Published on 04.02.2022
A conference open to all on the theme of gene therapy
Replacing a "sick" gene with a "healthy" one, eliminating a genetic abnormality using molecular scissors, gene therapy is exciting and raises questions, both about the hopes it can give, but also about its limits. On this International Rare Disease Day, the Imagine Institute, with the Necker-Enfants malades AP-HP Hospital and its rare disease expertise platform, takes stock of the situation with its teams of researchers and physicians.
On the agenda:
- Myths and realities of gene therapy, with Prof. Alain Fischer, first director of the Institute
- Concrete examples of gene therapy available today, with doctors from the Necker-Enfants malades Hospital and rare disease reference centers
- Prospects and current research, with researchers from the Imagine Institut
Places at the Institut Imagine are limited. To register, go here. You can also follow us live on Facebook
Gene therapies, therapies of the future
A pioneer in research and treatments based on gene therapies or derived from them, the Imagine Institute, within the campus of the Necker-Enfants malades AP-HP hospital, is home to teams at the forefront of this therapeutic approach. Some of its researchers, such as Prof. Alain Fischer and Prof. Marina Cavazzana, were the first in the world to conduct clinical trials and demonstrate the success of this therapy for certain diseases.
It took 20 years of work to achieve clinical results that transformed patients' lives. Since then, gene therapy has continued to improve and is now being deployed for other diseases. It is being explored to treat diseases of the blood, immune system, metabolism, brain, central nervous system, eye and skin.
Today, teams such as Annarita Miccio's are working on approaches that foreshadow the medicine of tomorrow. Thus, thanks to new research and advanced methodologies, gene therapy could in the future be applied to a much larger number of organs and diseases.
International Rare Disease Days at Imagine
Every year, for International Rare Disease Day, Institut Imagine opens its doors, physically or virtually, to inform and raise awareness about genetic diseases and rare diseases. Last year, we proposed a weekend of web-conferences dedicated to certain rare diseases. Find all these conferences here.
To learn more about Rare Disease Day, go here.