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Therapy: Gene Therapy

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Total 250 results found since Jan 2013.

Toxicological exploration of peptide-based cationic liposomes in siRNA delivery.
Abstract The toxicology of cationic liposomes was explored to advance clinical trials of liposome-mediated gene therapy through the analysis of a peptide cationic liposome with DOTAP as a positive control. We first investigated the delivery of luciferase siRNA by several peptide liposomes in mice bearing lung cancer A549 cell xenografts. Of these, a cationic liposome (CDO14) was selected for further investigation. CDO14 efficiently mediated IGF-1R-siRNA delivery and inhibited the growth of the A549 cell xenografts. The in vivo toxicity and toxicological mechanisms of the selected liposome were evaluated to assess ...
Source: Colloids and Surfaces - March 24, 2019 Category: Biotechnology Authors: Zhu Y, Meng Y, Zhao Y, Zhu J, Xu H, Zhang E, Shi L, Du L, Liu G, Zhang C, Xu X, Kang X, Zhen Y, Zhang S Tags: Colloids Surf B Biointerfaces Source Type: research

Tumor Chemosensitization through Oncogene Knockdown Mediated by Unique α-Tocopherylated Cationic Geminis.
Abstract Herein, siRNA transfection efficiency of a unique set of α-tocopherylated gemini lipids has been established in vitro and in vivo. High efficacy of oncogene silencing achieved using the biomacromolecular assembly, formed from siRNA complexes of co-liposomes containing an α-tocopherylated gemini lipid, has been utilized for tumor regression via chemosensitization. Delivery studies with the gemini bearing hydroxyethyl headgroup with octamethylene spacer (TH8S) pointed to a higher siRNA transfection efficacy than its analog without hydroxyethyl group (T8S). Owing to p53 upregulation, transfected cells show...
Source: Biomacromolecules - March 24, 2019 Category: Biochemistry Authors: Kamra M, Maiti B, Dixit A, Karande AA, Bhattacharya S Tags: Biomacromolecules Source Type: research

Engineering multifunctional bioactive citric acid-based nanovectors for intrinsical targeted tumor imaging and specific siRNA gene delivery in vitro/in vivo.
Abstract Targeted tumor imaging and efficient specific gene delivery in vivo has been one of the main challenges in gene-based cancer diagnosis and therapy. Herein, we engineered a citric acid-based polymer with intrinsical photoluminescence and gene loading capacity to achieve targeted delivery of siRNA and tumor imaging in vitro and in vivo. The multifunctional platform was formed from the self-assembling of poly (citric acid)-polymine conjugated with folic acid and rhodamine B (PPFR). PPFR showed stable photoluminescent ability and could effectively bind and protect the siRNA against RNase degradation. PPFR als...
Source: Biomaterials - February 2, 2019 Category: Materials Science Authors: Wang M, Guo Y, Xue Y, Niu W, Chen M, Ma PX, Lei B Tags: Biomaterials Source Type: research

Targeted delivery of Rab26 siRNA with precisely tailored DNA prism for lung cancer therapy.
In this study, a GTPase Rab26 was explored as a new potential therapeutic target using precisely tailored DNA prism for targeted lung cancer therapy. Specifically, a DNA prism platform with tunable targeting and siRNA loading capability is designed and synthesized. The DNA prisms were decorated with two functional units: a Rab26 siRNA as the drug and MUC-1 aptamers as targeting moiety for non-small cell lung cancer. The number and position of both siRNA and MUC-1 aptamer can be readily tuned. Native polyacrylamide gel electrophoresis (PAGE) and dynamic light scattering technique (DLS) demonstrate that all nanoprisms with d...
Source: Chembiochem - January 4, 2019 Category: Biochemistry Authors: Liu Q, Wang D, Xu Z, Huang C, Zhang C, He B, Mao C, Wang G, Qian H Tags: Chembiochem Source Type: research

Lipodendriplexes: A promising nanocarrier for enhanced gene delivery with minimal cytotoxicity.
In this study, we developed an advanced variant of the dendriplexes by encapsulating them within liposomes to enhance their gene delivery efficiency. This liposome encapsulated dendriplex system can further reduce unwanted cytotoxicity and enhance cellular uptake of nucleic acids. A broad range of lipid combinations were used to optimize the lipodendriplexes in terms of their physicochemical characteristics including size, shape and zeta potential. The optimized lipodendriplexes were tested for pDNA transfection, in vitro cell viability, cellular uptake, siRNA mediated knockdown, hemocompatibility, metastatic progression a...
Source: European Journal of Pharmaceutics and Biopharmaceutics - December 24, 2018 Category: Drugs & Pharmacology Authors: Tariq I, Reddy Pinnapireddy S, Duse L, Yasir Ali M, Ali S, Umair Amin M, Goergen N, Jedelská J, Schäfer J, Bakowsky U Tags: Eur J Pharm Biopharm Source Type: research

Solid Phase Synthesis and Self ‐Assembly of Higher‐Order siRNAs and their Bioconjugates
This article is protected by copyright. All rights reserved.
Source: Chemical Biology and Drug Design - November 27, 2018 Category: Biology Authors: Christopher N. Cultrara, Sunil Shah, Stephen D. Kozuch, Mayurbhai R. Patel, David Sabatino Tags: Special Issue Article Source Type: research

Methotrexate induced cell death mechanisms in MCF-7 adenocarcinoma breast cancer cells: Enhanced cytotoxicity following dff45-siRNA pre-treatment
Publication date: Available online 30 August 2018Source: SynergyAuthor(s): Fatemeh Kiani, Negin Rasouli, Tahereh Kashkoolinejad, Shahrokh Safarian, Seyed Jalal Zargar, Nader SheibaniAbstractThere are many efforts to diminish risks associated with the use of cancer chemotherapeutic agents through design and utilization of new strategies including gene therapy. We previously showed knockdown of dff45 or overexpression of dff40, the important apoptosis regulators, resulted in enhanced sensitization of cancer cells to chemotherapeutic agents. Here we show that pre-treatment with dff45-siRNA additively increased the overall cyt...
Source: Synergy - August 31, 2018 Category: Molecular Biology Source Type: research

