Gene Therapy: A Deep Dive to an Emerging Field
Hello and welcome, folks, to another science-packed post! Today, we are taking a deep dive into one of the most popular and advancing therapies in the medical industry: Gene therapy. More specifically, the types of gene therapy.
However, before we jump in, the question must be answered: What is Gene Therapy? Well, my dear reader, I'm glad you asked. Gene therapy is a form of therapy that works by specifically targeting the DNA or RNA. By doing so, the root cause of a number of neurological issues can be directly addressed. This therapy has received an immense amount of attention in recent years, and cutting-edge technology and research have been used to advance its advancement. There are4 categories under this therapy: Regulatory gene modulation, Gene editing, Gene Replacement, and Gene silencing. Each of those has several specific therapies within them; however, we won't get into that until next week ;). Now, let's begin our journey into the deep (scientifically speaking).
The first is gene editing: This therapy works by directly editing pieces of DNA within the nervous system. It can be administered either by using in vivo, meaning the components are delivered directly into the body in order to edit the cells, or ex vivo, meaning the cells are taken out, modified, and re-administered. Gene editing is done in a few different ways. The first is by gene integration, in which the correct DNA sequence is added to a gene so it can form more functional proteins in the event that the amount of proteins the gene makes is lacking. Next is gene activation, in which cells with genes that had previously been turned off are turned on again in order to combat a lack of protein. The third is to “knock out” a gene in a cell if there is too much protein being produced or the protein that is produced is dysfunctional.In the case of JHD/HD, gene editing is used to knock out the HTT gene (Gene Editing |, 2021).
The next therapy type is gene silencing: This therapy method is largely considered the most effective method for reducing mutant huntingtin protein growth due to its effectiveness in animal trials and in certain adult-onset HD trials. It works by essentially inactivating the HTT gene. By doing so, production of the faulty gene is blocked entirely, which tackles the problem at its root. This therapy method is done in vivo (Ofori Canacoo, 2019).
Continuing, there is regulatory gene modulation: In this therapy, genes are targeted by either being turned “on,” in which they are activated, or “off,” in which they are suppressed. This therapy is both in vivo and ex vivo. In the case of HD/JHD, this therapy method causes the gene to be turned off due to its harmfulness within the brain. This is done in vivo for these specific diseases (Butterfield et al., 2025).
However, before we jump in, the question must be answered: What is Gene Therapy? Well, my dear reader, I'm glad you asked. Gene therapy is a form of therapy that works by specifically targeting the DNA or RNA. By doing so, the root cause of a number of neurological issues can be directly addressed. This therapy has received an immense amount of attention in recent years, and cutting-edge technology and research have been used to advance its advancement. There are4 categories under this therapy: Regulatory gene modulation, Gene editing, Gene Replacement, and Gene silencing. Each of those has several specific therapies within them; however, we won't get into that until next week ;). Now, let's begin our journey into the deep (scientifically speaking).
The first is gene editing: This therapy works by directly editing pieces of DNA within the nervous system. It can be administered either by using in vivo, meaning the components are delivered directly into the body in order to edit the cells, or ex vivo, meaning the cells are taken out, modified, and re-administered. Gene editing is done in a few different ways. The first is by gene integration, in which the correct DNA sequence is added to a gene so it can form more functional proteins in the event that the amount of proteins the gene makes is lacking. Next is gene activation, in which cells with genes that had previously been turned off are turned on again in order to combat a lack of protein. The third is to “knock out” a gene in a cell if there is too much protein being produced or the protein that is produced is dysfunctional.In the case of JHD/HD, gene editing is used to knock out the HTT gene (Gene Editing |, 2021).
The next therapy type is gene silencing: This therapy method is largely considered the most effective method for reducing mutant huntingtin protein growth due to its effectiveness in animal trials and in certain adult-onset HD trials. It works by essentially inactivating the HTT gene. By doing so, production of the faulty gene is blocked entirely, which tackles the problem at its root. This therapy method is done in vivo (Ofori Canacoo, 2019).
Continuing, there is regulatory gene modulation: In this therapy, genes are targeted by either being turned “on,” in which they are activated, or “off,” in which they are suppressed. This therapy is both in vivo and ex vivo. In the case of HD/JHD, this therapy method causes the gene to be turned off due to its harmfulness within the brain. This is done in vivo for these specific diseases (Butterfield et al., 2025).
Finally, gene replacement: This therapy aims to target diseases using one gene to replace a faulty gene. In the case of HD/JHD, the replaced gene would be the HTT gene. While not considered as effective or popular as other gene therapy methods, this is still considered a potential candidate for combating HD/JHD. This is done in vivo and ex vivo depending on the case (Mayo Clinic, 2024).
Gene therapy is truly a marvel, and currently, I am in the process of researching it. I am writing a paper on different gene therapy treatments for each category and comparing them based on potential effectiveness in JHD. Anyway, to leave you off a question: What is something you are interested in researching, and do you believe you'll be able to? Thanks for reading, I hope you learned something new, and I will see you next week!
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