Gene therapy: Editing the genome to prevent and treat genetic disease

Scientists have been working on gene therapies where they edit the person’s gene inside the body to permanently change a person’s DNA to cure or prevent diseases. Gene editing could be done through insertion, deletion or replacement of DNA at a specific part of the genome of a cell (Horizon Discovery). This method requires precision; it is very similar to sending a mini surgeon to place a new gene in a specific part of the body. The gene stays inside the patient’s body for the rest of their life.

Genome editing have the potential to treat disease with a genomic basis, like cystic fibrosis and diabetes. The risks are not fully known, but to many doctors and researchers, this method is a big step for medicine because it gives a huge potential in removing and healing incurable diseases.

There are protections in place to help ensure the safety of this procedure. According to Dr. Howard Kaufman, a Boston scientist on the National Institutes of Health panel, stated that there’s been no evidence that this procedure is going to be dangerous (Associated Press).

How the Procedure Works

There are two types of gene therapies. Germline therapy change DNA in reproductive cells and is passed down from generation to generation. Somatic therapies, on the other hand, target the non-reproductive cells and the changes only affect the person who receives the gene therapy.

Doctors use a gene-editing tool to cut a specific piece of DNA. The new gene is placed in an altered virus which doesn’t cause infection to the body, but transports them into cells. Once it has been transported to the cells, billions of copies are then given through the vein. Once the new gene slips in, it directs the cell to make the enzyme the patient lacked (Associated Press).

Issues with the Procedure

Despite seeing the gene therapy procedure to be safer compared to several other procedures, there are some issues that doctors and researchers are concerned about such as safety. On a study done in 1999, a patient died in a gene therapy study from the virus causing an immune system attack.

Another issue was on the insertion of a new gene which may cause unforeseen effects on the other genes. This happened once when researchers used gene therapy to cure cases of an immune system disorder called “bubble boy” disease. Several occasions later the patient developed leukemia because the new gene unintentionally activated a cancer gene.

Technical Barriers & Ethical Concerns

These treatments are still experimental because there are still technical barriers and ethical concerns surrounding genome editing. At this stage, these treatments are still not perfect. If a genome editing tool cuts in the wrong spot, scientists are still not sure how these errors might affect the patients.

There are also ethical concerns that may emerge with the procedure, especially around human germline editing because changes made would be passed down to future generations.

Bioethicists and researchers believe that human genome editing for reproductive purposes should not be attempted anytime soon, but the other studies that show gene therapy as safe and effective should continue. But stakeholders agree that there should be continuous deliberation and debate with the public to decide whether or not germline editing should be allowed.

Another issue is on informed consent. Some people worry that it’s difficult to get consent especially for germline therapy because the patients affected by the edits are the embryo and the future generations.

There is also the gap between the rich and the poor, and as with many technologies, the concern is the accessibility of the genome editing. If it turns out to be expensive, it will only be accessible to the wealthy and increase disparities in access to health care between the rich and the poor.


It is important that the safety of gene therapies is improved to ensure that this technology is ready for use in patients. Until genome editing is said to be safe, more research needs to be done to ensure that if not most, but all safety concerns are addressed. Until then, it should not be used for clinical reproductive purposes as the benefit cannot be justified by the amount of risks.

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