Gene replacement therapy, a type of gene therapy, is a potential way to treat genetic diseases. Gene replacement therapy addresses the root or underlying cause of a disease by replacing a missing or nonworking gene with a new, working copy of the gene.
Gene replacement therapy aims to give the body a new, working copy of a missing or nonworking gene. The new gene sits inside the nucleus, or control center, of cells and allows the cells to produce the missing proteins that are critical for the body to function. Learn more
All therapies are required to undergo testing in clinical studies to be approved by the Food and Drug Administration (FDA). All clinical studies, including those being conducted in gene replacement therapy, are monitored for safety and effectiveness of a treatment. The clinical study team works closely with the FDA to monitor potential risks both during and after its clinical studies.
Viruses are used in gene replacement therapy because of their natural ability to enter into the cells of the body. In gene replacement therapy, scientists alter or reengineer a virus so it can be used as a vector, or delivery vehicle, without causing disease in humans. The vector acts like an envelope in that it holds the new, working copy of the gene and delivers it to where it needs to go in the body. Once the new gene has been delivered to cells, the rest of the vector, including the viral shell (the delivery vehicle), is broken down and removed from the body. Learn more
A monogenic disease can be caused by one missing or nonworking gene or gene pair. Because gene replacement therapy delivers a new, working gene to the body, it has the potential to help people with monogenic diseases. Learn more
While certain viruses are used as vectors, the vehicles used to carry the new, working genes into the nucleus of the cell, scientists have changed them in ways that stop them from making you sick. Even though the disease-causing parts of the virus have been removed, as with all therapies, there can be risks, so be sure to talk to your doctor before starting a new therapy. Learn more
Most of the risks of gene replacement therapy have to do with the viral vectors used to deliver the new gene into the cell. Long-term risks are not known. The general risks listed below may not apply to all gene-based therapies, but may include:
There are several different types of gene-based therapies. Gene replacement therapy addresses the root or underlying cause of a disease by replacing the function of a missing or nonworking gene with a new, working copy of the gene. Other types of gene-based therapies may insert, remove, change, or replace specific pieces of a person’s existing DNA or change how certain cells act. Learn more
Gene replacement therapy is a relatively new type of treatment. The first gene replacement therapy was approved by the FDA in late 2017. While clinical trials often last years and may demonstrate that a therapy is safe and effective during that time, it is difficult to know exactly how long gene replacement therapy will last for each and every person. The goal of gene replacement therapy is the long-term management of a genetic disease. However, because there are many other factors that may contribute to how long the benefits of a treatment last, this is an ongoing area of interest for scientists.
Gene editing enables scientists to add, remove, or replace part of person’s DNA at a specific spot along their DNA. At this time, there are no FDA-approved gene-editing therapies, and clinical trials have only recently started to investigate the effectiveness and safety in humans. There are many questions still to be answered, including the potential risk of editing the wrong segment of DNA, which could lead to the loss of working genes or increase the risk of developing certain types of cancer. Learn more
CAR-T stands for chimeric antigen receptor (CAR) T-cell therapy. It is a type of cell therapy that is used with gene therapy but it is not a type of gene therapy by itself. It involves changing a person’s own immune cells to recognize and fight cancer cells inside the body. At this time, the FDA has approved 2 CAR-T cell therapies, with others being investigated. Learn more
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