Viral and Non-viral Vectors in Gene Therapy: Technology Development and Clinical Trials
نویسندگان
چکیده
منابع مشابه
Barriers and recent advances in non-viral vectors targeting the lungs for cystic fibrosis gene therapy
Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in CFTR genes that affect chloride ion channel. The CF is a good nominee for gene therapy as the asymptomatic carriers are phenotypically normal, and the desired cells are accessible for vector delivery. Gene therapy shows promising effects involving the correction of gene or replacement of the mutant gene with the func...
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In a range of human trials, viral vectors have emerged as safe and effective delivery vehicles for clinical gene therapy, particularly for monogenic recessive disorders, but there has also been early work on some idiopathic diseases. These successes have been enabled by research and development efforts focusing on vectors that combine low genotoxicity and immunogenicity with highly efficient de...
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Uses of viral vectors have thus far eclipsed uses of non-viral vectors for gene therapy delivery in the clinic. Viral vectors, however, have certain issues involving genome integration, the inability to be delivered repeatedly, and possible host rejection. Fortunately, development of non-viral DNA vectors has progressed steadily, especially in plasmid vector length reduction, now allowing these...
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Cardiovascular disease represents the most common cause of mortality in the developed world but, despite two decades of promising pre-clinical research and numerous clinical trials, cardiovascular gene transfer has so far failed to demonstrate convincing benefits in the clinical setting. In this review we discuss the various targets which may be suitable for cardiovascular gene therapy and the ...
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Min Li *, Joel A. Rodriguez, William E. Fisher, Xiaoliu Zhang, Changyi Chen and Qizhi Yao * Molecular Surgeon Research Center, Elkins Pancreas Center, Michael E. DeBakey Department of Surgery, Department of Molecular Virology and Microbiology, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, Texas 77030 ______________________________________________________________________...
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ژورنال
عنوان ژورنال: Technology in Cancer Research & Treatment
سال: 2003
ISSN: 1533-0346,1533-0338
DOI: 10.1177/153303460300200513