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Current Gene Therapy

Xiao-Xiao Lu, Shao-Zhen Zhao
BACKGROUND: As one of the main blinding ocular diseases, corneal blindness results from neovascularization that disrupts the angiogenic privilege of corneal avascularity. Following neovascularization, inflammatory cells are infiltrating into corneal to strengthen corneal injury. How to maintain corneal angiogenic privilege to treat corneal disease has been investigated for decades. MATERIALS & METHODS: Local administration of viral and non-viral-mediated anti-angiogenic factors reduces angiogenic protein expression in situ with limited or free of off-target effects upon gene delivery...
December 13, 2018: Current Gene Therapy
Sushmita Nair, Jing-Bo Wang, Shih-Ting Tsao, Yuchen Liu, Wei Zhu, William B Slayton, Jan S Moreb, Lujia Dong, Lung-Ji Chang
Recent studies of CD19-specific chimeric antigen receptor (CAR)-modified T cells (CARTs) have demonstrated unprecedented successes in treating refractory and relapsed B cell malignancies. The key to the latest CART therapy advances can be attributed to the improved co-stimulatory signals in the CAR design. Here we established several novel CARs by incorporating T cell signaling domains of CD28 in conjunction with intracellular signaling motif of 4-1BB, CD27, OX40, ICOS, and IL-15Rα. These novel CARs were functionally assessed based on a simple target cell killing assay...
November 15, 2018: Current Gene Therapy
Thoufic Anam Azad, Umme Qulsum, Toshifumi Tsukahara
Members of the adenosine deaminase acting on RNA (ADAR) family of enzymes consist of double-stranded RNA-binding domains (dsRBDs) and a deaminase domain (DD) that converts adenosine (A) into inosine (I), which acts as guanosine (G) during translation. Using the MS2 system, we engineered the DD of ADAR1 to direct it to a specific target. The aim of this work was to compare the deaminase activities of ADAR1-DD and various isoforms of ADAR2-DD. We measured the binding affinity of the artificial enzyme system on a Biacore™ X100...
November 14, 2018: Current Gene Therapy
E A Borobova, D V Antonets, E V Starostina, L I Karpenko, A A Ilyichev, S I Bazhan
OBJECTIVE: Immunotherapy based on induction of T-cell response is a promising approach to treatment of oncological diseases that currently attracts particular attention of researchers. The study aims to design artificial epitope-based immunogens, DNA vaccine candidates against melanoma and evaluate their antitumor response within the system of T-cell response induction ex vivo. METHODS: The study used computer methods for predicting T-cell epitopes and designing polyepitope antigens, DNA vaccine candidates against melanoma...
November 12, 2018: Current Gene Therapy
Anna Aspesi, Chiara Borsotti, Antonia Follenzi
Diamond Blackfan Anemia (DBA) is an inherited erythroid aplasia with onset in childhood. Patients carry heterozygous mutations in one of 19 ribosomal protein (RP) genes, that lead to defective ribosome biogenesis and function. Standard treatments include steroids or blood transfusions but the only definitive cure is allogenic hematopoietic stem cell transplantation (HSCT). Although advances in HSCT have greatly improved the success rate over the last years, the risk of adverse events and mortality is still significant...
November 9, 2018: Current Gene Therapy
Mafalda G Moleirinho, Sara Rosa, Manuel J T Carrondo, Ricardo J S Silva, Åsa Hagner-McWhirter, Gustaf Ahlén, Mats Lundgren, Paula M Alves, Cristina Peixoto
Oncolytic virus therapy is currently considered as a promising therapeutic approach for cancer treatment. Adenovirus is well-known and extensively characterized as an oncolytic agent. The increasing number of clinical trials using this virus generates the demand for the development of a well-established purification approach. Triton X-100 is commonly used in cell lysis buffer preparations. The addition of this surfactant in the list of substances with very high concern of the Registration, Evaluation, Authorization and Restriction of Chemicals (REACH) regulation promoted the research for effective alternatives...
November 9, 2018: Current Gene Therapy
Shuai Fang, Jinchang Pan, Chengwei Zhou, Hui Tian, Jinxian He, Weiyu Shen, Xiaofeng Jin, Xiaodan Meng, Nan Jiang, Zhaohui Gong
Circular RNAs (circRNAs) are a class of non-coding RNAs (ncRNAs) that structurally form closed loops without 5'-end cap and 3'-end poly(A) tail unlike linear RNAs. CircRNAs are widely present in eukaryotic cells and are presented with capabilities and characteristics of structural stability, high abundance and cell-/tissue-specific expression. A growing body of research now suggests that the aberrant expression of circRNA is intimately relevant to the occurrence and development of cancer. In this review, we mainly discuss the differentially expressed circRNAs in cancer tissues, plasma and exosomes, which makes it possible for clinicians to use certain circRNAs as novel biomarker for cancer diagnosis and prognosis...
November 9, 2018: Current Gene Therapy
Jiajia Yuan, Yong Zhang, Hongli Liu, Zhen Tian, Xin Li, Yichen Zheng, Qin Gao, Lin Song, Xiao Xiao, Jian Sun, Zhitao Wang, Bin Li
BACKGROUND: Leber's hereditary optic neuropathy is a hereditary disease inherited from mitochondria. So far, there is no effective treatment, and gene therapy has become the most promising and promising method. Because of the possibility of spontaneous visual acuity recovery in this disease, we screened the patients in before gene therapy, excluding those with spontaneous visual acuity improvement, and prepared for the subsequent gene therapy. OBJECTIVE: To clinically observe the course of Leber's hereditary optic neuropathy for 6 months prior to gene therapy...
November 5, 2018: Current Gene Therapy
Liang Cheng, Yang Hu
No abstract text is available yet for this article.
November 1, 2018: 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
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
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