Gene Therapy For Deficiency Treatment

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We all have different genes, and it makes us who we are. It creates a distinction of our appearance in all aspect. That’s the reason why we differ in height, we have different eye colors, and we have unique skin types, and so on. Our genes hold the answers to our differences. But what if a part of our gene went missing? What if it gets defective? Or what if it mutates and changes into something we don’t know? It can inevitably cause a disease such as cancer and cystic fibrosis. Also, according to Traci Stein Ph.D., MPH, “This post may be of most significant interest to those who have been diagnosed with one of the mental health issues linked to having an MTHFR mutation, such as depression, bipolar disorder, schizophrenia, attention-deficit hyperactivity disorder (ADHD), or autism.” With that, we need to understand what gene therapy is and how it works.


All About The Biotechnology

With all the advancement of science in today’s era, everything is “almost” possible. That includes scientists’ capability in changing a bad gene with one that is healthy. The addition of a functioning gene to replace missing or defective ones is quite attainable. It helps in reducing bad and unwanted genes in the body while also turning off some of those problematic ones as well. But how does science do it?

Let’s say a scientist wants to get back a copy of a healthy gene to cells. The efficient way to do it is by using vehicles or vectors that will deliver the healthy gene into the targeted location. That is where they use modified viruses as a vector because it can easily pass through the body’s defense system and attach itself to the functioning cells. The process of editing a virus is through removing its ability to cause disease so it can carry a healthy gene into a cell.  It has the potential to eradicate genetic diseases. It shows endless possibilities in catering genetic defects such as deafness, blindness, sickle cell anemia, Huntington’s and Parkinson’s disease, and destroying cancer cells.


The Process Of Administering Gene Therapy

Gene therapy is working its wonders inside the body by injecting the virus into the affected area. It can be administered via IV where it typically consumes a more extended period. The process may take up a few hours depending on factors such as body weight too. With that, adults take most of the waiting time compared to kids. But the good thing is, the injection process can be completed immediately as soon as the fluid gets injected into the area.

And due to the advancement of technology, the process of cell regeneration can happen systematically as well. Usually, checking and observing a gene occurs in the laboratory where it gets tested for countless times by an expert scientist. After completion of the procedure and having to create a vector, it gets transplanted back to the body. But it is essential to know that the process often targets all diseased cells in particular.

The Different Responses

Every individual has complete different DNAs. Therefore, gene therapy shows variation. That’s the reason why it sometimes works better and faster with others, while it doesn’t show quite a bit of progress in some. There’s always variability on the effect of the drug depending on the dosage or number of cells needed to reach the other functioning and destroyed ones. Their impact also depends on the potential enzyme contribution that the introduced cell can produce. It is also crucial to take note that like many other drugs, side effects are typical. It is possible to happen because the immune system reacts to the virus. With that, an individual may experience a fever. And also, having severe responses can occur as well. But it is very vital to remember that no drug should be given, use, and take by humans until they are clinically proven safe and effective.


Is It A Cure?

Well, gene therapy is still in the experimental phase, so it seems early to conclude its overall performance as a cure. Just as Andrea Farkas Patenaude, PhD says, “the cost of the test depends on whether someone in your family who has been diagnosed with cancer has already been found to be a mutation carrier. If that is the case, your testing is likely to cost between $300 and $500, and your health insurance may pay for testing.” However, one thing is sure, the changes and its progress will eventually appear on future generation solution. Honestly, it is too early to conclude that this process can support the permanent outcome. However, its good progress compared to other treatment is quite impressive. The discovery is a big step to achieving a long and healthy life by addressing genetic diseases that people once thought was incurable. That’s perhaps enough reason to trust it for now. Ricki Lewis, PhD talks about several moms’ experience with gene therapy saying “Parents of kids in clinical trials, especially for something as unprecedented as gene therapy, must take care in describing what they may or may not see in their children. It’s human nature to note progress, but disease progression, not so much.”

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