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Current Gene Therapy

Gilar Gorji-Bahri, Atieh Hashemi, Hamid Reza Moghimi
Exosomes play a critical role in intercellular communication between cancer cells and their environments. These secreted nanovesicles can transfer different cargos such as mRNAs, proteins and microRNA (miRNA) to recipient cells. Exosomal miRNAs (exomiRs) derived from tumor cells have emerged as key players in cancer promotion via impairment of the immune system response, tumor growth, metastasis, angiogenesis, and chemotherapeutic drug resistance. Moreover, since dysregulation of miRNA expression in tumor cells can be reflected by distinct profiles of exomiRs extracted from the bodily fluids of cancer patients, they can be considered as non-invasive diagnostic, prognostic and predictive biomarkers...
October 17, 2018: Current Gene Therapy
Liang Cheng, Yang Hu
In recent years, genome-wide association studies (GWAS) and next-generation sequencing technologies have been widely used to detect the common genetic variants of diseases. Despite these successes, the majority of genetic architecture of human complex diseases remains unknown. In the post-genome era, the major challenge is to mine novel disease risks from multi-level omics data using system biology methods, which may expand our knowledge of the causes of genetic disease.
October 9, 2018: Current Gene Therapy
Meng Li, Mei-Song Lu, Mei-Ling Liu, Suo Deng, Xiao-Han Tang, Cui Han, Hong-Li Wang, Pei-Ling Li
Autophagy exists widely in various physiological and pathological conditions. Lots of investigations have verified that the autophagic activity is always related to the occurrence and the development of cancer. Endometriosis (EMs) is a disease that endometrium-like tissues abnormally grow outside the uterus and also considered to possess the characters of tumor because of its malignant biological behavior. Recently, several studies have already revealed that autophagy may play a potential role in proliferative-phase EMs...
October 8, 2018: Current Gene Therapy
Min Li, Jun Zhang, Bo Wu, Zhongliang Zhou, Yongdong Xu
In microbial communities, the keystone species have a greater impact on the performance and dynamics of ecosystem than that of other species, in which we can see from the results losing gut microbiome causes some specific diseases. A mass of ongoing studies aim at identifying links between microbial community structure and human diseases. In this paper, we are introducing a valid keystone species identification method, in which a new Spread Intensity (SI) algorithm is used. Because the accuracies of current keystone species identification algorithms are difficult to evaluate for the high diversity and uncultivated status of microbial communities, we simulated cross-sectional data of microbial communities with known interactions and set up standard keystoneness rankings using Generalized Lotka-Volterra(GLV) model...
October 8, 2018: Current Gene Therapy
Yan Wang, Yinping Xie, Lili Li, Yuan He, Di Zheng, Pengcheng Yu, Ling Yu, Lixu Tang, Yibin Wang, Zhihua Wang
Polycomb repressive complex 2 (PRC2) catalyzes histone methylation at H3 Lys27, and plays crucial roles during development and diseases in numerous systems. Its catalytic subunit EZH2 represents a key nuclear target for long non-coding RNAs (lncRNAs) that emerging to be a novel class of epigenetic regulator and participate in diverse cellular processes. LncRNAs are characterized by high tissue-specificity; however, little is known about the tissue profile of the EZH2-interacting lncRNAs. Here we performed a global screening for EZH2-binding lncRNAs in tissues including brain, lung, heart, liver, kidney, intestine, spleen, testis, muscle and blood by combining RNA immuno-precipitation and RNA sequencing...
October 8, 2018: Current Gene Therapy
Botao Yu, Changjing Wu, Tao Li, Feng Qin, Jiuhong Yuan
Erectile dysfunction (ED) is a major health problem in men. Over the past few decades, oral phosphodiesterase type 5 (PDE5) inhibitors have been adopted as the recommended strategy to treat ED in the majority of cases. However, these oral medications require on-demand access and are not effective in some hard-to-treat populations. Other alternative treatments are also not satisfactory. In recent years, a novel therapeutic approach against ED, gene therapy, has shown great potential in preclinical models designed to provide long-term improvement of erectile function and the underlying conditions...
