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Therapy: Gene Therapy

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Total 490 results found since Jan 2013.

A View on the Importance of “Multi-Attribute Method” for Measuring Purity of Biopharmaceuticals and Improving Overall Control Strategy
AbstractToday, we are experiencing unprecedented growth and innovation within the pharmaceutical industry. Established protein therapeutic modalities, such as recombinant human proteins, monoclonal antibodies (mAbs), and fusion proteins, are being used to treat previously unmet medical needs. Novel therapies such as bispecific T cell engagers (BiTEs), chimeric antigen T cell receptors (CARTs), siRNA, and gene therapies are paving the path towards increasingly personalized medicine. This advancement of new indications and therapeutic modalities is paralleled by development of new analytical technologies and methods that pro...
Source: The AAPS Journal - November 30, 2017 Category: Drugs & Pharmacology Source Type: research

Co ‑expression of murine double minute 2 siRNA and wild‑type p53 induces G1 cell cycle arrest in H1299 cells.
Co‑expression of murine double minute 2 siRNA and wild‑type p53 induces G1 cell cycle arrest in H1299 cells. Mol Med Rep. 2017 Oct 11;: Authors: Liu L, Zhang P, Guo H, Tang X, Liu L, Li J, Guo R, Cai Y, Liu Y, Li Y Abstract The therapeutic options available for the treatment of advanced non-small cell lung cancer have increased over the past decade. Small molecule gene therapy has emerged as an effective therapy for the treatment of lung cancer in vitro and in vivo although it has not been tested in a clinical setting. In particular, therapies that target the negative feedback loop between p53 an...
Source: Molecular Medicine Reports - October 20, 2017 Category: Molecular Biology Tags: Mol Med Rep Source Type: research

Amphiphilic small peptides for delivery of plasmid DNAs and siRNAs
This article is protected by copyright. All rights reserved. As novel nucleic acid carriers, linear and branched amphiphilic peptides were prepared. Each peptide was compared in terms of SNALP size, stability, and delivery activities for plasmid DNA (pDNA) and siRNA. Among the five peptides, a linear peptide H‐RCL was an effective delivery carrier for both siRNA and pDNA. Introduction of a benzoyl group to H‐RCL significantly enhanced siRNA delivery, but greatly reduced pDNA delivery. These demonstrated that a small structural modification of the peptides can modulate their delivery properties.
Source: Chemical Biology and Drug Design - October 20, 2017 Category: Biology Authors: Eun ‐Kyoung Bang, Hanna Cho, Sean S‐H Jeon, Na Ly Tran, Dong‐Kwon Lim, Wooyoung Hur, Taebo Sim Tags: Research Article Source Type: research

Effect of inhibiting Beclin-1 expression on autophagy, proliferation and apoptosis in colorectal cancer.
In conclusion, Beclin-1 played an important role in regulating autophagy, proliferation and apoptosis in HCT116 and SW620 cells. The inhibition of Beclin-1 by RNAi suppressed the autophagic activity and proliferation, but promoted apoptosis in CRC cells. Beclin-1 was the new target of gene therapy for CRC. PMID: 28989537 [PubMed]
Source: Oncology Letters - October 12, 2017 Category: Cancer & Oncology Tags: Oncol Lett Source Type: research

Functional analysis of ESM1 by siRNA knockdown in primary and metastatic head and neck cancer cells
ConclusionThese findings suggest that ESM1 has roles on proliferation and migration of head and neck cancer cells.This article is protected by copyright. All rights reserved.
Source: Journal of Oral Pathology and Medicine - October 11, 2017 Category: Pathology Authors: Onur Bender, Mehmet Gunduz, Sadik Cigdem, Omer Faruk Hatipoglu, Muradiye Acar, Mesut Kaya, Reidar Grenman, Esra Gunduz, Kadriye Serife Ugur Tags: Original Article Source Type: research

Targeting GRP78 as a Basis for Enhanced Glioma Cells Killing by anti-TfR antibody.
CONCLUSION: Our results show that the anti-tumor effects of anti-TfR antibody trigger the ER stress response, and that this effect is achieved via the siRNA against GRP78 for glioma cell growth and survival. These results hold promise as a clinical approach to gene therapy for malignant gliomas. PMID: 29091247 [PubMed - as supplied by publisher]
Source: Turkish Neurosurgery - October 2, 2017 Category: Neurosurgery Authors: Wen X, Cheng X, Wu S Tags: Turk Neurosurg Source Type: research

BTBD7 silencing inhibited epithelial- mesenchymal transition (EMT) via regulating Slug expression in human salivary adenoid cystic carcinoma.
CONCLUSIONS: BTBD7 silencing inhibited EMT through regulation of Slug expression in SACC-LM cells and might act as a potential molecular target for gene therapy of SACC. PMID: 28946551 [PubMed - as supplied by publisher]
Source: Cancer Biomarkers - September 29, 2017 Category: Cancer & Oncology Tags: Cancer Biomark Source Type: research

