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Therapy: Gene Therapy

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Total 490 results found since Jan 2013.

Efficient Transfection of siRNA by Peptide Dendrimer-Lipid Conjugates.
Abstract Efficient delivery of small interfering RNA (siRNA) into cells is the basis of target-gene-specific silencing and, ultimately, gene therapy. However, current transfection reagents are relatively inefficient, and very few studies provide the sort of systematic understanding based on structure-activity relationships that would provide rationales for their improvement. This work established peptide dendrimers (administered with cationic lipids) as siRNA transfection reagents and recorded structure-activity relationships that highlighted the importance of positive charge distribution in the two outer layers a...
Source: Chembiochem - November 8, 2016 Category: Biochemistry Authors: Kwok A, Eggimann GA, Heitz M, Reymond JL, Hollfelder F, Darbre T Tags: Chembiochem Source Type: research

Engineered Exosomes for Targeted Transfer of siRNA to HER2 Positive Breast Cancer Cells.
Abstract Exosomes are the best options for gene targeting, because of their natural, nontoxic, non-immunogenic, biodegradable, and targetable properties. By engineering exosome-producing cells, ligands can be expressed fusing with exosomal surface proteins for targeting cancer cell receptors. In the present study, HER2-positive breast cancer cells were targeted with a modified exosome producing engineered HEK293T cell. For this purpose, the HEK293T cells were transduced by a lentiviral vector bearing-LAMP2b-DARPin G3 chimeric gene. Stable cells expressing the fusion protein were selected, and the exosomes produced...
Source: Applied Biochemistry and Biotechnology - June 28, 2018 Category: Biochemistry Authors: Limoni SK, Moghadam MF, Moazzeni SM, Gomari H, Salimi F Tags: Appl Biochem Biotechnol Source Type: research

Anti-angiogenic treatment of endometriosis via anti-VEGFA siRNA delivery by means of peptide-based carrier in a rat subcutaneous model
Anti-angiogenic treatment of endometriosis via anti-VEGFA siRNA delivery by means of peptide-based carrier in a rat subcutaneous model, Published online: 25 September 2018; doi:10.1038/s41434-018-0042-7Anti-angiogenic treatment of endometriosis via anti-VEGFA siRNA delivery by means of peptide-based carrier in a rat subcutaneous model
Source: Gene Therapy - September 25, 2018 Category: Genetics & Stem Cells Authors: Anna Egorova Mariya Petrosyan Marianna Maretina Natalia Balashova Lyudmila Polyanskih Vladislav Baranov Anton Kiselev Source Type: research

Toxicological Exploration of Peptide-based Cationic Liposomes in siRNA Delivery
Publication date: Available online 25 March 2019Source: Colloids and Surfaces B: BiointerfacesAuthor(s): Yanyan Zhu, Yao Meng, Yinan Zhao, Jie Zhu, Hong Xu, Enxia Zhang, Lei Shi, Linying Du, Guoliang Liu, Chuanmin Zhang, Xiaodong Xu, Xiaohui Kang, Yuhong Zhen, Shubiao ZhangAbstractThe 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 catio...
Source: Colloids and Surfaces B: Biointerfaces - March 28, 2019 Category: Biochemistry Source Type: research

Hydroxyl-Rich PGMA-Based Cationic Glycopolymers for Intracellular siRNA Delivery: Biocompatibility and Effect of Sugar Decoration Degree.
Abstract The ErbB family of proteins, structurally related to the epidermal growth factor receptor (EGFR), is found to be over-expressed in many cancers such as gliomas, lung and cervical carcinomas. Gene therapy allows to modify the expression of genes like ErbB and has been a promising strategy to target oncogenes and tumor suppressor genes. In the current work, novel hydroxyl-rich polyglycidyl methacrylate (PGMA)-based cationic glycopolymers were designed for intracellular small interfering RNA (siRNA) delivery to silence the EGFR gene. The cationic polymers with different sugar decoration degrees (0%, 9%, and ...
Source: Biomacromolecules - April 9, 2019 Category: Biochemistry Authors: Chen Y, Diaz-Dussan D, Peng YY, Narain R Tags: Biomacromolecules Source Type: research