October 4, 2018: Current Gene Therapy
Cen Yan, Xiao-Jiang Quan, Ying-Mei Feng
Myocardial infarction (MI) is the most severe ischemic heart disease and directly leads to heart failure till death. Target molecules have been identified in the event of MI including increasing angiogenesis, promoting cardiomyocyte survival, improving heart function and restraining inflammation and myocyte activation and subsequent fibrosis. All of which are substantial in cardiomyocyte protection and preservation of cardiac function. To modulate target molecule expression, virus and non-virus-mediated gene transfer have been investigated...
October 3, 2018: Current Gene Therapy
Chaitra Venugopal, K Shobha, Kiranmai S Rai, Venkata Bharatkumar Pinnelli, Bindu M Kutty, Anandh Dhanushkodi
Mesenchymal stem cell (MSC) therapy in recent years have gained significant attention. Though the functional outcomes following MSC therapy for neurodegenerative diseases are convincing, various mechanisms for the functional recovery are being debated. Nevertheless, recent studies convincingly demonstrated that recovery following MSC therapy could be reiterated with MSC secretome per se thereby shifting the dogma from cell therapy to cell "based" therapy. In addition to various functional proteins, stem cell secretome also includes extracellular membrane vesicles like exosomes...
September 13, 2018: Current Gene Therapy
Lingling Zhao, Junjie Wang, Mahieddine Mohammed Nabil, Jun Zhang
MOTIVATION: Knowledge of the correct protein subcellular localization is necessary for understanding the function of a protein and revealing the mechanism of many human diseases due to protein subcellular mislocalization, which is required before approaching gene therapy to treat a disease. In addition, it is well-known that the gene therapy is an effective way to overcome disease by targeting a gene therapy product to a specific subcellular compartment.. Deep neural networks to predict protein function have become increasingly popular due to large increases in available genomics data due to its strong superiority in the non-linear classify ability...
September 12, 2018: Current Gene Therapy
Hong-Yan Lai, Chao-Qin Feng, Zhao-Yue Zhang, Hua Tang, Wei Chen, Hao Lin
Proteins with at least one carbohydrate recognition domain are lectins that can identify and reversibly interact with glycan moiety of glycoconjugates or a soluble carbohydrate. It has been proved that lectins can play various vital roles in mediating signal transduction, cell-cell recognition and interaction, immune defense, and so on. Most organisms can synthesize and secret lectins. A portion of lectins closely related to diverse cancers, called cancerlectins, are involved in tumor initiation, growth and recrudescence...
September 12, 2018: Current Gene Therapy
Samia M O'Bryan, J Michael Mathis
Breast cancer continues to be a leading cause of mortality among women. While at an early stage, localized breast cancer is easily treated; however, advanced stages of disease continue to carry a high mortality rate. The discrepancy in treatment success highlights that current treatments are insufficient to treat advanced-stage breast cancer. As new and improved treatments have been sought, one therapeutic approach has gained considerable attention. Oncolytic viruses are uniquely capable of targeting cancer cells through intrinsic or engineered means...
September 10, 2018: Current Gene Therapy
Mehdi Ghamgosha, Ali Mohammad Latifi, Gholam Hossein Meftahi, Alireza Mohammadi
Parkinson's disease (PD) is a frustrating condition characterized by motor and nonmotor deficits majorly caused by the loss of dopaminergic cells in the substantia nigra pars compacta (SNc) and destruction of the nigrostriatal pathway. Despite the very respectable advances in cutting-edge approaches for the treatment of PD, there exist numerous challenges that have incapacitated the definitive treatment of this disease. This review emphasized the development of various non-pharmaceutical therapeutic approaches and mainly highlighted the cutting-edge treatments for PD including gene- and stem cell-based therapies, targeted delivery of neurotrophic factors, and brain stimulation techniques such as transcranial magnetic stimulation (TMS), transcranial direct current stimulation (tDCS), and deep brain stimulation (DBS)...
