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Therapy: Gene Therapy

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Total 490 results found since Jan 2013.

Gene therapy for colorectal cancer by adenovirus-mediated siRNA targeting CD147 based on loss of the IGF2 imprinting system.
Abstract Colorectal cancer (CRC) is one of the most common malignant tumors worldwide. Loss of imprinting (LOI) of the insulin-like growth factor 2 (IGF2) gene is an epigenetic abnormality phenomenon in CRC. Recently observed association of CRC with cluster of differentiation 147 (CD147) could provide a novel approach for gene therapy. In the present study, we investigated the feasibility of using adenovirus‑mediated siRNA targeting CD147 based on the IGF2 LOI system for targeted gene therapy of CRC. A novel adenovirus-mediated siRNA targeting CD147, rAd-H19-CD147mirsh, which was driven by the IGF2 imprintin...
Source: International Journal of Oncology - September 23, 2015 Category: Cancer & Oncology Authors: Pan Y, He B, Chen J, Sun H, Deng Q, Wang F, Ying H, Liu X, Lin K, Peng H, Xie H, Wang S Tags: Int J Oncol Source Type: research

siRNA Delivery by Stimuli-Sensitive Nanocarriers.
Abstract Since its discovery in the late 1990, small interfering RNA (siRNA) have quickly crept into the biopharmaceutical research as a new and powerful tool for the treatment of different human diseases based on altered gene-expression. Despite promising data from many pre-clinical studies, concrete hurdles still need to be overcome to bring therapeutic siRNAs in clinic. The design of stimuli-sensitive nanopreparations for gene therapy is a lively area of the current research. Compared to conventional systems for siRNA delivery, this type of platform can respond to local stimuli that are characteristics of the p...
Source: Current Pharmaceutical Design - October 23, 2015 Category: Drugs & Pharmacology Authors: Salzano G, Costa DF, Torchilin VP Tags: Curr Pharm Des Source Type: research

Correction of mutant p63 in EEC syndrome using siRNA mediated allele specific silencing restores defective stem cell function
This study demonstrates the phenotypic correction of mutant stem cells (OMESCs) in EEC syndrome by means of siRNA mediated allele‐specific silencing with restoration of function. The application of siRNA, alone or in combination with cell‐based therapies, offers a therapeutic strategy for corneal blindness in EEC syndrome. This article is protected by copyright. All rights reserved.
Source: Stem Cells - February 17, 2016 Category: Stem Cells Authors: Vanessa Barbaro, Annamaria A. Nasti, Claudia Del Vecchio, Stefano Ferrari, Angelo Migliorati, Paolo Raffa, Vincenzo Lariccia, Patrizia Nespeca, Mariangela Biasolo, Colin E. Willoughby, Diego Ponzin, Giorgio Palù, Cristina Parolin, Enzo Di Iorio Tags: Regenerative Medicine Source Type: research

Correction of Mutant p63 in EEC Syndrome Using siRNA Mediated Allele‐Specific Silencing Restores Defective Stem Cell Function
This study demonstrates the phenotypic correction of mutant stem cells (OMESCs) in EEC syndrome by means of siRNA mediated AS silencing with restoration of function. The application of siRNA, alone or in combination with cell‐based therapies, offers a therapeutic strategy for corneal blindness in EEC syndrome. Stem Cells 2016; 00:000—000
Source: Stem Cells - March 16, 2016 Category: Stem Cells Authors: Vanessa Barbaro, Annamaria A. Nasti, Claudia Vecchio, Stefano Ferrari, Angelo Migliorati, Paolo Raffa, Vincenzo Lariccia, Patrizia Nespeca, Mariangela Biasolo, Colin E. Willoughby, Diego Ponzin, Giorgio Palù, Cristina Parolin, Enzo Iorio Tags: Regenerative Medicine Source Type: research

Enhancing radiosensitivity of TE1, TE8, and TE 11 esophageal squamous carcinoma cell lines by Hdm2-siRNA targeted gene therapy in vitro.
CONCLUSION: Increasing radiosensitivity of tumor cells may be provided by silencing the oncogenes. PMID: 27525226 [PubMed]
Source: BioImpacts - August 16, 2016 Category: Research Tags: Bioimpacts Source Type: research

BACH1 silencing by siRNA inhibits migration of HT-29 colon cancer cells through reduction of metastasis-related genes.
CONCLUSION: Our results indicated that BACH1 down-regulation in HT29 CRC cells had no effect on cell growth but did inhibit cell migration by decreasing metastasis-related genes expression. Collectively, these results suggest that BACH1 may function as an oncogenic driver in colon cancer and may represent as a potential target of gene therapy for CRC treatment. PMID: 27657827 [PubMed - as supplied by publisher]
Source: Biomedicine and pharmacotherapy = Biomedecine and pharmacotherapie - September 18, 2016 Category: Drugs & Pharmacology Authors: Davudian S, Shajari N, Kazemi T, Mansoori B, Salehi S, Mohammadi A, Shanehbandi D, Shahgoli VK, Asadi M, Baradaran B Tags: Biomed Pharmacother 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

