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Therapy: Gene Therapy

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Total 490 results found since Jan 2013.

Structural characterization of transfection nanosystems based on tricationic surfactants and short double stranded oligonucleotides.
We report the formation of stable complexes between 21 base pairs oligonucleotides - siRNA, enhancing DMPK gene, and dsDNA and two tricationic surfactants (1,2,3-propanetri[oxymethyl-3-(1-dodecylimidazolium)]chloride and 1,2,3-propanetri[(oxymethyl)dimethyldodecylammonium]chloride. Structural studies by SAXS and TEM have shown that the dominant structure of the obtained lipoplexes is based on hexagonal, lamellar and cubic phases, packed in highly ordered aggregates. It has been established that tricationic surfactants can be used as siRNA carriers in gene therapy. PMID: 31472953 [PubMed - as supplied by publisher]
Source: Biochemical and Biophysical Research communications - August 27, 2019 Category: Biochemistry Authors: Andrzejewska W, Wilkowska M, Peplińska B, Skrzypczak A, Kozak M Tags: Biochem Biophys Res Commun Source Type: research

Promising approaches of small interfering RNAs (siRNAs) mediated cancer gene therapy.
Abstract RNA interference (RNAi) has extensive potential to revolutionize every aspect of clinical application in biomedical research. One of the promising tools is the Small interfering RNA (siRNA) molecules within a cellular component. Principally, siRNA mediated innovative advances are increasing rapidly in support of cancer diagnosis and therapeutic purposes. Conversely, it has some delivery challenges to the site of action within the cells of a target organ, due to the progress of nucleic acids engineering and advance material science research contributing to the exceptional organ-specific targeted therapy. T...
Source: Gene - August 23, 2019 Category: Genetics & Stem Cells Authors: Senapati D, Patra BC, Kar A, Chini DS, Ghosh S, Patra S, Bhattacharya M Tags: Gene Source Type: research

Glycyrrhetinic acid-modified graphene oxide mediated siRNA delivery for enhanced liver-cancer targeting therapy.
Abstract Graphene oxide (GO) has attracted huge attention in biomedical field in recent years. However, limited attempts have been invested in utilizing GO on active targeted delivery for gene therapy in liver cancer treatments. Glycyrrhetinic acid (GA) has been reported to be widely used as a targeting ligand to functionalize nanomaterials to treat hepatocellular carcinoma. In this article, GA is employed as a liver targeting ligand to construct GA, polyethylene glycol (PEG), polyamidoamine dendrimer (Dendrimer) and nano-graphene oxide (NGO) conjugate (GA-PEG-NGO-Dendrimer, GPND) for siRNA delivery for the first ...
Source: European Journal of Pharmaceutical Sciences - August 21, 2019 Category: Drugs & Pharmacology Authors: Qu Y, Sun F, He F, Yu C, Lv J, Zhang Q, Liang D, Yu C, Wang J, Zhang X, Xu A, Wu J Tags: Eur J Pharm Sci Source Type: research

Lentivirus-mediated RNA interference targeting EBNA1 gene inhibits the growth of GT-38 cells in vitro and in vivo.
Authors: Wang J, Liang C, Meng F, Xu X, Wu Y, Lu L Abstract Epstein-Barr virus nuclear antigen 1 (EBNA1) is associated with the pathogenesis of Epstein-Barr virus-associated gastric carcinoma (EBVaGC). However, the function of EBNA1 in the growth of EBVaGC cells remains unclear. In the present study, the effects of silencing EBNA1, by RNA interference (RNAi), on the growth of EBVaGC cells were investigated in vitro and in vivo. A lentivirus-mediated RNAi targeting EBNA1 was transfected into the EBVaGC cell line GT-38. Reverse transcription-quantitative polymerase chain reaction (RT-qPCR), western blot analysis, MTT...
Source: Oncology Letters - August 14, 2019 Category: Cancer & Oncology Tags: Oncol Lett Source Type: research

