Production and clinical development of nanoparticles for gene delivery

Abstract

Gene therapy is a promising strategy for specific treatment of numerous gene-associated human diseases by intentionally altering the gene expression in pathological cells. A successful clinical application of gene-based therapy depends on an efficient gene delivery system. Many efforts have been attempted to improve the safety and efficiency of gene-based therapies. Nanoparticles have been proved to be the most promising vehicles for clinical gene therapy due to their tunable size, shape, surface, and biological behaviors. In this review, the clinical development of nanoparticles for gene delivery will be particularly highlighted. Several promising candidates, which are closest to clinical applications, will be briefly reviewed. Then, the recent developments of nanoparticles for clinical gene therapy will be identified and summarized. Finally, the development of nanoparticles for clinical gene delivery in future will be prospected.

Macrophages loaded CpG and GNR-PEI for combination of tumor photothermal therapy and immunotherapy

“…Significantly improved gene transfection efficiency was achieved by this novel system compared with commercial PEI, due to their capacities of targeting to tumor cells and escape from endo-lysosomal compartments. Although dramatic contributions to tumor therapy have been achieved by GNR based nanocarriers, as exogenous materials, they can be recognized and cleared immediately by immune systems once injected into the bloodstream, which severely restricts their clinical application [21][22][23][24].…”

Phosphonium polymers for gene delivery

“…[1][2][3][4][5] To this aim, synthetic polymers are particularly attractive, due to increasingly efficient chemical routes for their synthesis, and the possibility to further modify the delivery systems through post polymerisation strategies. 6 Within this context, controlled radical polymerisation (CRP, also known as reversibledeactivation radical polymerisation, RDRP) techniques have been extensively investigated, as they enable control over the polymer composition, architecture as well as molecular weight and dispersity.…”

Quantification of siRNA Duplexes Bound to Gold Nanoparticle Surfaces

“…To facilitate passage across negatively charged cell membranes, siRNAs are typically complexed with cationic transfection agents. These cationic agents are useful for in vitro studies, but their high toxicity precludes in vivo use [3, 4]. Accordingly, researchers are developing new strategies to enable in vivo siRNA delivery, and the most common approach is to use nanoparticles as siRNA carriers [3–5].…”

“…These cationic agents are useful for in vitro studies, but their high toxicity precludes in vivo use [3, 4]. Accordingly, researchers are developing new strategies to enable in vivo siRNA delivery, and the most common approach is to use nanoparticles as siRNA carriers [3–5]. Nanoparticles are advantageous as siRNA delivery vehicles because they can overcome several of the aforementioned challenges related to siRNA delivery [3–5].…”

“…Accordingly, researchers are developing new strategies to enable in vivo siRNA delivery, and the most common approach is to use nanoparticles as siRNA carriers [3–5]. Nanoparticles are advantageous as siRNA delivery vehicles because they can overcome several of the aforementioned challenges related to siRNA delivery [3–5]. The two main methods of siRNA delivery using nanoparticles are encapsulation, wherein siRNAs are entrapped inside porous nanoparticles or within layers of positively charged materials surrounding nanoparticles [4, 6–10], and conjugation, wherein siRNAs are bound to nanoparticle surfaces and exposed as the outer layer [5, 11–14].…”