About us

The Area of Major Interest (DIM) is the result of a structuring policy of the Paris Region, which aims to foster innovation by leveraging the excellence of Paris Region teams.

The DIM « thérapie génique » initiative was labelled in December 15^th, 2016, among 8 other DIM.

Concretely, the DIM is a research network labelled for a time period of 4 years (2017-2020) by the Paris Region. This labelling allows research teams in the Paris Region to benefit from funding, based on an annual program defined by its members.

The DIM supports research projects by financing doctoral or postdoctoral fellowships and scientific equipment. The DIM also organizes scientific symposia and promotes the development of R&D partnerships with industry.

The Paris Region is a pioneer in the field of gene therapy. In 1999, the first gene therapy protocol to treat children with severe immunodeficiency (X-SCID) was developed by Pr. Alain Fischer, Marina Cavazzana and Salima Hacein-Bey and launched at the Necker – Enfants malades University Hospital.

The Paris Region hosts several key actors involved in gene therapy. The DIM « thérapie génique » aims to federate them and accelerate the development of gene therapy to reinforce, in the light of personalized medicine, it grades among the most promising and innovative therapeutic strategies. This initiative aims to provide treatment for serious or chronic pathologies without treatment till today, or for which the medical care is still largely not satisfying.

The objectives of the DIM are to:

• Federate Paris Region actors to accelerate the development of gene therapy from research to care, particularly in therapeutic areas hitherto unsupported or poorly supported
• Capitalize on expertise to promote the transition of this innovative approach, from rare diseases to more frequent diseases
• Federate Paris Region people around the challenges in the field of gene therapy by organizing events for the general public
• Increase the scientific and economic attractiveness of the Paris Region

For the Paris Region, challenges are foremost medical: for example, it involves finding treatments for the 240 children born each year in Paris Region, suffering from one of the two most severe forms of sickle cell disease, and for which no curative treatments exist. Gene therapy also promises to cure AIDS patients, while Paris Region is the most metropolitan area affected by this disease.
Challenges are also economic: regarding the growing interest of industrials and private investors for this innovative therapeutic approach, the Paris Region wants to encourage the development of industrial partnerships and the creation of start-ups, generators of progress, value and employment.
Finally, the aim is to reinforce the medical and scientific excellence of the Region, a pioneer in gene therapy, and to increase its scientific influence.

The scientific coordination of the network is provided by Prof. Marina Cavazzana.

In 1999, she set up at the Necker Hospital, with Prof. Alain Fischer and Salima Hacein Bey Abina, a first gene therapy protocol for children with severe immunodeficiency, a rare genetic disease. This treatment consisted of harvesting hemopoietic stem cells (the stem cells responsible for all blood cell lines, including cells of the immune system) in patients, and then, inserting into these cells a “drug” gene with the help of a retroviral vector, modifying the defective gene. The modified cells, after checking their viability, were then reinjected into the children, without risk of rejection, unlike a bone marrow transplant treatment.

Marina Cavazzana
DIM Thérapie génique coordinator

Corinne Antignac

Kidney

Pablo Bartolucci

Sickle cell disease

Nathalie Boddaert
Clinical

Imagery

Christine Bodemer

Skin

Deniz Dalkara

Vision

Pascale de Lonlay
metabolic diseases

Metabolic and mitochondrial diseases

Olivier Goureau

Vision

Alain Hovnanian

Skin

Jean-Daniel Lelièvre

HIV

Fulvio Mavilio

Viral vectors

Judith Melki

Neurodegenerative diseases

Federico Mingozzi

Viral vectors

Vincent Mouly

Neurodegenerative diseases

Christine Petit

Audition

Agnès Rötig
mitochondrial diseases

Metabolic and mitochondrial diseases

Matthias Titeux
Preclinical

Imagery

Julien Zuber

Tolerance and Immunity

The DIM Thérapie Génique managing organization is the Imagine Institute of Genetic Diseases. As such, the Imagine Institute receives the regional grant and reverse it to the academic teams from the network according to the annual work package defined by the members of the network.

The Imagine Institute is a unique place combined research and cutting-edge care with the objectives to better understand genetic diseases and to provide the diagnostic and therapeutic solutions expected by patients and their families.