Gene therapy facts for kids
Gene therapy is a special way to treat diseases by changing a person's genes. Imagine your body's instructions (genes) have a typo or a missing part. Gene therapy tries to fix that problem or add the missing instruction.
For example, a treatment called Glybera was approved in Europe. It helps people who have trouble breaking down fats because of a damaged gene. When fat builds up in their blood, it can cause a lot of pain and serious health issues. Before this therapy, the only way to manage it was a very strict low-fat diet. Glybera uses a virus to put a working copy of the gene into muscle cells. This helps the body break down fats properly.
When a virus is used this way, it's called a vector. Think of it as a tiny delivery truck. The new gene is put inside the virus, and the virus then carries and delivers it into human cells. This process is known as transfection.
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Gene Therapy Around the World
China's First Approval
China was the first country to approve a gene therapy for sale. This happened in 2003. The therapy is used to treat a type of cancer that affects the head and neck. This cancer is quite common in China.
The gene therapy sold in China is called Gendicine. Like Glybera, it also uses a virus to carry a new gene into human cells.
The History of Gene Therapy
Scientists started talking about the idea of gene therapy a long time ago. This was as soon as they understood how DNA and RNA work. The first detailed paper about it was published in 1972.
The Future of Gene Therapy
Many more types of gene therapy are currently being tested. Scientists believe that in the future, doctors will often:
- Look closely at a person's DNA to find broken genes.
- Then, they will use gene therapy to fix those genes.
Sometimes, new treatments can have side effects. These are usually found during the testing stage. There is now a special online list that shows all the gene therapy tests happening around the world.
Images for kids
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A duplex of crRNA and tracrRNA acts as guide RNA to introduce a specifically located gene modification based on the RNA 5' upstream of the crRNA. Cas9 binds the tracrRNA and needs a DNA binding sequence (5'NGG3'), which is called protospacer adjacent motif (PAM). After binding, Cas9 introduces a DNA double strand break, which is then followed by gene modification via homologous recombination (HDR) or non-homologous end joining (NHEJ).
See also
In Spanish: Terapia génica para niños
