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EXP002440

Paper

Systemic peptide mediated delivery of an siRNA targeting α-syn in the CNS ameliorates the neurodegenerative process in a transgenic model of Lewy body disease (2019)

Peptide

ApoB11-9R

Sequence: NH2-RLTRKRGLKLAGGGGGRRRRRRRRR

RNA

siRNA

All experiment fields

Experiment Id EXP002440
Paper Systemic peptide mediated delivery of an siRNA targeting α-syn in the CNS ameliorates the neurodegen
Peptide ApoB11-9R
Delivery Success Class yes
In Vivo Flag yes
Uptake Confirmed yes
Label Confidence high
In Vitro Functional Effect
Endosomal Escape Evidence
Peptide Concentration
Rna Concentration intraperitoneal repeated dosing; exact siRNA mass not extracted from visible text
Mixing Ratio 1:10 siRNA:ApoB11
Formulation Format peptide/siRNA electrostatic complex
Formulation Components ApoB11 peptide + siα-syn
Size Nm
Zeta Mv
Model Scope in_vivo
Model Type in vivo
Cell Lines Or Primary Cells
Animal Model PDGF/α-syn transgenic mouse model of PD/DLB; non-transgenic mice also used for uptake controls
Administration Route intraperitoneal injection, twice weekly for 4 weeks
Output Type α-syn knockdown, neuronal protection, reduced neuroinflammation
Output Value Reduced α-syn immunoreactivity by ~65–70% in neocortex, ~40–50% in hippocampus, and ~50–55% in striatum; western blot showed ~30–40% reduction in α-syn; ameliorated NeuN neuronal loss and TH fiber loss; reduced GFAP/Iba1 neuroinflammation
Output Units
Output Notes FITC-siα-syn delivered across BBB to neurons and astrocytes; ApoB11 localized to ~55–60% of NeuN+ neurons and ~20–25% of GFAP+ astrocytes
Toxicity Notes No observable CNS toxicity stated for siα-syn delivery; study noted no TH loss/inflammation from partial knockdown
Curation Notes