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Effective lung-targeted RNAi in mice with peptide-based delivery of nucleic acid.

ABSTRACT: We have previously developed efficient peptide-based nucleic acid delivery vectors PF14 and NF55, where we have shown that these vectors preferentially transfect lung tissue upon systemic administration with the nucleic acid. In the current work, we have explored the utilization and potential of these vectors for the lung-targeted gene therapy. Accordingly, we assessed the efficacy of these peptides in (i) two different lung disease models - acute lung inflammation and asthma in mice and (ii) using two different nucleic acid cargos - siRNA and pDNA encoding shRNA. Using RNAi against cytokine TNF?, we showed efficient anti-inflammatory effects in both disease models and observed decreased disease symptoms. Our results highlight the potential of our transfection vectors for lung gene therapy.

SUBMITTER: Kurrikoff K

PROVIDER: S-EPMC6934651 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature

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Effective lung-targeted RNAi in mice with peptide-based delivery of nucleic acid.

Kurrikoff Kaido K Freimann Krista K Veiman Kadi-Liis KL Peets Elin Madli EM Piirsoo Andres A Langel Ülo Ü

Scientific reports 20191227 1

We have previously developed efficient peptide-based nucleic acid delivery vectors PF14 and NF55, where we have shown that these vectors preferentially transfect lung tissue upon systemic administration with the nucleic acid. In the current work, we have explored the utilization and potential of these vectors for the lung-targeted gene therapy. Accordingly, we assessed the efficacy of these peptides in (i) two different lung disease models - acute lung inflammation and asthma in mice and (ii) us ...[more]

PMID: 31882941

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