Countering Fragile X Syndrome with Reelin

Advantages: 

  • Precision Targeting
  • Minimized Side Effects
  • Durability

 Business Summary: 

Reelin in Fragile X Syndrome presents a novel and innovative strategy for addressing Fragile X Syndrome (FXS), a common inherited intellectual disability. This study is distinguished by its meticulous analysis of the distinctions between pre- and post-synaptic markers in an FXS mouse model. The researchers have devised a revolutionary Reelin construct known as Rfx, which is efficiently delivered via adeno-associated virus (AAV9) gene therapy. Examining the effects of Rfx on FXS rodents, this study demonstrates a remarkable cognitive recovery. These ground-breaking discoveries not only deepen our understanding of FXS, but also pave the way for targeted therapies that hold great promise for enhancing the lives of those affected by this condition. The research demonstrates a substantial advance in the field of Fragile X Syndrome and highlights the potential impact of Reelin-based interventions.

Figure 1 showcases a micrograph of a western blot analysis focused on PSD95, a post-synaptic marker, in the soluble fraction of the hippocampus. The experiment includes four groups: Fmr1 KO mice treated with AAV9-Rfx, Fmr1 KO mice treated with saline, wild-type (Tyr) mice treated with AAV9-Rfx, and Tyr mice treated with saline. Each group consists of six animals. The results provide a visual representation of the PSD95 levels in these different groups, offering compelling evidence for the impact of the treatment. The figure supports the study's conclusion that the administration of AAV9-Rfx to Fmr1 KO mice leads to the recovery of PSD95 levels, suggesting a potential mechanism for the cognitive rescue observed in these mice.

 

Desired Partnerships: 

  • License 
  • Sponsored Research 
  • Co-Development 
Patent Information: