Systemic Delivery of siRNA Down Regulates Brain Prion Protein and Ameliorates Neuropathology in Prion Disorder - Archive ouverte HAL Accéder directement au contenu
Article Dans Une Revue PLoS ONE Année : 2014

Systemic Delivery of siRNA Down Regulates Brain Prion Protein and Ameliorates Neuropathology in Prion Disorder

(1, 2) , (2) , (3) , (3) , (2) , (3) , (2) , (3) , (3) , (1) , (1) , (2) , (4) , (4) , (3) , (3) , (1, 2)
1
2
3
4

Résumé

One of the main challenges for neurodegenerative disorders that are principally incurable is the development of new therapeutic strategies, which raises important medical, scientific and societal issues. Creutzfeldt-Jakob diseases are rare neurodegenerative fatal disorders which today remain incurable. The objective of this study was to evaluate the efficacy of the down-regulation of the prion protein (PrP) expression using siRNA delivered by, a water-in-oil microemulsion, as a therapeutic candidate in a preclinical study. After 12 days rectal mucosa administration of Aonys/PrP-siRNA in mice, we observed a decrease of about 28% of the brain PrP(C) level. The effect of Aonys/PrP-siRNA was then evaluated on prion infected mice. Several mice presented a delay in the incubation and survival time compared to the control groups and a significant impact was observed on astrocyte reaction and neuronal survival in the PrP-siRNA treated groups. These results suggest that a new therapeutic scheme based an innovative delivery system of PrP-siRNA can be envisioned in prion disorders.
Fichier principal
Vignette du fichier
pone.0088797.pdf (7.31 Mo) Télécharger le fichier
Origine : Fichiers éditeurs autorisés sur une archive ouverte
Loading...

Dates et versions

hal-02507518 , version 1 (13-03-2020)

Licence

Paternité - CC BY 4.0

Identifiants

Citer

Sylvain Lehmann, Aroa Relaño-Ginès, Sarah Resina, Elsa Brillaud, Danielle Casanova, et al.. Systemic Delivery of siRNA Down Regulates Brain Prion Protein and Ameliorates Neuropathology in Prion Disorder. PLoS ONE, 2014, 9 (2), pp.e88797. ⟨10.1371/journal.pone.0088797⟩. ⟨hal-02507518⟩
60 Consultations
49 Téléchargements

Altmetric

Partager

Gmail Facebook Twitter LinkedIn More