Current Issue : January - March Volume : 2016 Issue Number : 1 Articles : 5 Articles
Alphavirus vectors present an attractive approach for gene therapy applications\ndue to the rapid and simple recombinant virus particle production and their broad range of\nmammalian host cell transduction. Mainly three types of alphavirus vectors, namely naked\nRNA, recombinant particles and DNA/RNA layered vectors, have been subjected to\npreclinical studies with the goal of achieving prophylactic or therapeutic efficacy,\nparticularly in oncology. In this context, immunization with alphavirus vectors has provided\nprotection against challenges with tumor cells. Moreover, alphavirus intratumoral and\nsystemic delivery has demonstrated substantial tumor regression and significant prolonged\nsurvival rates in various animal tumor models. Recent discoveries of the strong association\nof RNA interference and disease have accelerated gene therapy based approaches, where\nalphavirus-based gene delivery can play an important role....
Bone marrow gene therapy remains an attractive option for treating chronic\nimmunological diseases, including acquired immunodeficiency syndrome (AIDS) caused\nby human immunodeficiency virus (HIV). This technology combines the differentiation\nand expansion capacity of hematopoietic stem cells (HSCs) with long-term expression of\ntherapeutic transgenes using integrating vectors. In this review we summarize the potential\nof bone marrow gene therapy for the treatment of HIV/AIDS. A broad range of antiviral\nstrategies are discussed, with a particular focus on RNA-based therapies. The idea is\nto develop a durable gene therapy that lasts the life span of the infected individual, thus\ncontrasting with daily drug regimens to suppress the virus. Different approaches have been\nproposed to target either the virus or cellular genes encoding co-factors that support virus\nreplication. Some of these therapies have been tested in clinical trials, providing proof of\nprinciple that gene therapy is a safe option for treating HIV/AIDS. In this review several\ntopics are discussed, ranging from the selection of the antiviral molecule and the viral\ntarget to the optimal vector system for gene delivery and the setup of appropriate preclinical\ntest systems. The molecular mechanisms used to formulate a cure for HIV infection are\ndescribed, including the latest antiviral strategies and their therapeutic applications. Finally,\na potent combination of anti-HIV genes based on our own research program is described....
Background: Modification of the glutamic acid decarboxylase (GAD) level is a promising future treatment tool for Parkinson's disease (PD). GAD is the rate-limiting enzyme in the synthesis of the inhibitory neurotransmitter gamma amino butyric acid (GABA) and modification of the GAD level could therefore potentially be used to alter the GABA output from a nucleus.\n\nMethods: We have previously developed a synthetic microRNA (smiRNA1550) designed to knockdown the GAD isoform GAD67 and shown that it can efficiently knockdown the target protein. In this study we have further evaluated the therapeutic potential of smiRNA1550 in treating motor symptoms using the 6-OHDA rat model of PD and lentiviral vectors. The smiRNA was evaluated in striatum and substantia nigra pars reticulata (SNpr), two nuclei with increased GAD levels and GABA output in PD. The result was evaluated by behaviour tests and western blot. Differences between groups were assessed using analysis of variance (ANOVA) followed by the Tukey's multiple comparison test for group comparisons.\n\nResults: There was a beneficial effect in apomorphine-induced rotations after injection of LV-smi RNA1550 into SNpr.\n\nConclusion: These findings suggest that manipulation of the GABA system could be a valid approach for development of novel gene therapies for PD that warrant further study....
Gene carriers with cell specific ligand molecules are needed for the treatment of several diseases. Mannose is known to be\nrecognized and incorporated into the cells through mannose recognition lectins that are exclusively expressed on macrophages.\nIn this study, we synthesized two types of mannose-modified lipids with different stereoisomer (...
Gene therapy was originally thought to cover replacement of malfunctioning\ngenes in treatment of various diseases. Today, the field has been expanded to application\nof viral and non-viral vectors for delivery of recombinant proteins for the compensation of\nmissing or insufficient proteins, anti-cancer genes and proteins for destruction of tumor cells,\nimmunostimulatory genes and proteins for stimulation of the host defense system against\nviral agents and tumors. Recently, the importance of RNA interference and its application in\ngene therapy has become an attractive alternative for drug development....
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