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Therapy: Gene Therapy

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Total 490 results found since Jan 2013.

Molecules, Vol. 22, Pages 231: Construction and Biological Evaluation of a Novel Integrin ανβ3-Specific Carrier for Targeted siRNA Delivery In Vitro
Conclusion: The chimeric peptide of c(RGD)2-9R can be developed as an effective siRNA delivery carrier and shows potential as a new strategy for RNAi-based gene therapy.
Source: Molecules - February 3, 2017 Category: Chemistry Authors: Xueqi Chen Meng Liu Rongfu Wang Ping Yan Chunli Zhang Chao Ma Lei Yin Tags: Article 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

Cholic acid-based mixed micelles as siRNA delivery agents for gene therapy
In this study, we demonstrate the use of lipids as co-surfactants for the preparation of mixed micelles to improve the siRNA delivery of cholic acid-based block copolymers. Poly(allyl glycidyl ether) (PAGE) and poly(ethylene glycol) (PEG) were polymerized on the surface of cholic acid to afford a star-shaped block copolymer with four arms (CA-PAGE-b-PEG)4. The allyl groups of PAGE were functionalized to bear primary or tertiary amines and folic acid was grafted onto the PEG chain end to increase cell uptake. (CA-PAGE-b-PEG)4 functionalized with either primary or tertiary amines show high siRNA complexation with close to 10...
Source: International Journal of Pharmaceutics - January 26, 2020 Category: Drugs & Pharmacology Source Type: research

Multiscale modeling of the cellular uptake of C6 peptide-siRNA complexes
In this study, multiscaled computational simulations of these peptides were performed in aqueous media, interrogating the relationship between the structure and behaviour. All atom molecular dynamic (MD) simulation results show that all CPPs show stable α-helical amphipathic secondary structures. Furthermore, docking calculations indicate that the C6 peptides can fit into the major groove of the siRNA double-helix, and once filled, could bind randomly along the minor grooves and to other, previously bound peptides. Coarse grained MD simulations were also used to generate free energy profiles for the dimerization of peptid...
Source: Computational Biology and Chemistry - April 24, 2022 Category: Bioinformatics Authors: P W C M Purijjala P V G M Rathnayake B T Kumara B C M Gunathunge R A A P Ranasinghe D N Karunaratne R J K U Ranatunga Source Type: research

Rigid nanoparticle-based delivery of anti-cancer siRNA: Challenges and opportunities.
Abstract Gene therapy is a promising strategy to treat various genetic and acquired diseases. Small interfering RNA (siRNA) is a revolutionary tool for gene therapy and the analysis of gene function. However, the development of a safe, efficient, and targetable non-viral siRNA delivery system remains a major challenge in gene therapy. An ideal delivery system should be able to encapsulate and protect the siRNA cargo from serum proteins, exhibit target tissue and cell specificity, penetrate the cell membrane, and release its cargo in the desired intracellular compartment. Nanomedicine has the potential to deal with...
Source: Biotechnology Advances - September 4, 2013 Category: Biotechnology Authors: Wang Z, Liu G, Zheng H, Chen X Tags: Biotechnol Adv Source Type: research

Inhibition of hepatocellular carcinoma growth using immunoliposomes for co-delivery of adriamycin and ribonucleotide reductase M2 siRNA.
Abstract The chemotherapy combined with gene therapy has received great attention. We developed targeted LPD (liposome-polycation-DNA complex) conjugated with anti-EGFR (epidermal growth factor receptor) Fab' co-delivering adriamycin (ADR) and ribonucleotide reductase M2 (RRM2) siRNA (ADR-RRM2-TLPD), to achieve combined therapeutic effects in human hepatocellular carcinoma (HCC) overexpressing EGFR. The antitumor activity and mechanisms of ADR-RRM2-TLPD were investigated. The results showed that RRM2 expression was higher in HCC than in non-HCC tissue, and RRM2 siRNA inhibited HCC cell proliferation, suggesting th...
Source: Biomaterials - September 20, 2013 Category: Materials Science Authors: Gao J, Chen H, Yu Y, Song J, Song H, Su X, Li W, Tong X, Qian W, Wang H, Dai J, Guo Y Tags: Biomaterials Source Type: research

Exosomes are natural carriers of exogenous siRNA to human cells in vitro
Conclusions: The results strongly suggest that exosomes effectively delivered the siRNA into the target cells. The therapeutic potential of exosome-mediated siRNA delivery was demonstrated in vitro by the strong knockdown of RAD51, a prospective therapeutic target for cancer cells. The results give an additional evidence of the ability to use human exosomes as vectors in cancer therapy, including RNAi-based gene therapy.
Source: Cell Communication and Signaling - November 18, 2013 Category: Molecular Biology Authors: Tatyana ShtamRoman KovalevElena VarfolomeevaEvgeny MakarovYury KilMichael Filatov Source Type: research

