Sylvie Ramboz

Exacerbation of a Subset of Behavioral Phenotypes by Early Treatment with AAV FOXG1 Gene Replacement Therapy in a Mouse Model of FOXG1 Syndrome

This study shows that early AAV FOXG1 gene therapy in female mice worsens motor behaviors, while it benefits males, highlighting gender-specific risks in potential human treatments.

Behavioral Efficacy of AAV FOXG1 Gene Replacement Therapy in a Mouse Model of FOXG1 Syndrome

This study shows that AAV FOXG1 gene therapy improves behavioral deficits in a mouse model of FOXG1 syndrome with a Q84P mutation.

A Systematic Assessment of Robustness in CNS Safety Pharmacology

This study assesses the robustness of Irwin tests across multiple sites for detecting drug-induced neurological effects in mice, finding reduced variability after protocol harmonization.

Benefits of global mutant huntingtin lowering diminish over time in a Huntington's disease mouse model

This study shows that moderate global lowering of mutant Huntingtin in a HD mouse model delays some deficits but benefits diminish over time, suggesting long-term mHtt lowering may be needed for sustained benefit.