What is Gene Therapy?
Gene Therapy is an experimental form of treatment that helps introduce new genes into the genetic material in cells that help treat and prevent diseases. While this is still an experimental treatment it has helped treat diseases such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia, and AIDS (acquired immunodeficiency syndrome).
How does Gene Therapy work?
Gene therapy allows doctors to treat diseases on a cellular level; a doctor treats a patient's disease or disorder with gene therapy by inserting a functioning gene into a patient's targeted cell instead of using drugs or surgery. Many researchers have introduced new genes to a patient's target cell by using a vector. There are viral and non-viral vectors; viral vectors are commonly used for In vivo gene therapy and non-viral vectors are commonly used for ex vivo gene therapy.
Ex vivo gene therapy is when the genetic modification of cells happens outside of the body. So, therapy-targeted cells are taken out of the body and exposed to the desired genes or virus. After the targeted cell is exposed, it is then re-infused to add the working gene back into the body. Ex vivo gene therapy has been used to treat neurological disorders such as Parkinson's disease, Alzheimer's disease, ALS, Huntington's disease, and stroke.
In vivo gene therapy is where the genetic modification of cells happens inside the body; this type of gene therapy has been used to treat immunodeficiency. Scientists use retrovirus like HIV to treat immunodeficiency because Retrovirus like HIV are good at inserting genes into targeted cells. According to Scientific American, “More than 30 patients have been treated for SCID, and more than 90 percent of those children have been cured of their disorder—an improvement over the 50 percent chance of recovery offered by bone marrow transplants.” However, there are some risks with retroviruses; not only are they able to insert genes into targeted cells, but they are also capable of inserting their genes anywhere. When a change like this happens in DNA it can disrupt genes leading to a disrupted body. While there are many successful cases, there have been “five of the 30 children treated for SCID have experienced this complication” (Scientific American).
While there are positive and successful cases in gene therapy, there are also potential risks that can heavily affect the body. Therefore, gene therapy is still an experimental technique that still needs to be made sure that it will be safe and effective.
Resources:
https://www.pfizer.com/science/research-development/gene-therapy/genes-as-medicines
https://www.scientificamerican.com/article/experts-gene-therapy/
https://www.docwirenews.com/docwire-pick/how-does-gene-therapy-work/
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3848228/
Images:
https://www.aop.org.uk/-/media/ot/archive/content-listing-images/science-and-vision/research/research-2017/dna.jpg
https://foglets.com/content/images/size/w2000/2021/03/gene-therapy-1.jpg
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