Tau Silencing by siRNA in the P301S Mouse model of Tauopathy.
Abstract Suppression of tau protein expression has been shown to improve behavioral deficits in mouse models of tauopathies, offering an attractive therapeutic approach. Experimentally this had been achieved by switching off the promoters controlling the transgenic human tau gene (MAPT), which is not possible in human patients. The aim of the present study was therefore to evaluate the effectiveness of small interfering RNAs (siRNAs) and their cerebral delivery to suppress human tau expression in vivo, which might be a therapeutic option for human tauopathies. We used primary cortical neurons of transgenic mice ex...
Source: Current Gene Therapy - November 12, 2014 Category: Genetics & Stem Cells Authors: Xu H, Rösler TW, Carlsson T, de Andrade A, Fiala O, Höllerhage M, Oertel WH, Goedert M, Aigner A, Höglinger GU Tags: Curr Gene Ther Source Type: research

Plasma hydrogenated cationic detonation nanodiamonds efficiently deliver to human cells in culture functional siRNA targeting the Ewing sarcoma junction oncogene.
Abstract The expression of a defective gene can lead to major cell dysfunctions among which cell proliferation and tumor formation. One promising therapeutic strategy consists in silencing the defective gene using small interfering RNA (siRNA). In previous publications we showed that diamond nanocrystals (ND) of primary size 35 nm, rendered cationic by polyethyleneimine-coating, can efficiently deliver siRNA into cell, which further block the expression of EWS/FLI-1 oncogene in a Ewing sarcoma disease model. However, a therapeutic application of such nanodiamonds requires their elimination by the organism, partic...
Source: Biomaterials - February 13, 2015 Category: Materials Science Authors: Bertrand JR, Pioche-Durieu C, Ayala J, Petit T, Girard HA, Malvy CP, Le Cam E, Treussart F, Arnault JC Tags: Biomaterials Source Type: research

Regulation of vascular smooth muscle cell autophagy by DNA nanotube-conjugated mTOR siRNA.
Abstract The efficient delivery of short interfering RNA (siRNA) is an enormous challenge in the field of gene therapy. Herein, we report a delivery nanosystem based on programmed DNA self-assembly mammalian target of rapamycin (mTOR) siRNA-loaded DNA nanotubes (DNA-NTs). We demonstrate that these siRNA-DNA-NTs can be effectively transfected into pulmonary arterial smooth muscle cells (PASMCs) via endocytosis; and that the loaded mTOR siRNA can induce obvious autophagy and inhibit cell growth under both normal and hypoxic conditions. Moreover, we found that mTOR siRNA can control the autophagy and proliferation of...
Source: Biomaterials - July 16, 2015 Category: Materials Science Authors: You Z, Qian H, Wang C, He B, Yan J, Mao C, Wang G Tags: Biomaterials Source Type: research

Gold-nanorods-siRNA nanoplex for improved photothermal therapy by gene silencing.
Abstract Nanomaterials-mediated photothermal therapy (PTT) often suffers from the fundamental cellular defense mechanism of heat shock response which leads to therapeutic resistance of cancer cells and reduces the therapeutic efficacy. Herein, a gold nanorods (GNRs)-siRNA platform with gene silencing capability is produced to improve the PTT efficiency. After surface modification, the GNRs show the ability to deliver siRNA oligos targeting BAG3 which is an efficient gene to block the heat-shock response. The synthesized GNRs-siRNA nanoplex exhibits excellent ability in the delivery of siRNA into cancer cells with ...
Source: Biomaterials - November 19, 2015 Category: Materials Science Authors: Wang BK, Yu XF, Wang JH, Li ZB, Li PH, Wang H, Song L, Chu PK, Li C Tags: Biomaterials Source Type: research

Ultrasound-Responsive Microbubbles for Sonography-guided siRNA Delivery
Publication date: Available online 28 December 2015 Source:Nanomedicine: Nanotechnology, Biology and Medicine Author(s): Ping Wang, Tinghui Yin, Jingguo Li, Bowen Zheng, Xiaoli Wang, Yiru Wang, Jian Zheng, Rongqin Zheng, Xintao Shuai RNA interfering is a gene therapeutic approach of great potential for cancer. However, tumor-targeted delivery of small interfering RNA (siRNA) solely based on the enhanced permeability and retention effect of nanocarriers is often insufficient. To address this challenge, siRNA encapsulated ultrasound-responsive microbubble (MB) was developed from polymeric siRNA micelles and liposo...
Source: Nanomedicine: Nanotechnology, Biology and Medicine - December 29, 2015 Category: Nanotechnology Source Type: research

Phenylboronic acid-functionalized polyamidoamine-mediated Bcl-2 siRNA delivery for inhibiting the cell proliferation
In conclusion, the PPP-mediated Bcl-2 siRNA delivery could potentially be an effective platform for solving the drug resistance and further achieving the combined chemotherapy and gene therapy in tumor treatment. Graphical abstract
Source: Colloids and Surfaces B: Biointerfaces - June 29, 2016 Category: Biochemistry Source Type: research