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Therapy: Gene Therapy

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Total 490 results found since Jan 2013.

Composite liposome-PEI/nucleic acid lipopolyplexes for safe and efficient gene delivery and gene knockdown
Publication date: 1 October 2017 Source:Colloids and Surfaces B: Biointerfaces, Volume 158 Author(s): Shashank Reddy Pinnapireddy, Lili Duse, Boris Strehlow, Jens Schäfer, Udo Bakowsky Cytotoxicity is a major drawback impeding the therapeutic use of gene delivery and gene down-regulation vehicles. Apart from cytotoxicity, rapid degradation and low cellular uptake are other major factors affecting therapeutic use. Considering the above factors, formulation and development of PEI (Polyethylenimine) based, liposome encapsulated delivery vehicles with improved transfection efficiency and low cytotoxicity which can be used fo...
Source: Colloids and Surfaces B: Biointerfaces - July 4, 2017 Category: Biochemistry Source Type: research

Current state of the art for cardiac arrhythmia gene therapy
Publication date: Available online 19 June 2017 Source:Pharmacology & Therapeutics Author(s): J. Kevin Donahue Cardiac arrhythmias are a leading cause of morbidity and mortality. Currently available therapeutic options lack sufficient efficacy and safety. Gene therapy has been proposed for treatment of cardiac arrhythmias. This review will discuss the current state of development for arrhythmia gene therapy. So far, all published studies are short-term, proof-of-concept animal studies. Potential replacement of cardiac pacemakers has been shown for combination gene therapy using the HCN2 gene and either the gene fo...
Source: Pharmacology and Therapeutics - June 20, 2017 Category: Drugs & Pharmacology Source Type: research

Non-viral Delivery Systems for breast cancer gene therapy.
CONCLUSION: In this review, we focus on recent advances in the intracellular delivery of DNA and siRNA to the cancer cells with emphasis on breast cancer. PMID: 28595562 [PubMed - as supplied by publisher]
Source: Current Gene Therapy - June 6, 2017 Category: Genetics & Stem Cells Authors: Vaseghi G, Rafiee L, Javanmard SH Tags: Curr Gene Ther Source Type: research

siRNA delivery system based on magnetic nanovectors: Characterization and stability evaluation.
Abstract Gene therapy and particularly small interfering RNA (siRNA) is a promising therapeutic method for treatment of various human diseases, especially cancer. However the lack of an ideal delivery system limits its clinical applications. Effective anticancer drug development represents the key for translation of research advances into medicines. Previously we reported, the optimization of magnetic siRNA nanovectors (MSN) formulation based on superparamagnetic iron oxide nanoparticles (SPION) and chitosan for systemic administration. This work aimed at using rational design to further optimize and develop MSN. ...
Source: European Journal of Pharmaceutical Sciences - June 1, 2017 Category: Drugs & Pharmacology Authors: Abdelrahman M, Eyrolles LD, Alkarib SY, Hervé-Aubert K, Djemaa SB, Marchais H, Chourpa I, David S Tags: Eur J Pharm Sci Source Type: research

Mesoporous silica nanoparticles as a carrier platform for intracellular delivery of nucleic acids
AbstractVirus-mediated gene delivery has been, to date, the most successful and efficient method for gene therapy. However, this method has been challenged because of serious safety concerns. Over the past decade, mesoporous silica nanoparticles (MSNs) have attracted much attention for intracellular delivery of nucleic acids. Delivery of cellular plasmid DNA (pDNA) is designed to replace the function of a defective gene and restore its normal function in the cell. Delivery of small interfering RNAs (siRNAs) can selectively knockdown genes by targeting specific mRNAs. The biocompatibility and unique structures of MSNs make ...
Source: Biochemistry (Moscow) - June 1, 2017 Category: Biochemistry Source Type: research

