GENE THERAPY: AN OVERVIEW
Namrata Patel*, Dr. Amresh Gupta, Dr. Arpita Singh, Nitish Kumar and Mohd. Aqil Siddiqui
The ability to form site-specific modifications to the human genome has been an objective in medicine since the popularity of the gene because the basic unit of heredity. Thus, gene therapy is known because the ability of genetic improvement through the correction of altered (mutated) genes or site-specific modifications that focus on therapeutic treatment. This therapy became possible through the advances of genetics and bioengineering that enabled manipulating vectors for delivery of extrachromosomal material to focus on cells. One of the most focuses of this system is that the optimization of delivery vehicles (vectors) that are mostly plasmids, nanostructured or viruses. The viruses are more often investigated thanks to their excellence of invading cells and inserting their genetic material. However, there's great concern regarding exacerbated immune responses and genome manipulation, especially in germ line cells. In vivo studies in in vegetative cell showed satisfactory results with approved protocols in clinical trials. These trials are conducted within the us, Europe, Australia and China. Recent biotechnological advances, like induced pluripotent stem cells in patients with liver diseases, chimeric antigen receptor T-cell immunotherapy, and genomic editing by CRISPR/Cas9, are addressed during this review.
Keywords: Gene therapy, Genetic Vectors, Gene transfer.
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