Current non-viral siRNA delivery systems as a promissing treatment of skin diseases.
CONCLUSION: The treatment of skin diseases based on topical delivery of siRNA, which act by inhibiting the expression of target transcripts, offers many potential therapeutic advantages for suppressing genes into the skin. PMID: 30084329 [PubMed - as supplied by publisher]
Source: Current Pharmaceutical Design - August 7, 2018 Category: Drugs & Pharmacology Authors: Rosa J, Suzuki I, Kravicz M, Caron A, Pupo AV, Praca FG, Bentley MVLB Tags: Curr Pharm Des Source Type: research

Nanostructured Hyaluronic acid-based materials for the delivery of siRNA.
CONCLUSION: To overcome the problems associated with these emerging genetic tools, investigators have employed glycosaminoglycan HA-based biopolymers. This biopolymer offers a variety of properties such as biodegradability, biocompatibility, aqueous solubility, viscoelasticity, and non-immunogenicity. PMID: 30084325 [PubMed - as supplied by publisher]
Source: Current Pharmaceutical Design - August 7, 2018 Category: Drugs & Pharmacology Authors: Shah K, Chawla S, Agrawal A, Reddy G, Maheshwari R, Kalia K, Tekade RK Tags: Curr Pharm Des Source Type: research

Polyester-based nanoparticles for nucleic acid delivery
Publication date: Available online 25 July 2018Source: Materials Science and Engineering: CAuthor(s): Jing Zhao, Guojun Weng, Jianjun Li, Jian Zhu, Junwu ZhaoAbstractGene therapy is promising for the treatment of inherited diseases and complex diseases such as chronic infections and cancer. The advancement in science and technology has extended the scope of gene therapy from gene transfer to the delivery of a variety of nucleic acids such as mRNA, antisense oligonucleotides (ASOs), siRNA and miRNA. Nanoparticle delivery systems can efficiently protect the nucleic acids from enzymatic degradation and immune recognition, fac...
Source: Materials Science and Engineering: C - July 26, 2018 Category: Materials Science Source Type: research

Micelle-like nanoparticles as siRNA and miRNA carriers for cancer therapy.
Abstract Gene therapy has emerged as an alternative in the treatment of cancer, particularly in cases of resistance to chemo and radiotherapy. Different approaches to deliver genetic material to tumor tissues have been proposed, including the use of small non-coding RNAs due to their multiple mechanisms of action. However, such promise has shown limits in in vivo application related to RNA's biological instability and stimulation of immunity, urging the development of systems able to overcome those barriers. In this review, we discuss the use of RNA interference in cancer therapy with special attention to the role...
Source: Biomedical Microdevices - July 12, 2018 Category: Biomedical Engineering Authors: Costa DF, Torchilin VP Tags: Biomed Microdevices Source Type: research

Nanoscale polyelectrolyte complexes encapsulating mRNA and long-chained siRNA for combinatorial cancer gene therapy
Publication date: 25 August 2018Source: Journal of Industrial and Engineering Chemistry, Volume 64Author(s): Myung Goo Kim, Sung Duk Jo, Ji Hoon Jeong, Sun Hwa KimAbstractTo precisely regulate target genes that are abnormally expressed in cancers, we suggest an RNA-mediated multigene targeting system that co-encapsulates siRNA against vascular endothelial growth factor (VEGF) and mRNA encoding phosphatase and tensin homolog (PTEN). Polymerized long-chain siRNAs (L-siRNAs) formed stable and condensed nanocomplexes with mRNAs using thiolated glycol chitosans (tGCs) as gene carriers. The mRNA/L-siRNA/tGC nanocomplexes (MSNs) ...
Source: Journal of Industrial and Engineering Chemistry - July 11, 2018 Category: Chemistry Source Type: research

Potential of siRNA-albumin complex against cancer
Publication date: Available online 27 April 2018Source: Chemico-Biological InteractionsAuthor(s): Na Liu, Yan-Hua Qi, Chuan-Tao Cheng, Wen Bin Yang, Anshoo Malhotra, Qi ZhouAbstractRNA interference is a highly specific as well as efficient technology for gene therapy application in molecular oncology. The present study was planned to develop an efficient and stable tumor selective delivery mechanism for siRNA gene therapy for the purpose of both diagnosis as well as therapy. We have used 20 Male wistar rats for the formation of colon cancer model and utilized albumin as carrier molecule for the delivery of siRNA against va...
Source: Chemico Biological Interactions - July 11, 2018 Category: Biochemistry Source Type: research

Evaluation of siRNA and Cationic Liposomes Complexes as a Model for in vitro siRNA Delivery to Cancer Cells
This study aims to investigate the effect of different molar charge ratios (R+/-) between positive charges from microfluidics-produced cationic liposomes (CL) (egg phosphatidylcholine, 1,2-dioleoyl-3-trimethylammonium-propane and 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine) and negative charges from siRNA and on physico-chemical and morphological properties of the lipoplexes (CL/siRNA) as well as their in vitro luciferase silencing effect in HeLa cells. R+/- 3.27 was found to be the optimum point for complexation. This finding was confirmed by gel retardation and siRNA accessibility assays. According to Cryo-TEM analysis...
Source: Colloids and Surfaces A: Physicochemical and Engineering Aspects - July 11, 2018 Category: Chemistry Source Type: research