Glossaire

Terme général incluant le conditionnement primaire, l’emballage extérieur, l’étiquetage et la notice d’un médicament.

Embryonic stem cells exist during the earliest stages of development. They have the potential to become any cell type in the body, meaning that the cells are pluripotent. These cells are found in developing embryos for a short period after fertilization, before the cells start to develop (differentiate) into different cell types. The diversity of embryonic stem cells makes them incredibly valuable for research and regenerative medicine.

Epigenetic reprogramming refers to any alterations of epigenetic markers (that is, changes which are not part of the DNA, but will affect gene expression. This reprogramming is important during gametogenesis (the formation of sex cells) and fertilisation. It ensure that the 'correct' DNA is passed from the parent to the offspring. 

Epigenetic reprogramming can also be important in cell culture, to reverse changes to the adult cell in order to create induced pluripotent stem cells (iPSCs).

Epigenetics refers to mechanisms that affect the functionality of the genes (how genes work) without any change to the DNA sequence itself. Epigenetic regulation of gene expression enables differences in cells, and their functions, with all the cells still containing the same genetic information. For example, identical twins that carry the exact same genes may develop unique differences during their lifetime, due to environment- or lifestyle-dependent epigenetic changes.

Epigenome editing is a technique used to modify the epigenetic traits of an organism's genome. It involves changing specific epigenetic features to alter the expression of a specific gene without altering the DNA sequence (such as DNA methylation or histone acetylation). Scientists use epigenome editing to modify the instructions on how the DNA is read, either to increase or decrease the expression of a desired gene. This enables us to understand how genes or epigenetic patterns are read in the body and what impacts they have on cell function.

Ex vivo refers to something happening outside a living organism. In ex vivo experiments, researchers aim to collect information on, or modify, the functionality of cells or tissues in a controlled laboratory environment outside a living organism.

Ex vivo gene therapy involves the collection of cells from an individual patient, followed by modifications (insertion, deletion or correction) of the disease-causing gene (called the gene of interest) in a laboratory, and finally, reintroduction of the modified cells back into the patient to treat a specific disorder. This approach allows for controlled treatment and selection of cells that are reintroduced back into the patient.

Régime juridique spécifique pour un petit nombre de Médicaments de Thérapie Innovante (MTI) prescrits par un médecin à un patient dans un hôpital d’un pays de l’Union Européenne.

See Gene expression.

Toute étude menée avant une étude clinique pour fournir la preuve de concept d’un traitement. Il peut s’agir d’études sur un médicament, une procédure ou un traitement dans des modèles cellulaires ou animaux. Une étude préclinique est un sous-type d’étude non clinique. ‘Étude non clinique’ est une expression large couvrant toute étude qui n’est pas clinique. L’expression ‘Étude préclinique’ couvre toute étude non clinique destinée à évaluer la sécurité avant le début d’un essai clinique.

Procédure réduisant le délai d’évaluation d’une demande d’autorisation de mise sur le marché (de 210 à 150 jours) pour les médicaments répondant à un besoin médical non satisfait.

Méthode pluridisciplinaire d’évaluation des technologies de santé. Elle synthétise les informations sur les aspects d’ordre médical, social et relatifs aux patients, sur les questions économiques et éthiques de l’utilisation d’une technologie de santé et ses conséquences.