Readthrough of SCN5A Nonsense Mutations R1623X and S1812X Question Gene-therapy in Brugada Syndrome.
CONCLUSIONS: These results demonstrated that readthrough-enhancing methods effectively suppressed nonsense mutations in SCN5A and restored the expression of full-length channels. But the restored channels may increase the risk of arrhythmia. The strategies for nonsense mutations suppression provides potential evidence for personalized medicine for the treatment of genetic disorders. PMID: 28552050 [PubMed - as supplied by publisher]
Source: Current Gene Therapy - May 28, 2017 Category: Genetics & Stem Cells Authors: Teng S, Huang J, Gao Z, Hao J, Yang Y, Zhang S, Pu J, Hui R, Wu Y, Fan Z Tags: Curr Gene Ther Source Type: research

Delivering siRNA with Dendrimers: In Vivo Applications.
Abstract Over the last decades, gene therapy has emerged as a pioneering therapeutic approach to treat or prevent several diseases. Among the explored strategies, the short-term silencing of protein coding genes mediated by siRNAs has a good therapeutic potential in a clinical setting. However, the widespread use of siRNA will require the development of clinically suitable, safe and effective vehicles with the ability to complex and deliver siRNA into target cells with minimal toxicity. Lately, dendrimers have gained considerable attention as non-viral vectors in nucleic acid delivery due to their unique structura...
Source: Current Gene Therapy - May 10, 2017 Category: Genetics & Stem Cells Authors: Leiro V, Santos SD, Pêgo AP Tags: Curr Gene Ther Source Type: research

Nucleic acids-based nanotherapeutics crossing the blood brain barrier.
Abstract The restless endeavors revealing the molecular pathways underlying many neurodegenerative diseases and brain tumors have paved the way for the introduction of the selective exogenous gene-based therapeutics. The implicated active biomolecules encompass mainly negatively-charged nucleic acids ranging from DNA, mRNA, non-coding RNAs (small-interfering RNA, siRNA, and microRNA, miRNA), to antisense oligonucleotides. They selectively interfere with the genes translational and/or transcriptional processes. Although many reviews previously addressed brain targeting, a thorough correlation between the molecular ...
Source: Current Gene Therapy - May 10, 2017 Category: Genetics & Stem Cells Authors: Nafee N, Gouda N Tags: Curr Gene Ther Source Type: research

Smart micro/nano-robotic systems for gene delivery.
Abstract Small scale robotics have attracted growing attention for the prospect of targeting and accessing cell-sized sites, necessary for high precision biomedical applications and drug/gene delivery. The loss of controlled gene therapy, inducing systemic side effects and reduced therapeutic efficiency, can be settled utilizing these intelligent carriers. Newly proposed solutions for the main challenges of control, power supplying, gene release and final carrier extraction/degradation have shifted these smart miniature robots to the point of being employed for practical applications of transferring oligonucleotid...
Source: Current Gene Therapy - May 10, 2017 Category: Genetics & Stem Cells Authors: Pedram A, Pishkenari HN Tags: Curr Gene Ther Source Type: research

Silencing of CXCR4 and CXCR7 expression by RNA interference suppresses human endometrial carcinoma growth in vivo.
Authors: Huang Y, Ye Y, Long P, Zhao S, Zhang L, A Y Abstract In this paper, the effect of silencing the expression of CXCR4 and CXCR7 by RNAi on the growth of endometrial carcinoma (EC), in vivo, was evaluated. To establish endometrial carcinoma model, thirty nude mice were subcutaneously inoculated with 1 × 10(7) Ishikawa cells. All tumor-bearing mice were randomly assigned to five groups (six mice in each group) when the tumor xenografts reached 5-7 mm in diameter, and treated with CXCR4-siRNA (5 nmol), CXCR7-siRNA (5 nmol), CXCR4-siRNA (5 nmol) plus CXCR7-siRNA (5 nmol), negative-siRNA (5 nmol) and normal sali...
Source: American Journal of Translational Research - May 6, 2017 Category: Research Tags: Am J Transl Res Source Type: research