September 10, 2018: Current Gene Therapy
Oscar A Brown, Martina Canatelli-Mallat, Gloria M Console, Gisela Camihort, Georgina Luna, Eduardo Spinedi, Rodolfo G Goya
BACKGROUND: Insulin-like growth factor1 (IGF1) is a powerful neuroprotective molecule. We have previously shown that short-term hypothalamic IGF1 gene therapy restores tuberoinfundibular dopaminergic neuron function in aging female rats. OBJECTIVE: Our aim was to implement long-term IGF-I gene therapy in pituitary prolactinomas in senile female rats. METHODS: Here, we assessed the long-term effect of IGF1 gene therapy in the hypothalamus of young (4 mo...
September 5, 2018: Current Gene Therapy
Serap Gur, Asim B Abdel-Mageed, Suresh C Sikka, Alma R Bartolome, Wayne J G Hellstrom
Erectile Dysfunction (ED) is a common health problem in roughly 50% of males of advanced age (40-70 years old). Recent attention related gene therapy to ED cases; this received much interest to further progress gene therapy ideals to ED treatment. This review is an attempt to analyze key challenges and to emphasize primary areas, including mostly preclinical and few clinical trials, cellular target(s), and different viral vectors/nanoparticles for gene delivery in ED. While overexpression of target genes can be silenced by RNA interference (RNAi), down-regulation of these mechanisms has been implicated in ED...
July 29, 2018: Current Gene Therapy
Inmaculada Perez-Sanchez, Maria Sabater-Molina, Maria Elisa Nicolas Rocamora, Guillermo Glover, Fuensanta Escudero, Pedro de Mingo Casado, Juan Ramon Gimeno-Blanes
Duchenne muscular dystrophy is a disorder with variable expression caused by frame-disrupting mutations in the dystrophin gene. It is characterized by progressive muscle weakness and dilated cardiomyopathy. In-frame dystrophin mutations cause a clinically moderate disorder named Becker muscular dystrophy. Our aim was to study the clinical and genetic characteristics of a family with inherited cardiomyopathy and Becker muscular dystrophy. The index case was diagnosed with psychomotor retardation at 5 years of age...
July 9, 2018: Current Gene Therapy
Maria Giovanna Sabbieti, Giovanna Lacava, Andrea Amaroli, Luigi Marchetti, Roberta Censi, Piera Di Martino, Dimitrios Agas
BACKGROUND: During last years, DNA vaccine immunogenicity has been optimized by the employment of co-stimulatory molecules and molecular adjuvants. It has been reported that plasmid (pATRex), encompassing the DNA sequence for the von Willebrand A (vWA/A) domain of the Anthrax Toxin Receptor-1 (ANTXR-1, alias TEM8, Tumor Endothelial Marker 8), acts as strong immune adjuvant by inducing formation of insoluble intracellular aggregates. Markedly, we faced with upsetting findings regarding the safety of pATRex as adjuvant since the aggregosome formation prompted to osteopenia in mice...
February 1, 2018: Current Gene Therapy
Ling-Yan Zhou, Zhi-Yao He, Ting Xu, Yu-Quan Wei
Small activating RNAs (saRNAs) are small double-stranded RNAs that could mediate the target-specific gene expression by targeting selected sequences in gene promoters at both the transcriptional and epigenetic levels. This phenomenon of gene manipulation is known as RNA activation (RNAa), which opens up a new pathway for RNA-based gene therapeutics in contrast to RNA interference. Although the exact molecular mechanism of RNAa mediated by saRNAs still remains foggy, some studies have provided the possible ones to explain it...
2018: Current Gene Therapy
Divyanshi Karothia, Paban Kumar Dash, Manmohan Parida, Sameer Bhagyawant, Jyoti S Kumar
BACKGROUND: The West Nile Virus (WNV) has emerged as one of the most significant arboviral infection in many parts of the world and is associated with the encephalitis affecting mainly human and horses. In spite of the fact that the WNV is threat for the public health, there is no vaccine or therapeutic available for the treatment of WNV. METHODS: In this study, we tested a novel RNA interference based technique to inhibit WNV replication in Vero cells. Two siRNAs were designed against the NS2A and NS5 regions of WNV which are highly conserved among Flaviviruses as it play important role in apoptosis and in viral replication respectively...
2018: Current Gene Therapy
Lihua Peng
No abstract text is available yet for this article.
2018: Current Gene Therapy
Lung-Ji Chang, Alfred S Lewin
No abstract text is available yet for this article.
2018: Current Gene Therapy
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