A Combination of Guanidyl and Phenyl Groups on Dendrimer Enables Efficient siRNA and DNA Delivery.
This study provides a new strategy to develop multifunctional polymers for efficient siRNA and DNA delivery. PMID: 28686016 [PubMed - as supplied by publisher]
Source: Biomacromolecules - July 7, 2017 Category: Biochemistry Authors: Chang H, Zhang J, Wang H, Lv J, Cheng Y Tags: Biomacromolecules Source Type: research

Amelioration of cirrhotic portal hypertension by targeted cyclooxygenase-1 siRNA delivery to liver sinusoidal endothelium with polyethylenimine grafted hyaluronic acid
In this study, hyaluronate-graft-polyethylenimine (HA-PEI) was synthesized for the targeted delivery of COX-1 siRNA to LSECs. Compared to non-targeted PEI, HA-PEI mediated much more efficient siRNA delivery, which resulted in potent targeted gene silencing in LSECs. In vivo, HA-PEI notably increased the accumulation of siRNA along the sinusoidal lining of the liver, inhibited over-activation of the COX-1/TXA2 pathway in LSECs, and successfully reduced portal pressure in cirrhotic mice. These results highlight the potential of HA-PEI complexed siRNA to serve as a LSECs-specific nanomedical system for effective gene therapy ...
Source: Nanomedicine: Nanotechnology, Biology and Medicine - July 13, 2017 Category: Nanotechnology Source Type: research

A black phosphorus nanosheet-based siRNA delivery system for synergistic photothermal and gene therapy.
Abstract Gene therapy with small interfering RNA (siRNA) has been proved to be a promising technology to treat various diseases by hampering the production of target proteins. However, developing a delivery system that has high efficiency in transporting siRNA without obvious side effects remains a challenge. Herein, we designed a new survivin siRNA delivery system based on polyethyleneimine functionalized black phosphorus (BP) nanosheets which could suppress tumor growth by silencing survivin expression. Combined with the photothermal properties of the BP nanosheets, the presented delivery system shows excellent ...
Source: Chemical Communications - March 12, 2018 Category: Chemistry Authors: Wang H, Zhong L, Liu Y, Xu X, Xing C, Wang M, Bai SM, Lu CH, Yang HH Tags: Chem Commun (Camb) Source Type: research

AKT2 siRNA delivery with amphiphilic-based polymeric micelles show efficacy against cancer stem cells.
Authors: Rafael D, Gener P, Andrade F, Seras-Franzoso J, Montero S, Fernández Y, Hidalgo M, Arango D, Sayós J, Florindo HF, Abasolo I, Schwartz S, Videira M Abstract Development of RNA interference-based therapies with appropriate therapeutic window remains a challenge for advanced cancers. Because cancer stem cells (CSC) are responsible of sustaining the metastatic spread of the disease to distal organs and the progressive gain of resistance of advanced cancers, new anticancer therapies should be validated specifically for this subpopulation of cells. A new amphihilic-based gene delivery system that combines Plu...
Source: Drug Delivery - April 20, 2018 Category: Drugs & Pharmacology Tags: Drug Deliv Source Type: research

Lipid-based nanocarriers for siRNA delivery: challenges, strategies and the lessons learned from the DODAX:MO liposomal system.
Abstract The possibility of using the RNA interference (RNAi) mechanisms in gene therapy was one of the scientific breakthroughs of the last century. Despite the extraordinary therapeutic potential of this approach, the need for an efficient gene carrier is hampering the translation of the RNAi technology to the clinical setting. Although a diversity of nanocarriers have been described, liposomes continue to be one of the most attractive siRNA vehicles due to their relatively low toxicity, facilitated siRNA complexation, high transfection efficiency and enhanced pharmacokinetic properties. This review focuses on R...
Source: Current Drug Targets - July 3, 2018 Category: Drugs & Pharmacology Authors: Oliveira ACN, Fernandes J, Goncalves A, Gomes AC, Oliveira R Tags: Curr Drug Targets Source Type: research

Lipid-based siRNA Nanodelivery Systems: a Learning Process for Improving Transfer from Concepts to Clinical Applications.
CONCLUSION: Formulation design should be increasingly addressed with industrial criteria; it should be based on quality by design and on the estimation of critical attributes that affect performance, and supported by a range of characterization techniques and appropriate analytical methods. PMID: 30160217 [PubMed - as supplied by publisher]
Source: Current Clinical Pharmacology - August 29, 2018 Category: Drugs & Pharmacology Authors: Perez SE, Carlucci AM Tags: Curr Clin Pharmacol 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

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