[HTML][HTML] Systemic delivery of siRNA down regulates brain prion protein and ameliorates neuropathology in prion disorder
S Lehmann, A Relano-Gines, S Resina, E Brillaud… - PLoS …, 2014 - journals.plos.org
S Lehmann, A Relano-Gines, S Resina, E Brillaud, D Casanova, C Vincent, C Hamela…
PLoS One, 2014•journals.plos.orgOne 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 …
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 …
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 PrPC 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.
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