What question did this study set out to answer?

To evaluate the retinal uptake, distribution, gene-silencing efficacy, and ocular safety of novel self-delivering asymmetric RNAi compounds compared with conventional stabilized siRNAs.

November 1, 2013Journal of Ocular Pharmacology and Therapeutics

Novel Hydrophobically Modified Asymmetric RNAi Compounds (sd-rxRNA) Demonstrate Robust Efficacy in the Eye

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To evaluate the retinal uptake, distribution, gene-silencing efficacy, and ocular safety of novel self-delivering asymmetric RNAi compounds compared with conventional stabilized siRNAs.
Compared sd-rxRNA compounds to chemically stabilized siRNAs in vitro using ARPE-19 human retinal pigment epithelial cells.
Assessed retinal penetration, duration of target mRNA knockdown, and ocular safety following intravitreal injection in mouse and rabbit eye models without delivery vehicles.
sd-rxRNAs achieved uniform cellular uptake and full retinal penetration in both animal models, whereas stabilized siRNAs showed no detectable uptake in vitro or in vivo.
Treatment without transfection reagents led to significant target mRNA reduction that persisted for 14 to 21 days in vivo.
A single intravitreal administration caused no alterations in retinal morphology or visual function, indicating preliminary ocular safety.

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Implication

Preclinical study demonstrates robust cellular uptake and sustained gene silencing in ocular models, highlighting the potential of self-delivering RNAi compounds for treating eye diseases.

Cite This Study

Byrne et al. (2013) studied this question.

synapsesocial.com/papers/6a83b0568df3fed3c85bce50 https://doi.org/10.1089/jop.2013.0148

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Implication

Preclinical study demonstrates robust cellular uptake and sustained gene silencing in ocular models, highlighting the potential of self-delivering RNAi compounds for treating eye diseases.

Novel Hydrophobically Modified Asymmetric RNAi Compounds (sd-rxRNA) Demonstrate Robust Efficacy in the Eye

Key Points

Discussion

Implication

Cite This Study

Novel Hydrophobically Modified Asymmetric RNAi Compounds (sd-rxRNA) Demonstrate Robust Efficacy in the Eye

Key Points

Discussion

Implication

Cite This Study

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