Shielded vectors improve liver gene therapy
نویسندگان
چکیده
منابع مشابه
Beta thalassemia gene therapy using lentiviral vectors
Recent years, allogeneic bone marrow transplantation (BMT) has proved to be the successful cure for patients with thalassemia major, however this is restricted due to limited matched-related donor. Its complications include chronic graft-versus-host disease in 5-8% of patients. So, a molecular approach, such as gene therapy for direct normal beta globin gene transmission, seems quite promising ...
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Herpes simplex virus type 1 (HSV-1) is a common human virus, best known as the causative agent of recurrent labial herpes (cold sores) [1]. The unique properties of HSV-1 have increasingly been exploited in vector design, particularly for therapy of the central nervous system diseases. HSV has natural properties useful for a therapy vector: the latent, lifelong infections in the nervous system ...
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Gene therapy is a novel method under investigation for the treatment of genetic, metabolic and neurologic diseases, cancer and AIDS. The primary goal of gene therapy is to deliver a specific gene to a pre-determined target cell, and to direct expression of such a gene in a manner which will result in a therapeutic effect. Retroviral vectors have the ability to integrate in the host cell DNA irr...
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Bacteria can be used for gene therapy via two strategies - either by transfection of eukaryotic host cells using bacteria (bactofection) or by alternative gene therapy that does not alter the host genome, but uses the prokaryotic expression system, which can be controlled or stopped from outside. While bactofection is optimal for gene substitution and DNA vaccination, alternative gene therapy i...
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PURPOSE Metabolic inherited liver diseases are attractive targets for gene therapy. Recombinant lentiviruses are very powerful viral vectors able to infect nonmitotic cells. We wanted to develop a new surgical approach to improve gene transfer in adult liver using low viral doses. MATERIALS AND METHODS Adult rats were injected with 2.108 infectious particles of lentiviral vectors encoding the...
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ژورنال
عنوان ژورنال: Lab Animal
سال: 2019
ISSN: 0093-7355,1548-4475
DOI: 10.1038/s41684-019-0367-x