Essay on The Concept Of Gene Therapy

1155 Words Feb 5th, 2015 null Page
Gene Therapy:
The concept of gene therapy emerged in the 1970s. The basic premise of gene therapy is using DNA enclosed in a vector as a therapeutic treatment for diseases. In the decades after it was conceptualized, many clinical trials of gene therapy were developed but stalled in phase I or phase II. During the 90s, only 1% of gene therapy trials made it to phase III and none of them went past phase III. The excitement for gene therapy was reduced when a participant of a clinical trial died in 1999. Since the 1990s, more modern vectors, vehicles to deliver the DNA, were developed including viruses, plasmids, and minicircle DNA. These vectors are engineered to contain the therapeutic genes. Viral vectors hold the advantage of a higher transfection profile as well as the ability to target certain cells. However, viral vectors are limited by the amount of genes they can carry, in vivo immune response, and safety concerns. Most gene therapy cases have employed viral vectors despite their drawbacks. On the other hand, plasmids can carry larger genes but has a lower efficiency. Minicircle DNA has been proposed as an alternative vector and shown to have more transfection efficiency and more resistance to in vivo silencing. Once these vectors are delivered by intravenous injection, they transfect the target cells. When these vectors transfer the targeted cells, the genes are expressed, resulting in the production of therapeutic proteins and thus treating protein deficiency…

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