OncotargetDec 29, 2016

Targeted delivery of gene-editing tools to prostate cancer using modified RNA and flexible carrier particles

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Mar 29, 2026

Abstract

The aptamer-liposome-CRISPR/Cas9 chimera shows significant cell-type binding specificity and gene silencing effects in vitro.

A flexible delivery system was developed to target and inhibit specific genes in prostate cancer cells.
The chimera utilized an RNA aptamer that binds to prostate-specific membrane antigen found on cancer cells.
Cationic liposomes were used to effectively deliver CRISPR/Cas9 to tumor cells targeting the polo-like kinase 1 gene.
In vitro experiments demonstrated a remarkable ability to silence the target gene.
In vivo studies indicated that gene silencing led to noticeable regression of prostate cancer.

Simplified

The potent ability of CRISPR/Cas9 system to inhibit the expression of targeted gene is being exploited as a new class of therapeutics for a variety of diseases. However, the efficient and safe delivery of CRISPR/Cas9 into specific cell populations is still the principal challenge in the clinical development of CRISPR/Cas9 therapeutics. In this study, a flexible aptamer-liposome-CRISPR/Cas9 chimera was designed to combine efficient delivery and increased flexibility. Our chimera incorporated an RNA aptamer that specifically binds prostate cancer cells expressing the prostate-specific membrane antigen as a ligand. Cationic liposomes were linked to aptamers by the post-insertion method and were used to deliver therapeutic CRISPR/Cas9 that target the survival gene, polo-like kinase 1, in tumor cells. We demonstrate that the aptamer-liposome-CRISPR/Cas9 chimeras had a significant cell-type binding specificity and a remarkable gene silencing effect in vitro. Furthermore, silencing promoted a conspicuous regression of prostate cancer in vivo. Importantly, the approach described here provides a universal means of cell type-specific CRISPR/Cas9 delivery, which is a critical goal for the widespread therapeutic applicability of CRISPR/Cas9 or other nucleic acid drugs.

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Funding

Competing interests

CONFLICTS OF INTEREST . The authors have no conflicts of interest.

PubMed

Targeted delivery of gene-editing tools to prostate cancer using modified RNA and flexible carrier particles

Abstract

Full Text

Funding

Competing interests

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