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Recent advances in gene therapy: genetic bullets to the root of the problem
AbstractGenetics and molecular genetic techniques have changed many perspectives and paradigms in medicine. Using genetic methods, many diseases have been cured or alleviated. Gene therapy, in its simplest definition, is application of genetic materials and related techniques to treat various human diseases. Evaluation of the trends in the field of medicine and therapeutics clarifies that gene therapy has attracted a lot of attention due to its powerful potential to treat a number of diseases. There are various genetic materials that can be used in gene therapy such as DNA, single- and double-stranded RNA, siRNA and shRNA....
Source: Clinical and Experimental Medicine - October 25, 2022 Category: Research Source Type: research

Regulation of The Methylation and Expression Levels of the BMPR2 Gene by SIN3a As A Novel Therapeutic Mechanism in Pulmonary Arterial Hypertension
Conclusions: Altogether, our study unveiled the protective/beneficial role of SIN3a in pulmonary hypertension. We also identified a novel and distinct molecular mechanism by which SIN3a regulates BMPR2 in hPASMC. Our study also identified lung-targeted SIN3a gene therapy using AAV1 as a new promising therapeutic strategy for treating patients with PAH.PMID:34078089 | DOI:10.1161/CIRCULATIONAHA.120.047978
Source: Circulation - June 3, 2021 Category: Cardiology Authors: Malik Bisserier Prabhu Mathiyalagan Shihong Zhang Firas Elmastour Peter Dorfm üller Marc Humbert Gregory David Sima Tarzami Thomas Weber Fr édéric Perros Yassine Sassi Susmita Sahoo Lahouaria Hadri 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

Silencing of Phospholipase C gamma 2 Promotes Proliferation of Rat Hepatocytes In Vitro
In conclusion, these findings suggest that interference with PLCγ2 expression could relieve the inhibitory effect of PLCγ2 on hepaocyte apoptosis, thus promote proliferation through inactivating PKCδ‐mediated JNK1/2 signaling pathway. This article is protected by copyright. All rights reserved
Source: Journal of Cellular Biochemistry - December 13, 2017 Category: Biochemistry Authors: Xiaoguang Chen, Xuemin Zhu, Yumei Liu, Qiongxia Lv, Jun Ma Tags: Article Source Type: research

Efficient Gene Suppression in Dorsal Root Ganglia and Spinal Cord Using Adeno-Associated Virus Vectors Encoding Short-Hairpin RNA.
Abstract RNA interference is a powerful tool used to induce loss-of-function phenotypes through post-transcriptional gene silencing. Small interfering RNA (siRNA) molecules have been used to target the central nervous system (CNS) and are expected to have clinical utility against refractory neurodegenerative diseases. However, siRNA is characterized by low transduction efficiency, insufficient inhibition of gene expression, and short duration of therapeutic effects, and is thus not ideal for treatment of neural tissues and diseases. To address these problems, viral delivery of short-hairpin RNA (shRNA) expression ...
Source: Herpes - October 18, 2015 Category: Infectious Diseases Authors: Enomoto M, Hirai T, Kaburagi H, Yokota T Tags: Methods Mol Biol Source Type: research

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

Adeno-associated virus type 8 vector-mediated expression of siRNA targeting vascular endothelial growth factor efficiently inhibits neovascularization in a murine choroidal neovascularization model.
CONCLUSIONS: These results suggest that siRNA-VEGF can be expressed across the retina and that long-term suppression of CNV is possible through the use of stable AAV2/8-mediated siRNA-VEGF expression. In vivo gene therapy may thus be a feasible approach to the clinical management of CNV in conditions such as age-related macular degeneration. PMID: 24744609 [PubMed - indexed for MEDLINE]
Source: Molecular Vision - November 16, 2014 Category: Molecular Biology Tags: Mol Vis Source Type: research

Antitumor activities of an oncolytic adenovirus equipped with a double siRNA targeting Ki67 and hTERT in renal cancer cells.
In this study, we constructed novel oncolytic adenoviruses in which the Ki67 core promoter drove expression of the E1A gene. These adenoviruses were equipped with either a Ki67 small interfering RNA (siRNA), a human telomerase reverse transcriptase (hTERT) siRNA or a double siRNA targeting Ki67 and hTERT. We identified the antitumor activities of oncolytic adenoviruses in 3 renal cancer cell lines, human normal renal tube cell HK-2 and also in nude mice bearing KETR-3-xenografted tumors. Our results showed that these oncolytic adenoviruses, especially Ki67-ZXC2-double siRNA, could effectively induce silencing of the Ki67 a...
Source: Virus Research - January 21, 2014 Category: Virology Authors: Fang L, Cheng Q, Li W, Liu J, Li L, Xu K, Zheng J Tags: Virus Res Source Type: research

Suppression of persistent atrial fibrillation by genetic knockdown of caspase 3: a pre-clinical pilot study
Conclusion Knockdown of caspase 3 by atrial Ad-siRNA-Cas3 gene transfer suppresses or delays the onset of persistent AF by reduction in apoptosis and prevention of intra-atrial conduction delay in a porcine model. These results highlight the significance of apoptosis in the pathophysiology of AF and demonstrate short-term efficacy of gene therapy for suppression of AF.
Source: European Heart Journal - January 6, 2013 Category: Cardiology Authors: Trappe, K., Thomas, D., Bikou, O., Kelemen, K., Lugenbiel, P., Voss, F., Becker, R., Katus, H. A., Bauer, A. Tags: BASIC SCIENCE Source Type: research