Bioreducible branched poly(modified nona-arginine) cell-penetrating peptide as a novel gene delivery platform

Yoo, Jisang; Lee, DaeYong; Gujrati, Vipul; Rejinold, N. Sanoj; Lekshmi, Kamali Manickavasagam; Uthaman, Saji; Jeong, Chan-Uk; Park, In Kyu; Jon, Sangyong; Kim, Yeu‐Chun

doi:10.1016/j.jconrel.2016.04.040

Cited by 64 publications

(52 citation statements)

References 44 publications

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“…Cell penetrating peptides (CPP) and other cationic peptides can be complexed with nucleic acids to form highly efficient delivery systems. Introducing a thiol group in the peptide helps to create a bioreducible polymeric peptide that can be specifically released in the intracellular environment and deliver both siRNA and plasmid DNA [100].…”

Section: Synthetic Nanoparticlesmentioning

confidence: 99%

MicroRNA-Based Drugs for Brain Tumors

et al. 2018

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MicroRNAs (miRNAs) are key regulatory elements encoded by the genome. A single miRNA can downregulate the expression of multiple genes involved in diverse functions. Because cancer is a disease with multiple gene aberrations, developing novel approaches to identify and modulate miRNA pathways may result in a breakthrough for cancer treatment. With a special focus on glioblastoma (GBM), this review provides an up-to-date summary of miRNA biogenesis, the role of miRNA in cancer resistance, and essential tools for modulating miRNA expression, as well as of clinically promising RNAi delivery systems and how they can be adapted for therapy.

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Section: Synthetic Nanoparticlesmentioning

confidence: 99%

MicroRNA-Based Drugs for Brain Tumors

et al. 2018

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“…A disulfide bond is a prevalent linker for reduction at tumor sites due to these having a more reducing intracellular environment than normal sites (Figure 4a). Yoo et al synthesized branched polyarginine peptides with cysteine residues forming disulfide bonds that release VEGF siRNA in the presence of intercellular GSH, as shown in Figure 4b [97]. Introducing the disulfide bonds into the cationic polymers reduced cell toxicity and improved transfection efficiency [98,99].…”

Section: Triggers For Gene Release In Cancer Cells Using Gene Tranmentioning

confidence: 99%

“…( b ) Schematic illustration of the synthesis of the branched modified R9 (B-mR9) cell penetrating peptide (CPP) and construction of pDNA and siRNA polyplexes. Reproduced with permission from [97]. Journal of Controlled Release, Elsevier 2017.…”

Section: Figurementioning

confidence: 99%

Trigger-Responsive Gene Transporters for Anticancer Therapy

et al. 2017

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In the current era of gene delivery, trigger-responsive nanoparticles for the delivery of exogenous nucleic acids, such as plasmid DNA (pDNA), mRNA, siRNAs, and miRNAs, to cancer cells have attracted considerable interest. The cationic gene transporters commonly used are typically in the form of polyplexes, lipoplexes or mixtures of both, and their gene transfer efficiency in cancer cells depends on several factors, such as cell binding, intracellular trafficking, buffering capacity for endosomal escape, DNA unpacking, nuclear transportation, cell viability, and DNA protection against nucleases. Some of these factors influence other factors adversely, and therefore, it is of critical importance that these factors are balanced. Recently, with the advancements in contemporary tools and techniques, trigger-responsive nanoparticles with the potential to overcome their intrinsic drawbacks have been developed. This review summarizes the mechanisms and limitations of cationic gene transporters. In addition, it covers various triggers, such as light, enzymes, magnetic fields, and ultrasound (US), used to enhance the gene transfer efficiency of trigger-responsive gene transporters in cancer cells. Furthermore, the challenges associated with and future directions in developing trigger-responsive gene transporters for anticancer therapy are discussed briefly.

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“…Recently, biodegradable CPPs have been developed to address the release issues. They can release the payloads into the cytoplasm via chemical reaction pathways, such as the reductive hydrolysis of disulfide bonds 146. Such biodegradable CPPs show a broad prospect for delivering CRISPR/Cas9 components.Ramakrishna et al147 reported a carrier-free strategy for delivering Cas9 ribonucleoproteins by conjugating CPPs with Cas9 proteins.…”

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confidence: 99%

Delivery of CRISPR/Cas9 for therapeutic genome editing

Wan

Xin

et al. 2019

The Journal of Gene Medicine

110

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The clustered, regularly‐interspaced, short palindromic repeat (CRISPR)‐associated nuclease 9 (CRISPR/Cas9) is emerging as a promising genome‐editing tool for treating diseases in a precise way, and has been applied to a wide range of research in the areas of biology, genetics, and medicine. Delivery of therapeutic genome‐editing agents provides a promising platform for the treatment of genetic disorders. Although viral vectors are widely used to deliver CRISPR/Cas9 elements with high efficiency, they suffer from several drawbacks, such as mutagenesis, immunogenicity, and off‐target effects. Recently, non‐viral vectors have emerged as another class of delivery carriers in terms of their safety, simplicity, and flexibility. In this review, we discuss the modes of CRISPR/Cas9 delivery, the barriers to the delivery process and the application of CRISPR/Cas9 system for the treatment of genetic disorders. We also highlight several representative types of non‐viral vectors, including polymers, liposomes, cell‐penetrating peptides, and other synthetic vectors, for the therapeutic delivery of CRISPR/Cas9 system. The applications of CRISPR/Cas9 in treating genetic disorders mediated by the non‐viral vectors are also discussed.

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Bioreducible branched poly(modified nona-arginine) cell-penetrating peptide as a novel gene delivery platform

Cited by 64 publications

References 44 publications

MicroRNA-Based Drugs for Brain Tumors

MicroRNA-Based Drugs for Brain Tumors

Trigger-Responsive Gene Transporters for Anticancer Therapy

Delivery of CRISPR/Cas9 for therapeutic genome editing

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