By Sean Rowell Writer Imagine that you had the opportunity to rid the world of disease by preventing the suffering of millions through one single treatment. Would you do it? This question, once a far-fetched idea, is becoming a reality as gene therapy techniques become more advanced and precise. Gene therapy is a branch of science that involves manipulating genes to treat a disease or even prevent one from occurring in the first place. There are many ways in which this can be done. A researcher may replace a gene that is not functioning properly and causing disease with a healthy version of the gene. This researcher could also introduce a brand new gene into the patient that would help fight a certain disease. Whatever the method, this type of gene therapy offers a way for patients to be treated for their diseases at the source. Instead of merely treating the symptoms of a disease, gene therapy attacks a disease at its origin – the DNA of the patient. This type of gene therapy is known as somatic gene therapy, which targets the defect in the specific tissue that causes the disease of a patient. The other type of gene therapy is germ line therapy. Here, the sperm or eggs of a patient are treated in order to prevent a genetic disorder from affecting offspring. While these two types of gene therapy both accomplish the same goal of eradicating disease, they have drastically different implications. Somatic cell therapy only affects the treated patient. With germ line therapy, the results are much more extensive. The changes made in the sperm or eggs of the patient will not only affect their offspring, but can also affect future generations, thereby introducing a genetic change into the human population. One new gene therapy technique that is causing great concern in the scientific community is the Clustered Regularly Interspaced Short Palindromic Sequences, or CRISPR technique. This technique allows for highly specific gene manipulation, with DNA sequences able to be cut out or added with ease. The CRISPR technique uses an enzyme called Cas9 along with a strand of RNA that will guide it to the target sequence of DNA. Once at the desired DNA sequence, the Cas9 enzyme can edit the DNA or insert new sequences. The reason this technique causes concern is that it allows for the convenient and cheap modification of genes in cells, including sperm and eggs. While CRISPR has not been extensively tested in humans and human germ lines, the rapid growth and popularity of this technique raises concerns. One worry is that CRISPR could eventually be used to create heritable changes in the human genome by introducing genes and traits that did not exist before. Others worry that these genetic mutations could prove to be harmful, as it may disrupt the delicate balance of entire ecosystems through introducing genetically engineered organisms. This cutting edge technique will continue to be at the forefront of the ethics of gene therapy for years, and only time will tell where it will take the human population. Sources: [1] http://learn.genetics.utah.edu/content/genetherapy/gtintro/ [2] http://ghr.nlm.nih.gov/handbook/therapy/genetherapy [3] https://www.sciencebasedmedicine.org/crispr-and-the-ethics-of-gene-editing/ [4] https://www.ndsu.edu/pubweb/~mcclean/plsc431/students/bergeson.htm [5] http://www.nature.com/news/crispr-the-disruptor-1.17673
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AuthorsThe authors of these blog posts are staff writers of The Triple Helix at Georgetown University. Archives
November 2016
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