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Gene Therapy

Author: Bryan Lin

Editors: Oscar Chen, Fiona Cheng 

Artist: Christina Chen


In 2012, at just six years old, Emily Whitehead became the first pediatric patient to receive CAR-T cell therapy for leukemia, which is a blood cancer where the bone marrow produces too many abnormal white blood cells. Today, she's a thriving college student whose treatment paved the way for thousands of other cancer patients. Gene therapy is a process that has saved Whitehead, along with many others’ lives. But what exactly is gene therapy, and how did we figure it out?

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Gene therapy is the process of replacing, modifying, or introducing a new gene into the body to help treat a disease. To insert new genes directly into the cell, scientists use a delivery system known as vectors. Vectors are genetically engineered viruses that can help deliver therapeutic genes to target cells. Viruses are used because of their ability to deliver genetic material into a cell, which has two functions: regulating gene expression and introducing genes that can help counteract a disease. By replacing the disease-causing gene, you are effectively removing the root of the problem. Modifying the gene can cause it to deactivate, and introducing a new gene can actively counteract its effects. If an altered gene causes a necessary protein to be faulty or missing, gene transfer therapy can introduce a normal copy of the gene to recover the function of that protein and help your body system to function normally. But how did such a breakthrough in medicine come about? 

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The earliest studies that laid the foundation for gene therapy started with experiments on bacteria’s ability to transform. These experiments showed that moving genetic material from one organism to another would change how it behaved. Then in 1932, a scientist named J. Lionel Alloway discovered that he could take material from dangerous bacteria and add it to harmless bacteria, making the harmless bacteria become dangerous. This simple experiment demonstrated that genes are able to be transferred between living things and actually change their characteristics, which laid the basis for gene therapy. 

This idea was further expanded upon in 1972, when physician Theodore Friedmann and biochemist Richard Roblin wrote a paper on how DNA could be manipulated to help treat human diseases. However, they also warned of the potential consequences of using such methods until more studies were conducted. The first therapeutic use of gene transfer, as well as the first direct insertion of human DNA into the nuclear genome, was performed by American scientist William French Anderson in a trial starting in September 1990. However,  gene therapy progression wasn’t all pleasant and smooth. In fact, the field experienced a major setback in 1999 when 18 year old Jesse Gelsinger died during a gene therapy trial at the University of Pennsylvania.  Gelsinger had suffered a severe immune reaction to the adenovirus vector that was used to deliver genes and died four days after the trial. The news of Gelsinger’s death severely impacted gene therapy’s reputation in the public’s eye, leading to increased oversight and a halt in many gene therapy trials as researchers worked to improve safety. 

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From the early foundations of the craft to the groundbreaking and lifesaving treatments given today, gene therapy has evolved from a theoretical concept into a life-saving option.

Citations:

Boston Children's Hospital. (n.d.). 

Emily Whitehead Foundation. (n.d.). 

Malech, H. L. (2022). Evolution of gene therapy, historical perspective. 

Hematology/Oncology Clinics of North America, 36(4), 627-645.

MedlinePlus. (n.d.). How does gene therapy work? National Library of Medicine.

National Human Genome Research Institute. (n.d.). Gene therapy.

https://www.genome.gov/genetics-glossary/Gene-Therapy

U.S. Food and Drug Administration. (n.d.). What is gene therapy? 

https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-

gene-therapy

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