Health and Medicine
107 Genetic Therapy of Sickle Cell Disease
Riley Reyes
Genetic Therapy and its effects on society
Connection to sts
Genetic Therapy is a new technology that can manipulate the genetic information of someone’s genetic makeup to alter biological properties. Gene therapy, specifically the therapy for sickle cell disease is an example of the importance of using science and technology coherently, along with their effects on society. Gene therapy is a science studied relatively recently, using newly discovered technologies like CRISPR-Cas9 to potentially cure diseases, making it a prime example for how science, technology, and society affect each other. While gene therapy has the potential to save lives, but can also have some drawbacks, especially due to its relative recency (Abraham, A. A. et al., 2021).
Introduction
Genetics is the “coding” of the body. Each gene contains DNA which is made into proteins through biological processes. Genes can be malfunctional due to a change in the chemical makeup or they can be a mutation, where they are changed during the passing down from parents or faulty reproduction of genes. Gene therapy is being studied to fix a malfunctioning gene or replace a malfunctioning gene with a healthy one. While the possible results of genetic therapy are exciting and extremely beneficial, there are reasons researchers have a difficult time getting therapies approved. Many of the risks that are involved with gene therapy are the processes and techniques used to insert a gene. Most gene therapies use something called a “vector” or a carrier that would be used to transport genes out of and into the body. The most common vector used is a virus (U.S. FDA n.d.). Researchers have found viruses to be very useful in multiple ways, but they can also present risks such as a negative immune system reaction, an infection, and targeting the wrong cells
There are a few different ways that gene therapy can alter biological properties. The first is replacing a gene that is causing a disease. The disease-causing gene would be removed, and replaced with a gene that would produce healthy proteins to benefit the body. The second way gene therapy could work is by inactivating a disease-causing gene. This can be done by inserting a gene into the same chromosome that would block the production of proteins by the diseased-gene. The last way gene therapy could work is by introducing a new gene into the body. Inserting a new gene into the body can negate, or act oppositely to the disease-causing gene (Mayo Clinic, 2024). Each of these techniques have different qualities that make them better fits for different diseases as gene therapy aims to relieve diseases like cancer, heart disease, and sickle cell disease.
Gene therapy for sickle cell disease
More specifically, there is research about many clinical trials done while trying to approve two genetic therapies for sickle cell disease (SCD). These therapies are called Casgevy and Lyfgeni