Biodegradable Polyester of Poly (Ethylene glycol)-sebacic Acid as a Backbone for β-Cyclodextrin-polyrotaxane: A Promising Gene Silencing Vector.
CONCLUSION: The combinatory effect of various factors such as biodegradability, favourable complexation ability, near zero zeta potentials, good cytotoxicity properties of poly (ethylene glycol)-sebacic acid based β-Cyclodextrin-polyrotaxane makes it a potential gene delivery vector for therapeutic applications. PMID: 31393245 [PubMed - as supplied by publisher]
Source: Current Gene Therapy - August 6, 2019 Category: Genetics & Stem Cells Authors: Ghodke S, Mahajan P, Gupta K, Ver Avadhani C, Dandekar P, Jain R Tags: Curr Gene Ther Source Type: research

RNA-based Therapy for Osteogenesis
Publication date: Available online 5 August 2019Source: International Journal of PharmaceuticsAuthor(s): Pinpin Wang, Federico Perche, Delphine Logeart-Avramoglou, Chantal PichonAbstractNucleic acid-based therapy has shown great promise in accelerating bone regeneration as well as other diseases. Nucleic acids used in gene therapy mainly are either plasmid DNA (pDNA) or RNAs. Although pDNA therapy has been extensively studied for decades with encouraging preclinical and clinical results, side effects, and low efficiency associated with nuclear trafficking are hard to bypass. Unlike pDNA, RNAs (mRNA, siRNA, miRNA) exert the...
Source: International Journal of Pharmaceutics - August 6, 2019 Category: Drugs & Pharmacology Source Type: research

Combination of photodynamic therapy and gene silencing achieved through the hierarchical self-assembly of porphyrin-siRNA complexes
Publication date: Available online 31 July 2019Source: International Journal of PharmaceuticsAuthor(s): Nabila Laroui, Maëva Coste, Laure Lichon, Yannick Bessin, Magali Gary-Bobo, Geneviève Pratviel, Colin Bonduelle, Nadir Bettache, Sébastien UlrichAbstractIn this work, we implemented a supramolecular approach in order to combine photodynamic therapy (PDT) with gene therapy. We made use of a simple cationic guanidylated porphyrin (H2‑PG) with the hypothesis that porphyrin aggregates should be capable of complexing siRNA through multivalent interactions and thus contribute to its intracellular delivery, while remaining...
Source: International Journal of Pharmaceutics - August 1, 2019 Category: Drugs & Pharmacology Source Type: research

Akt1 and Jak1 siRNA based silencing effects on the proliferation and apoptosis in head and neck squamous cell carcinoma.
In conclusion, the combination of siRNA-mediated gene-silencing strategy can be considered as a valuable and safe approach for sensitizing cancer cells to chemotherapeutic agents thus proposed further studies regarding this issue to approve some siRNA based therapeutics for using in clinic. PMID: 31348981 [PubMed - as supplied by publisher]
Source: Gene - July 22, 2019 Category: Genetics & Stem Cells Authors: Saatloo MV, Aghbali AA, Koohsoltani M, Khosroushahi AY Tags: Gene Source Type: research

Supramolecular Assemblies of Peptides or Nucleopeptides for Gene Delivery
Using non-covalent interactions between nucleic acids (DNA, siRNA, miRNA, and mRNA) with peptides or nucleopeptides is a promising strategy to construct supramolecular assemblies for gene delivery and therapy. Comparing to conventional strategies for gene delivery, the assemblies of peptides or nucleopeptides provide several unique advantages: i) reversible interactions between the assemblies and the nucleic acids; ii) minimal immunogenicity; iii) biocompatibility. This field has advanced considerably in recent years so that it is worth summarizing the recent progresses and future challenges. In this review, we introduce t...
Source: Theranostics - June 13, 2019 Category: Molecular Biology Authors: Huaimin Wang, Zhaoqianqi Feng, Bing Xu Tags: Review Source Type: research

