Gene Replacement Therapy
one-time "Forever Fix"
What is a Gene?
Most of us have heard the word "gene". We are made of genes and collectively they make us what we are. Half of your genes come from your mother, and the other half come from your father.
Genes have different functions in our body:
Some genes encode proteins that make something in a body, like skin, bones, blood and other structures.
Next, genes can encode proteins that help direct the body to do something. It can be muscles flexing, hair growing or digesting food.
Other genes encode proteins that support bodily functions in an indirect role, like helping the immune system to respond to a specific area in the body, or to assist blood flow through our circulatory system.
Genes can also directly influence our personal characteristics, or traits! Hair color, eye color, skin tone and more, can be directly coded into our genes.
Changes in genes
As always, changes can be good and bad. We have different color eyes, different shoe size, personality traits, etc. that make us who we are thanks to changes in genes from one person to another. Changes (inborn or acquired) in other genes make us have certain diseases like breast cancer, high cholesterol or blindness.
One of the options to cure these type of diseases is to go after the gene, trying to replace or alter it.
How gene therapy can help cure AGU?
What is Gene Replacement Therapy?
Disease can be present from birth when someone is born with a defective or missing gene. Alternatively, someone can develop a disease during adult life due to a gene mutation, different expression or change, which can disrupt correct protein production.
Gene therapy is an innovative process that involves the introduction of a gene into cells. The new gene can produce the missing enzyme or protein, restoring function of malfunction gene. The new gene is inserted into a patient's cells through a vector, which is genetically engineered to be able to carry and deliver the gene into cells. If the gene injection is successful, the new gene can replace a disease-causing gene, help the body fight a disease, or turn off a gene causing problems. Therefore, this method can be used as an alternative to drugs and/or surgery, in order to treat, cure, or even prevent different diseases and medical conditions.
The gene called AGA is not working in a body of a child who has AGU. This means that their body is not producing an important cellular enzyme. Gene replacement therapy (GT) can be used to deliver the enzyme to the human body. It is a long and expensive process to develop a successful treatment. While working on GT, we funded the development of a Chaperone therapy. This keeps the children with AGU healthy and stops the progression of disease deterioration.