi
Spark Therapeutics
Audentes Therapeutics
Establishments of affiliation