Mutation-Targeted siRNA Therapy for Connexin 26-Associated Keratitis-Ichthyosis-Deafness Syndrome
ConclusionTo that end, a KID syndrome cell line (KID-KC) was established from primary keratinocytes of a KID syndrome patient with heterozygous p.D50N mutation, which displayed impaired gap junction intercellular communication and hyperactive hemichannels, confirmed by dye transfer, patch clamp and neurobiotin uptake assays. In KID-KCs, treatment with AS-siRNA led to robust inhibition of the mutant GJB2 allele without altering expression of the wildtype allele. This resulted in correction of both gap junction intercellular communication and hemichannel activity. Furthermore, AS-siRNA treatment caused only low-level off-tar...
Source: Cytotherapy - May 24, 2019 Category: Cytology Source Type: research

Engineered targeting tLyp-1 exosomes as gene therapy vectors for efficient delivery of siRNA into lung cancer cells
In conclusion, the targeting tLyp-1 exosomes are successfully engineered, and can be used for gene therapy with a high transfection efficiency. Therefore, the engineered targeting tLyp-1 exosomes offer a promising gene delivery platform for future cancer therapy.Graphical AbstractThe tLyp-1-lamp2b plasmid transfected HEK293T cells can secreted tumor targeting tLyp-1 exosomes. By electroporation technology, targeting tLyp-1 exosomes were loaded with siRNA. When targeting tLyp-1 exosome ruptured in cytoplasm, siRNA was loaded into the RNA-induced silencing complex (RISC). The sense (passenger) strand was degraded while the a...
Source: Asian Journal of Pharmaceutical Sciences - May 8, 2019 Category: Drugs & Pharmacology Source Type: research

Sprouty2 —a Novel Therapeutic Target in the Nervous System?
AbstractClinical trials applying growth factors to alleviate symptoms of patients with neurological disorders have largely been unsuccessful in the past. As an alternative approach, growth factor receptors or components of their signal transduction machinery may be targeted directly. In recent years, the search for intracellular signaling integrator downstream of receptor tyrosine kinases provided valuable novel substrates. Among them are the Sprouty proteins which mainly act as inhibitors of growth factor-dependent neuronal and glial signaling pathways. In this review, we summarize the role of Sprouties in the lesioned ce...
Source: Molecular Neurobiology - May 7, 2019 Category: Neurology 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

Gene Therapy Leaves a Vicious Cycle
Reena Goswami1, Gayatri Subramanian2, Liliya Silayeva1, Isabelle Newkirk1, Deborah Doctor1, Karan Chawla2, Saurabh Chattopadhyay2, Dhyan Chandra3, Nageswararao Chilukuri1 and Venkaiah Betapudi1,4* 1Neuroscience Branch, Research Division, United States Army Medical Research Institute of Chemical Defense, Aberdeen, MD, United States 2Department of Medical Microbiology and Immunology, University of Toledo College of Medicine and Life Sciences, Toledo, OH, United States 3Roswell Park Comprehensive Cancer Center, Buffalo, NY, United States 4Department of Physiology and Biophysics, Case Western Reserve University, Clev...
Source: Frontiers in Oncology - April 23, 2019 Category: Cancer & Oncology Source Type: research

375 The chromatin architectural protein CTCF regulates expression of pro-inflammatory cytokines in the skin cells
Increased evidence suggests that alterations in chromatin structure result in aberrant pro-inflammatory cytokines expression that leads to prolonged inflammatory responses. CCCTC-binding factor (CTCF) is a transcriptional repressor that insulates the expression of neighbouring genes and is involved in chromatin interactions between distal and proximal gene regulatory elements. However, the role of CTCF in the control of pro-inflammatory cytokines expression in the skin cells remains unknown. Here, we show that CTCF is expressed in the nuclei of normal human epidermal keratinocytes (NHEK) and dermal fibroblasts (DF), while ...
Source: Journal of Investigative Dermatology - April 19, 2019 Category: Dermatology Authors: M. Boboljova, I. Asamaowei, V. Botchkarev, M. Fessing, A.N. Mardaryev Tags: Genetic Disease, Gene Regulation, and Gene Therapy Source Type: research