Neuroplasticity & Therapeutic
The team’s research focuses on the use of pluripotent stem cells (embryonic or induced) to model human brain development and brain diseases such as Lesch-Nyhan disease, Phelan-McDermid syndrome and dystrophinopathies.
Our team is developing protocols to differentiate stem cells into different types of brain cells, including different types of neurons. We focus on reconstructing in vitro the key stages of human development and brain plasticity. These cellular models, in 2 or 3 dimensions, are then used to study the influence of genetic mutations that cause so-called neurodevelopmental diseases, which are characterized by severe neurological and psychiatric disorders in very young children. We are working in particular on genetic forms of autism spectrum disorders (SHANK3 mutations), on Lesch-Nyhan disease (HGPRT) and on neurological disorders found in certain forms of myopathies (Duchenne and Becker muscular dystrophy, DMD gene mutations)
We use high-throughput, high-content screening techniques to identify pharmaceutical compounds that can correct the abnormalities caused by these different mutations, and then work in partnership with clinicians and industry to test these approaches in children affected by these diseases.
As part of our research on neurological damage associated with dystrophinopathies, we welcome Lise Morizur as a visiting associate researcher. Lise develops 3D models of muscle fibers. She benefits from our environment and our experience in 3D tissue culture and modeling for pharmacological research. In return, the 3D muscle models will allow us to delineate the specificities of the mechanisms of action of dystrophin isoforms in the brain.
Team members
Alexandra Benchoua
Team Manager (CECS)
Passionate about neuroscience and developmental biology, Alexandra’s work focuses on personalized medicine for patients suffering from rare neurological and psychiatric diseases.
Laure Chatrousse
Research associate (CECS)
Laure is working on the modeling of neuropsychiatric disorders associated with Phelan-McDermid syndrome and Duchenne Muscular Dystrophy using pluripotent stem cell derived neurons.
Claire Boissart
Engineer (CECS)
Claire works on Lesch-Nyhan disease via three different approaches: 2D and 3D in vitro modeling, a pharmacology approach and gene therapy.
Thifaine Poullion
Engineer (CECS)
Thifaine works on the modeling of neuropsychiatric disorders using 3-D systems such as organoids derived from pluripotent stem cells.
Collaborations
ANR SynDivAutism : study of genes coding for synaptic proteins in autism spectrum disorders.
Clinical trial LISPHEM, ClinicalTrials.gov Identifier: NCT04623398
Genetics of autism and intellectual disability disorders. Biological and hormonal factors influencing the expression of ASD.
Genetics of neurodevelopmental disorders.
Emergence of equine zoonotic diseases. Study and treatment of equine encephalopathies transmissible to humans.
Gene therapy of dopaminergic deficiency. AFM/IGNITION project: Lenti-LND.
ANR IMPROVE : Modeling and treatment of neonatal epileptic encephalopathy.
Modeling and treatment of in utero CMV infections.
Brain organoids for modeling neurodevelopmental disorders and glioblastoma.
ANR TreatBD : Pharmacological approach to lithium-resistant bipolar disorder.
Publications
How do gravity alterations affect animal and human systems at a cellular/tissue level?
21 October 2023
NPJ microgravity
Establishment of heterozygous and homozygous SHANK3 knockout clonal pluripotent stem cells from the parental hESC line SA001 using CRISPR/Cas9.
01 October 2023
Stem cell research
Identification of signaling pathways modifying human dopaminergic neuron development using a pluripotent stem cell-based high-throughput screening automated system: purinergic pathways as a proof-of-principle.
01 January 2023
Frontiers in pharmacology