Effective Gene Therapy in a Mouse Model of Prion Diseases
Author Information
Author(s): Toupet Karine, Compan Valérie, Crozet Carole, Mourton-Gilles Chantal, Mestre-Francés Nadine, Ibos Françoise, Corbeau Pierre, Verdier Jean-Michel, Perrier Véronique
Primary Institution: Univ Montpellier 2, Montpellier, France
Hypothesis
Can prion diseases be treated with the prion protein itself?
Conclusion
Chronic injections of dominant negative lentiviral vectors into the brain may be a promising approach for a curative treatment of prion diseases.
Supporting Evidence
- Chronic injections of PrPQ167R virions extended the survival of treated mice by 30 days.
- Behavioral improvements were observed in mice treated with PrPQ167R.
- Significant reduction in spongiosis and astrocytic gliosis was noted in the brains of treated mice.
Takeaway
Scientists found a way to help sick mice live longer by giving them special medicine that helps fight a brain disease called prion disease.
Methodology
Mice were treated with lentiviral vectors carrying dominant negative mutants of the PrP gene and observed for changes in survival and behavior.
Limitations
The study was conducted in mice, and results may not directly translate to humans.
Participant Demographics
C57Bl/6J mice were used in the study.
Statistical Information
P-Value
p<0.001
Statistical Significance
p<0.001
Digital Object Identifier (DOI)
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