Gene therapy vectors

MyoVector platform

The MyoVector platform is headed by Sofia Benkhelifa-Ziyyat, PhD, a researcher at the Institute’s Center of Research in Myology. Its aim is to produce gene therapy vectors derived from adeno-associated viruses (AAV« adeno-associated virus » , ou virus adéno-associé est un petit virus à ADN simple brin. Il fait partie de la famille des Parvoviridae et appartient au genre des Dependovirus. La particule virale est constituée d’un brin d’ADN de polarité positive ou négative protégé par une capside. La taille moyenne d’une particule d’AAV et de 18 à 22 nm. Les AAV sont les seuls parvovirus non autonomes. Lorsqu’on emploie « rAAV » , il s’agit du virus AAV recombinant, c’est-à-dire qu’il a été modifié pour devenir un vecteur (et n’est donc plus virulent).) in order to support therapeutic and exploratory research projects. 

The platform produces high-quality vectors in about one week, using a process based on the transient triple transfection of cells in suspension, followed by purificationEnchaînement d’une ou plusieurs techniques permettant d’accroître la pureté d’une molécule ou d’un composé chimique. and final formulation steps. It offers several serotypes (AAV1, AAV2, AAV5, AAV6, AAV7, AAV8, AAV9, AAVrh10, AAVMYO), allowing the expression of therapeutic transgenes, reporter genes or other genetic constructs according to the needs of each research project.

By combining the expertise of MyoVector with that of the Généthon laboratory, a scalable production method for preclinical and clinical applications is currently being developed within MyoVector with the support of the DIM Thérapie GéniqueEnsemble d’approches thérapeutiques où une information génétique est transférée aux cellules, tissus ou organes d’un patient, dans le but de pallier un défaut génétique ou de fournir une fonction protectrice ou réparatrice..

In 2025, the MyoVector platform continued and scaled up its activity, in particular:

• Production of vectors with a strong muscle tropism (AAVmyo and LICA1) for the strategic project “‘Decoy’ gene therapy for DM1” (myotonic dystrophy type 1). Evaluation of the various optimised constructs produced in this context by D. Furling’s team in collaboration with Généthon.
• 75 production runs carried out for six teams of the Center of Research in Myology. The serotypes produced are AAV1, AAV2, AAV8, AAV9, AAVMYO and LICA1
• Through a collaboration between MyoVector and MyoImage, production of a catalogue of AAV vectors expressing fluorescent reporter genes that specifically label various cellular compartments (actin, microtubules, Golgi, mitochondria, nuclei, centrosomes, histones, etc.), made available to the research teams. The aim is to build a resource that is easily and rapidly accessible for research projects and to encourage new collaborations.
• Presentation of MyoVector activity at ESGCT 2025 in Seville and at the “Innovation Day 2025” DIM BioConvS symposium (Région Île-de-France).

Team members

Sofia Benkhelifa-Ziyyat, research director (DR)
Pierre Meunier, study engineer (IE)