Gene therapy is the use of genetic material to treat, cure, or prevent a disease or medical condition caused by a deficient or dysfunctional protein. It involves the introduction of genetic material into a target cell. The principle of gene therapy is based on the transfer of a therapeutic gene using viral (adenoviruses, adeno-associated viruses, lentiviruses, herpes viruses) or non-viral vectors as a result of which, the recepient cells will start to produce proteins that will correct a genetic disorder or acquired disease.

Equipments

THE VECTOROLOGY research facility comprises two rooms where users can produce or handle biological material categorised as Biosecurity Level 2 or 3 (BSL):

There are two main parts to the vectorology platform:

1. The L3 cell culture room meets all the legal requirements for the production and handling of class 2 or 3 GMO’s microorganisms. It is available for any team who wishes to make their own viral vectors for a given project.

2. The A2 room is located in the animal facility. It is where the viral vectors are tested in vivo: The viral vectors are safely administered to rodents in the A2 room. Many techniques are available to users:

• Systemic injection ;
• Intra-ocular injection

We use these rooms for producing vectors for gene therapy.

Gene therapy is the use of genetic material to treat, cure, or prevent a disease or medical condition caused by a deficient or dysfunctional protein. It involves the introduction of genetic material into a target cell.

The principle of gene therapy is based on the transfer of a therapeutic gene using viral (adenoviruses, adeno-associated viruses, lentiviruses, herpes viruses) or non-viral vectors as a result of which, the recipient cells will start to produce proteins that will correct a genetic disorder or acquired disease.

The viral vectors are used in the institute as a tools for transferring different genes into cells:
• Replacing a mutated gene by a healthy copy
• Gene knockdown, via tools like shRNA
• Expression of neuroprotective genes
• Expression of genetically encoded light sensitive modulators of membrane potential (optogenetics) or calcium sensors (Gamp)
• or molecules derived from a screen

Many different projects use these tools to study a broad range of subject, including neural development, tracing of neurons in the brain, replacement gene therapy, expression of optogenetic tools in targeted retinal cells, etc.

TEAM

Deniz Dalkara
Scientific supervisor

Email: deniz.dalkara@gmail.com

Melissa Desrosiers
Technical manager
On the vectorology research core: Mélissa Desrosiers supervises and participates in the viral vector production, user training, monitoring the budget and billing, planning organisation. She is also the lab manager in Deniz Dalkara’s team, is taking part in the whole process of the production of viral vectors, from the design to expression’s analysis by histology and imaging.

Skills: Molecular biology, Cell culture, Viral vector, Confocal imaging

Scientific interest: Gene therapy, gene transfert, optogenetic

Email: melissa.desrosiers@inserm.fr