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"A pioneer DNA": in 30 years, the Telethon lab has revolutionized research

2020-12-03T09:29:59.493Z

The Généthon was created in 1990 in Evry (Essonne) by the AFM Téléthon. Thirty years later, the first treatments to fight against ma



In the corridors, white blouse on his back, Frédéric Revah announces the good news to everyone they meet.

"We have just had the authorization for clinical trials on Duchenne muscular dystrophy," welcomes the director of Généthon, the laboratory created by AFM Téléthon in 1990, which today has 220 employees.

In a few weeks, in this building in Evry-Courcouronnes (Essonne), close to the national 7 and the Sud Francilien hospital, a brand new treatment will be tested against this genetic disease which causes a progressive degeneration of the all the muscles of the body.

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Nearly twenty other drugs are also in the Genethon test or pretest phase to treat diseases of the blood, liver and muscles.

And last year, for the very first time, one of the remedies developed within these walls received marketing authorization.

Yet 30 years ago, few believed in this brand new lab.

"At the time, no one could have said that all these advances would come from Evry, between the A6 and McDonald's", smiles Frédéric Revah.

“The Genethon is the DNA of a pioneer,” sums up the man who took the helm of this structure in 2010. And today, it is thanks to the Telethon that we are able to develop all these drugs.

"

"Do better and faster"

Because the AFM Téléthon is the keystone of this non-profit laboratory, born "of a unique link between researchers and patients".

“In 1958, it was also with Duchenne muscular dystrophy that it all began,” recalls this scientist.

Parents of children with this rare disease come together to create this structure.

“In the beginning, it was above all an association that came to the aid of families to finance armchairs in particular,” says Serge Braun, the scientific director of AFM Téléthon since 2005.

Frédéric Revah has been Director of the Généthon since 2010 LP / Pauline Darvey  

The idea of ​​creating a laboratory dedicated to research on rare genetic diseases was born at the end of the 1980s. “The association was already funding labs but its members believed that we could do better and faster,” continues Serge Braun.

For that, it took a lot of money.

"

In 1987, the very first Telethon accelerated the movement.

“It was an immediate success,” recalls the technical director.

180 million francs were collected, while on the TV set, the counter was only planned for eight digits!

"

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A year earlier, the gene responsible for Duchenne muscular dystrophy had been discovered.

“But it took ten years of research to isolate this gene,” says the researcher.

Few labs can afford such an investment.

Hence the idea for the association to create its own.

"

"A real revolution!"

The headquarters of the association was then in Paris.

But space is needed to set up this new lab.

"And Bernard Barataud, the former president of the AFM Telethon, lived in Essonne, which must have made things easier", specifies Serge Braun.

It is decided, the Genethon will open its doors in Evry.

"The transformation of the premises and the move took place in barely four months", continues the scientific director.

The objective of the “armed wing” of the Telethon is initially to detect the genes responsible for rare diseases, which are 90% genetic.

"For that, it was first necessary to establish maps of the human genome", indicates this specialist.

To implement this titanic project, the Généthon researchers decided to rely on robots, capable of cutting DNA at different places.

A world first.

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Barely two years later, the first maps of the human genome are drawn.

“It was a real revolution, insists Serge Braun.

All the labs specializing in genetics used these cards.

At the time, we had isolated a handful of genes responsible for genetic diseases.

Today, just under 4,000 have been discovered.

"

From 1996, the Généthon decided to move towards gene therapy.

A method which consists of replacing a faulty gene with a healthy gene.

New researchers join the lab.

But here again, there is strong reluctance in the scientific community.

“Many thought that it would not work,” confirms Serge Braun.

"In 30 years, it will be routine"

These new therapies, however, showed their first evidence in the early 2000s on diseases of the immune system.

“Bubble babies” - who must be placed in a sterile environment due to weak immune systems - will be among the first to benefit from these advances.

"We take the spinal cord, we inject the missing gene and we reinject everything into the body of babies," explains the scientific director.

The cells can then start to make functioning white blood cells.

"

These techniques are now applied to many uses.

"This is also the principle of the gene therapy vaccines which have just been developed against Covid-19", illustrates Serge Braun.

"In 30 years, I am almost certain that gene therapy will be routine," warns Isabelle Richard, research director at the CNRS, who is also leading a team within the Généthon which is preparing to launch trials. clinics to treat a certain type of myopathy.

It will be a type of medicine like any other.

In medicine, this is going to have a huge impact.

"

In a few months, it may be the turn of Duchenne muscular dystrophy to have its own treatment.

Quite a symbol for the Genethon and for its current director.

"We must rethink all those families who began to fight in 1958 for future generations ..."

Source: leparis

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