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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).


  • Mrs Anne GALY

Innovation themes

Map and access

1 bis, rue de l'Internationale
91002 ÉVRY

Our research areas


-gene therapy, gene transfer, genome edition
-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

Total number of employees : 44
Number of researchers : 30
Number of doctoral students : 14

Equipment(s) open to collaboration

-imaging and cytometry platform


Our results

Valuation offer

- bioinformatics
- imaging and cytometry platform

Projects examples

-hematopoietic gene therapy
-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

Spark Therapeutics
Audentes Therapeutics


Establishments of affiliation