A new combination therapy for asthma using dual-function dexamethasone-conjugated polyethylenimine and vitamin D binding protein siRNA
T Rhim
Source: Gene Therapy - September 21, 2017 Category: Genetics & Stem Cells Authors: M Choi J Gu M Lee T Rhim Source Type: research

SiRNA Delivery with PEGylated Graphene Oxide Nanosheets for Combined Photothermal and Genetherapy for Pancreatic Cancer
In this study, we utilized multi-functionalized monolayer GO as a gene delivery system to efficiently co-deliver HDAC1 and K-Ras siRNAs (small interfering RNAs targeting the HDAC1 gene and the G12C mutant K-Ras gene, respectively) to specifically target pancreatic cancer cells MIA PaCa-2. The systematic mechanistic elucidation of the dual gene silencing effects indicated the inactivation of both the HDAC1 and the K-Ras gene, thereby causing apoptosis, proliferation inhibition and cell cycle arrest in treated MIA PaCa-2 cells. The synergistic combination of gene silencing and NIR light thermotherapy showed significant antic...
Source: Theranostics - September 12, 2017 Category: Molecular Biology Authors: Feng Yin, Kuan Hu, Yangzi Chen, Mengying Yu, Dongyuan Wang, Qianqian Wang, Ken-Tye Yong, Fei Lu, Yongye Liang, Zigang Li Tags: Research Paper Source Type: research

A new combination therapy for asthma using dual function dexamethsone-conjugated polyethylenimine and vitamin D binding protein siRNA
Gene Therapy, Published online: 28 August 2017; doi:10.1038/gt.2017.83
Source: Gene Therapy - August 28, 2017 Category: Genetics & Stem Cells Authors: M Choi J Gu M Lee T Rhim Source Type: research

Downregulation of BZW2 inhibits osteosarcoma cell growth by inactivating the Akt/mTOR signaling pathway.
Authors: Cheng DD, Li SJ, Zhu B, Yuan T, Yang QC, Fan CY Abstract Osteosarcoma is the most common malignant bone tumor in adolescents. The function of basic leucine zipper and W2 domains 2 (BZW2) in tumor progression has been reported. However, the role and mechanisms of BZW2 in osteosarcoma remain to be determined. The aim of the present study was to reveal the expression and biological functions of BZW2 in osteosarcoma and to elucidate the proximal mechanisms underlying these functions. The expression of BZW2 in osteosarcoma tissues and cell lines was assessed by qRT-PCR, western blotting and immunohistochemistr...
Source: Oncology Reports - August 11, 2017 Category: Cancer & Oncology Tags: Oncol Rep Source Type: research

Modulating circulating sFlt1 in an animal model of preeclampsia using PAMAM nanoparticles for siRNA delivery
Excessive circulating sFlt1 plays a major role in the pathogenesis of preeclampsia (PE). Using RNAi to silence sFlt1 may be a therapy for treating PE. Because of the rapid degradation of siRNA, gene therapy in vivo remains limited. Poly-amidoamine (PAMAM) has been demonstrated to be an excellent nanocarrier for siRNA delivery with no discernible toxicity.
Source: Placenta - August 1, 2017 Category: Reproduction Medicine Authors: Jun Yu, Jing Jia, Xijiao Guo, Ruibao Chen, Ling Feng 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

Ly6Clo monocytes drive immunosuppression and confer resistance to anti-VEGFR2 cancer therapy
Current anti-VEGF therapies for colorectal cancer (CRC) provide limited survival benefit, as tumors rapidly develop resistance to these agents. Here, we have uncovered an immunosuppressive role for nonclassical Ly6Clo monocytes that mediates resistance to anti-VEGFR2 treatment. We found that the chemokine CX3CL1 was upregulated in both human and murine tumors following VEGF signaling blockade, resulting in recruitment of CX3CR1+Ly6Clo monocytes into the tumor. We also found that treatment with VEGFA reduced expression of CX3CL1 in endothelial cells in vitro. Intravital microscopy revealed that CX3CR1 is critical for Ly6Clo...
Source: Journal of Clinical Investigation - July 10, 2017 Category: Biomedical Science Authors: Keehoon Jung, Takahiro Heishi, Omar F. Khan, Piotr S. Kowalski, Joao Incio, Nuh N. Rahbari, Euiheon Chung, Jeffrey W. Clark, Christopher G. Willett, Andrew D. Luster, Seok Hyun Yun, Robert Langer, Daniel G. Anderson, Timothy P. Padera, Rakesh K. Jain, Dai 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