The Forever Fix: Genetic Analysis

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Ricki Lewis’s The Forever Fix: Gene Therapy and the Boy Who Saved It tells the tale of gene therapy’s rocky road from a wild idea people considered to be a “daydream” to a growing field providing lucky individuals with treatment to prevent their life-shattering genetic diseases. In her novel, Lewis discusses two major biological concepts: mutation and gene expression.
To give the reader the molecular basis for genetic disorders, mutation is briefly addressed. Lewis gives two different examples of mutations- specifically, the mutations that Nancy and Ethan gave to Corey. Ethan’s mutation is one in which “one DNA base subs for another, like a one-letter typo” (Lewis, 2013, p.27). The “base” that she is referring to is a nitrogenous base of a
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Recently, for example, a study funded by the NIH found that a mutation to a gene called DISC1, which regulates several genes, causes “errant expression of genes known to be involved in synaptic transmission, brain development, and key extensions of neurons where synapses are located” ("Suspect Gene Corrupts Neural Connections," 2014). This aberrant expression has been linked with several mental disorders, such as schizophrenia, depression, and bipolar disorder. The researchers then attempted to correct the damage done by the mutation by giving the test subjects-neural cells- functional DISC1 genes. They found that the gene therapy amended the troubles to the cells caused by the mutations, giving hope for those who have mental disorders rooted in the DISC1 mutation; a simple one-gene fix could cure them, but more research would be needed to determine if gene therapy would be possible for their …show more content…
And consequently, to understand gene expression is to understand why these mutations can have such grave effects on an individual. For DNA to direct cellular processes, it must be transcribed and translated, The AP Biology Curriculum Framework explains. It goes on to say that “the products of transcription and translation play an important role in determining metabolism, i.e. cellular activities and phenotypes.” So if “changes in a nucleotide sequence, if present in a protein-coding region, can change the amino acid sequence of the polypeptide” (AP Biology Curriculum Framework, p. 37), changes to these essential proteins prevents cellular activities from being performed. The field of gene therapy began due to knowing about mutations and their effects on gene expression. By knowing which of Corey’s genes have changes to their nucleotide sequence, the researchers working on him were able to give him the correct gene to make the protein necessary for his eyes to use “vitamin A [for sending] visual signals to his brain” (Lewis, 2013, p. 8). Learning objectives 3.24 to 3.28 apply to mutation, while LOs 3.4 and 3.6 apply to gene

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