Use of polymeric CXCR4 inhibitors as siRNA delivery vehicles for the treatment of acute myeloid leukemia
Use of polymeric CXCR4 inhibitors as siRNA delivery vehicles for the treatment of acute myeloid leukemia, Published online: 26 April 2019; doi:10.1038/s41417-019-0095-9Use of polymeric CXCR4 inhibitors as siRNA delivery vehicles for the treatment of acute myeloid leukemia
Source: Cancer Gene Therapy - April 25, 2019 Category: Cancer & Oncology Authors: Yiqian Wang Ying Xie Jacob Williams Yu Hang Lisa Richter Michelle Becker Catalina Amador David Oupick ý R. Katherine Hyde Source Type: research

Silencing of IL-6 and STAT3 by siRNA loaded hyaluronate-N,N,N-trimethyl chitosan nanoparticles potently reduces cancer cell progression.
Abstract The immunosuppressive nature of the tumor microenvironment is a critical problem that should be considered before the design of immunotherapies. Interleukin (IL)-6 and its related downstream molecules such as signal transducer and activator of transcription (STAT)3 play an important role in the cancer progression, which can be considered as potential therapeutic targets. In the present study, we generated the active-targeted hyaluronate (HA) recoated N, N, N-trimethyl chitosan (TMC) nanoparticles (NPs) to deliver IL-6- and STAT3-specific small interfering RNAs (siRNAs) to the CD44-expressing cancer cells....
Source: International Journal of Biological Macromolecules - January 27, 2020 Category: Biochemistry Authors: Masjedi A, Ahmadi A, Atyabi F, Farhadi S, Irandoust M, Khazaei-Poul Y, Chaleshtari MG, Fathabad ME, Baghaei M, Haghnavaz N, Baradaran B, Hojjat-Farsangi M, Ghalamfarsa G, Sabz G, Hasanzadeh S, Jadidi-Niaragh F Tags: Int J Biol Macromol Source Type: research

Study of Double-Targeting Nanoparticles Loaded With MCL-1 siRNA and Dexamethasone for Adjuvant-induced Arthritis Therapy.
In this study, HA-coated pH-responsive nanoparticles loaded with MCL-1 small interfering RNA (siRNA) and dexamethasone (HNPs/MD) were developed for RA treatment. The HNPs/MD had a mean particle size of 117.07 ± 2.21 nm and zeta potential of 15.53 ± 1.06 mV. The release rates of both MCL-1 siRNA and dexamethasone from the HNPs/MD at pH 4.5 and 6.0 were higher than those at pH 7.4, indicating that the nanoparticles were acid-sensitive. Cytotoxicity assays showed that compared with single drug loaded nanoparticles, the HNPs/MD showed higher cytotoxicity to activated macrophages. The superior therapeutic effect of HNPs/MD wa...
Source: European Journal of Pharmaceutics and Biopharmaceutics - July 14, 2020 Category: Drugs & Pharmacology Authors: Li X, Yu C, Meng X, Hou Y, Cui Y, Zhu T, Li Y, Teng L, Sun F, Li Y Tags: Eur J Pharm Biopharm Source Type: research

Branched Antisense and siRNA Co-assembled Nanoplatform for Combined Gene Silencing and Tumor Therapy.
Abstract Chemically modified DNA has been widely developed to fabricate various nucleic acid nanostructures for biomedical applications. Herein, we report a facile strategy for construction of branched antisense DNA and small interfering RNA (siRNA) co-assembled nanoplatform for combined gene silencing in vitro and in vivo. In our design, the branched antisense can efficiently capture siRNA with 3' overhangs through DNA-RNA hybridization. After being equipped with an active targeting group and an endosomal escape peptide by host-guest interaction, the tailored nucleic acid nanostructure functions efficiently as bo...
Source: Angewandte Chemie - October 15, 2020 Category: Chemistry Authors: Liu J, Lu X, Wu T, Wu X, Han L, Ding B Tags: Angew Chem Int Ed Engl Source Type: research