532 siRNA based non-viral gene therapy for the treatment of epidermolysis bullosa simplex(EBS)
EBS is an inherited, skin fragility disorder predominantly caused by dominant-negative mutations in genes encoding for the cytoskeletal proteins, Keratin5 and Keratin14 within the basal cell layer. To date, EBS is incurable with only symptomatic therapies currently available. The downregulation of these mutant genes would provide an excellent curative therapy for treating EBS. siRNA therapeutics have been identified as an attractive therapy option for EBS given the highly accessible nature of skin tissue.
Source: Journal of Investigative Dermatology - April 12, 2017 Category: Dermatology Authors: J. O'Keeffe Ahern, D. Zhou, L. Cutlar, I. Lara-S áez, W. Wang Tags: Genetic Disease, Gene Regulation and Gene Therapy Source Type: research

482 Inhibition of cyclooxygenase-2 and prostaglandin E-PGE receptor 4 pathway restores ultraviolet B-induced ATP2A2/SERCA2 downregulation in keratinocytes
Background: ATP2A2 encoding the sarcoplasmic/endoplasmic reticulum Ca2+ -ATPase2 (SERCA2) is a responsible gene for Darier disease (DD). Ultraviolet (UV) B irradiation downregulates ATP2A2/SERCA2 expression in keratinocytes, whereas cyclooxygenase-2 (COX-2) expression is dramatically upregulated by UVB. Objectives: To analyze the involvement of COX-2 and subsequent Prostaglandin E (PGE) -PGE receptor signaling in ATP2A2/SERCA2 expression. Methods: Normal human keratinocytes (NHKs) were transfected with COX-2 siRNA, PGE receptor (EP1-4) siRNA and treated with COX-2 inhibitor, celecoxib, to evaluate the effect of COX-2 and P...
Source: Journal of Investigative Dermatology - April 12, 2017 Category: Dermatology Authors: M. Kaga-Kamijo, A. Wada, R. Mineki, T. Sakanishi, C. Nishiyama, S. Ikeda Tags: Genetic Disease, Gene Regulation and Gene Therapy Source Type: research

478 Mutation-specific siRNA Knockdown of GJB2 − Potential gene therapy for Keratitis-ichthyosis-deafness Syndrome
Mutations in the GJB2 gene, which encodes connexin 26 (Cx26), cause keratitis-ichthyosis-deafness (KID) syndrome. 87% of patients carry a dominant mutation D50N, which is thought to cause a dominant negative effect on the normal allele product. Current treatment is limited to symptomatic care. We hypothesised that a mutation-specific siRNA to selectively suppress the D50N mutant allele may potentially reverse the disease phenotype. We first tested whether suppression of a single Cx26 allele can lead to haplo-insufficiency in human keratinocytes (KCs).
Source: Journal of Investigative Dermatology - April 12, 2017 Category: Dermatology Authors: M. Lee, V.A. Kinsler, S.L. Hart, W. Di Tags: Genetic Disease, Gene Regulation and Gene Therapy Source Type: research

Effects of RNA interference combined with ultrasonic irradiation and SonoVue microbubbles on expression of STAT3 gene in keratinocytes of psoriatic lesions
SummaryThe most effective sequence of small interfering RNA (siRNA) silencing STAT3 of psoriatic keratinocytes (KCs) was screened out, and the effects of the most effective siRNA combined with ultrasonic irradiation and SonoVue microbubbles on the expression of STAT3 of KCs and the dose- and time-response were investigated. Three chemically-synthetic siRNAs targeting STAT3 carried by Lipofectamine 3000 were transfected into KCs, and the effects on STAT3 expression were detected, then the most effective siRNA was selected for the subsequent experiments. The negative controls of siRNA (siRNA-NC) labeled with Cy3 carried by L...
Source: Journal of Huazhong University of Science and Technology -- Medical Sciences -- - April 1, 2017 Category: Research Source Type: research

Genetic pharmacology: progresses in siRNA delivery and therapeutic applications
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Source: Gene Therapy - February 16, 2017 Category: Genetics & Stem Cells Authors: D Scherman A Rousseau P Bigey V Escriou Source Type: research