Anna Victor

Gene editing and (fragile x syndrome or rett syndrome)

Started Mar. 14, 2024

Abstract or project description

I would first describe some of the many forms of gene editing and gene therapy. I would then talk about some of the various uses of genetic engineering. I would then delve deeper into one of the genetic diseases, Fragile X Syndrome (or Rett syndrome). I would give context about what the disease is, and how it is caused. For example, if I was talking about FXS, I would explain that it is caused by a single gene mutation in the fragile X mental retardation 1 gene, causing intellectual disability and repetitive behaviors. Then I would discuss some studies that have been shown to provide a means of genetic treatment to FXS. One example of the type of paper I may discuss is the one linked below, which uses nanoparticles to deliver CRISPR-Cas-9 into neurons due to the consequences of viral transportation. The paper attempts to prove that injection of the nanoparticle into the striatum is not hazardous to neurons, that deletion of the repetitive gene decreases the detrimental protein, thus reducing the mice’s repetitive behavior, and that the nanoparticle is capable of knocking out genes. After talking about various clinical trials such as the one I have summarized above, I would talk about the ethics and pros/cons of gene editing.