Targeted Gene Therapy Via Lentiviral Vectors

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Gene Therapy Overview

Figure 1. Schematic drawing of ex vivo and in vivo gene therapy Bulcha et al. (2021)

By Drew Albrecht

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. Genetic modification can rewire the cells to either produce a therapeutic effect[Kaji] or simply just replace the mutated or missing DNACite error: Invalid <ref> tag; invalid names, e.g. too many [1]

A citation code consists of a hyperlinked reference within "ref" begin and end codes.
To repeat the citation for other statements, the reference needs to have a names: "<ref name=aa>"
The repeated citation works like this, with a forward slash.[2]

  1. Bartlett et al.: Oncolytic viruses as therapeutic cancer vaccines. Molecular Cancer 2013 12:103.
  2. Cite error: Invalid <ref> tag; no text was provided for refs named aa