Non‐Viral Nucleic Acid Delivery System for RNA Therapeutics

Liu, Lianxiao; Zhou, Yiming; Xu, Anqiu; Wang, Chenglong; Li, Ning; Huang, Hongwei; Zhang, Yuntao; Yang, Xiaoming

doi:10.1002/adtp.202300005

Cited by 4 publications

(4 citation statements)

References 249 publications

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“…The CPCs are well soluble (>5 mg/mL) in alcohol and PBS buffer (20 mM, pH 7.4). 1 H NMR spectrum analysis (Figure 2a) indicated that the chemical shifts of the CPCs were in good agreement with their chemical structures. Moreover, no signals at δ 1.5 ppm, corresponding to the protons of the boc-protecting group, were observed, suggesting complete removal of this group.…”

Section: Preparation and Characterization Of Cpcs As Nonviral Gene Ca...mentioning

confidence: 65%

“…The THP-PNC product was purified on a silica column with DCM as a dilute and obtained as a solid powder after solvent evaporation (yield: 0.33 g, 32%). The monomers were characterized by 1 1b). As a procedure for the synthesis of BHE-BAP, BHE-PNC (0.83 g, 1.5 mmol) and BAP (0.31 g, 1.5 mmol) were dissolved in anhydrous DMF (2 mL) as a solvent.…”

Section: Methodsmentioning

confidence: 99%

“…Numerous cationic polymers as nonviral gene carriers for nucleic acid delivery have been under extensive investigation during the past decades. 1 Among these carriers, the polyethylenimines (PEIs) containing several protonatable amino groups have received much attention on their applications for nonviral gene therapy against cancers. 2 In this regard, 25 kDa branched PEI (BPEI) is well recognized as one of the most potent polymeric carriers for in vitro gene delivery against different cancer cells because of its robust gene binding ability to form nanopolyplexes and high buffering capacity to help efficient gene escape from acidified endosomes.…”

Section: Introductionmentioning

confidence: 99%

“…Numerous cationic polymers as nonviral gene carriers for nucleic acid delivery have been under extensive investigation during the past decades . Among these carriers, the polyethylenimines (PEIs) containing several protonatable amino groups have received much attention on their applications for nonviral gene therapy against cancers .…”

Section: Introductionmentioning

confidence: 99%

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Degradable Cationic Polycarbamates Designed for Robust IL-12 Gene Therapy against Metastatic Lung Cancer

An,

Li,

et al. 2024

ACS Appl. Polym. Mater.

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Polyethylenimine (PEI)-based gene carriers hold a massive promise for nonviral gene delivery against cancers. However, their poor degradability regularly results in rather high cytotoxicity, thus seriously hampering further clinical translations. Herein, a group of degradable cationic polycarbamates (CPCs) were finely designed to possess similar chemical structures with the PEIs for nonviral gene transfection. These CPCs containing multiple primary (or secondary) plus tertiary amino groups were synthesized by the polycondensation reaction of tertiary amino-containing primary diamines and the biscarbonates with butoxycarbonyl-protected primary (or secondary) amino groups, followed by the removal of the protecting group. These CPCs displayed degradation profiles under physiological conditions and degraded more rapidly when comprising the secondary amino groups compared with the primary amino residues. Moreover, these CPCs had favorable gene delivery properties including strong gene condensation ability and high buffering capacity as well as less cytotoxicity in comparison with nondegradable PEI controls, giving rise to efficient in vitro gene transfection in varied cells. Particularly, the secondary amino-containing CPCs based on the Nmethyl-2, 2′-diaminodiethylamine (denoted as BHE-MDE) caused excellent in vitro transfection activity selectively in type-II alveolar epithelial cells compared to other cells. The transfection efficiency of the BHE-MDE in the epithelial cells was comparable to that of 25 kDa linear PEI (LPEI) as a positive control. In the C57BL/6 mouse model, similar to this positive control, the BHE-MDE induced a high level of transgene expression in the lung and marked IL-12 expression in the mouse peripheral blood. As such, the BHE-MDE-mediated IL-12 expression afforded highly robust gene therapy against metastatic LLC cells in the lung of mice, thereby prolonging their survival time. The results indicate great potential of degradable LPEI-mimetic CPCs for nonviral gene therapy against metastatic lung cancer.

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Section: Preparation and Characterization Of Cpcs As Nonviral Gene Ca...mentioning

confidence: 65%

Section: Methodsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Degradable Cationic Polycarbamates Designed for Robust IL-12 Gene Therapy against Metastatic Lung Cancer

An,

Li,

et al. 2024

ACS Appl. Polym. Mater.

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Targeting DNA damage repair mechanism by using RAD50-silencing siRNA nanoparticles to enhance radiotherapy in triple negative breast cancer

Zetrini,

Abbasi,

et al. 2024

Materials Today Bio

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Delivery of nucleic acids using nanomaterials

Qin,

Ou,

Zha

et al. 2023

Mol Biomed

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The increasing number of approved nucleic acid therapeutics demonstrates the potential for the prevention and treatment of a broad spectrum of diseases. This trend underscores the significant impact and promise of nucleic acid-based treatments in the field of medicine. Nevertheless, employing nucleic acids as therapeutics is challenging due to their susceptibility to degradation by nucleases and their unfavorable physicochemical characteristics that hinder delivery into cells. Appropriate vectors play a pivotal role in improving nucleic acid stability and delivering nucleic acids into specific cells. The maturation of delivery systems has led to breakthroughs in the development of therapeutics based on nucleic acids such as DNA, siRNA, and mRNA. Non-viral vectors have gained prominence among the myriad of nanomaterials due to low immunogenicity, ease of manufacturing, and simplicity of cost-effective, large-scale production. Here, we provide an overview of the recent advancements in nanomaterials for nucleic acid delivery. Specifically, we give a detailed introduction to the characteristics of polymers, lipids, and polymer-lipid hybrids, and provide comprehensive descriptions of their applications in nucleic acid delivery. Also, biological barriers, administration routes, and strategies for organ-selective delivery of nucleic acids are discussed. In summary, this review offers insights into the rational design of next-generation delivery vectors for nucleic acid delivery.

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Non‐Viral Nucleic Acid Delivery System for RNA Therapeutics

Cited by 4 publications

References 249 publications

Degradable Cationic Polycarbamates Designed for Robust IL-12 Gene Therapy against Metastatic Lung Cancer

Degradable Cationic Polycarbamates Designed for Robust IL-12 Gene Therapy against Metastatic Lung Cancer

Targeting DNA damage repair mechanism by using RAD50-silencing siRNA nanoparticles to enhance radiotherapy in triple negative breast cancer

Delivery of nucleic acids using nanomaterials

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