Suppressing Sart1 to modulate macrophage polarization by siRNA-loaded liposomes: a promising therapeutic strategy for pulmonary fibrosis
Conclusion: Our study provides a valuable reference for modulating macrophage polarization and a promising strategy for the treatment of pulmonary fibrosis in clinical settings.
Source: Theranostics - January 15, 2021 Category: Molecular Biology Authors: Ting Pan, Qing Zhou, Kang Miao, Lei Zhang, Guorao Wu, Jun Yu, Yongjian Xu, Weining Xiong, Yong Li, Yi Wang Tags: Research Paper Source Type: research

Preparation and Preliminary Evaluation of Dual-functional Nanoparticles for MRI and siRNA Delivery
In conclusion, the visualized polycationic lipid siRNA delivery vehicle (LP-PEI1800-SPION) was successfully prepared in this experiment, which provides a research basis for further theranostics of liver cancer.
Source: Iranian Journal of Pharmaceutical Research - December 26, 2021 Category: Drugs & Pharmacology Source Type: research

Synthetic Conjugated Oligoelectrolytes Are Effective siRNA Transfection Carriers: Relevance to Pancreatic Cancer Gene Therapy
Biomacromolecules. 2022 Feb 9. doi: 10.1021/acs.biomac.1c01498. Online ahead of print.ABSTRACTConjugated oligoelectrolyte COE-S6 contains an elongated conjugated core with three cationic charges at each termini of the internal core. As an analogue of bolaamphiphiles, these structural attributes lead to the formation of spherical nanoplexes with Dh = 205 ± 5.0 nm upon mixing with small interfering RNA (siRNA). COE-S6/siRNA nanocomplexes were shown to be protective toward RNase, stimulate endosome escape, and achieve transfection efficiencies comparable to those achieved with commercially available LIP3000. Moreover, COE-S6...
Source: Biomacromolecules - February 9, 2022 Category: Biochemistry Authors: Chengbin Yang Haoqiang Huang Nishtha Manish Singh Cheng Zhou Guang Yang Zhourui Xu Haoming Lin Gaixia Xu Ken-Tye Yong Guillermo C Bazan Source Type: research

PreS/2-21-Guided siRNA Nanoparticles Target to Inhibit Hepatitis B Virus Infection and Replication
A viable therapy is needed to overcome the deadlock of the incurable chronic hepatitis B (CHB). The prolonged existence of covalently closed circular DNA (cccDNA) and integrated HBV DNA in the nucleus of hepatocytes is the root cause of CHB. As a result, it is critical to successfully suppress HBV DNA replication and eliminate cccDNA. RNA interference has been proven in recent research to silence the expression of target genes and thereby decrease HBV replication. However, siRNA is susceptible to be degraded by RNA enzymes in vivo, making it difficult to deliver successfully and lacking of tissue targeting. To exploit the ...
Source: Frontiers in Immunology - April 29, 2022 Category: Allergy & Immunology Source Type: research

Cancers, Vol. 14, Pages 3597: Photochemical Internalization of siRNA for Cancer Therapy
obo In the race to design ever more effective therapy with ever more focused and controlled actions, nanomedicine and phototherapy seem to be two allies of choice. Indeed, the use of nanovectors making it possible to transport and protect genetic material is becoming increasingly important. In addition, the use of a method allowing the release of genetic material in a controlled way in space and time is also a strategy increasingly studied thanks to the use of lasers. In parallel, the use of interfering RNA and, more particularly, of small-interfering RNA (siRNA) has demonstrated significant potential for gene therapy....
Source: Cancers - July 23, 2022 Category: Cancer & Oncology Authors: Lamiaa Mohamed Ahmed Ali Magali Gary-Bobo Tags: Review Source Type: research