Targeted Gene Therapy Via Lentiviral Vectors

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Gene Therapy Overview

Figure 1. Schematic drawing of ex vivo gene therapy FDA


By Drew Albrecht


Gene therapy is the (HELP). Genetic disorders like cystic fibrosis, phenylketonuria, sickle-cell anemia, and many more, are a result of mutated or absent genes. The goal of gene therapy is to treat or cure such diseases via genetic modification of the cells in an affected individual. This genetic modification can rewire the cells to either produce a therapeutic effect[Kaji] or simply just replace the mutated or missing DNA[Lai].




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Subscript: H2O
Superscript: Fe3+

Lentiviruses



Sample citations: [1] [2]

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  1. ↑ 1.0 1.1 Hodgkin, J. and Partridge, F.A. "Caenorhabditis elegans meets microsporidia: the nematode killers from Paris." 2008. PLoS Biology 6:2634-2637.
  2. ↑ Bartlett et al.: Oncolytic viruses as therapeutic cancer vaccines. Molecular Cancer 2013 12:103.
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