COUNCIL CHRONICLE – In a medical first, doctors are trying an experimental gene editing treatment that could cure a man who was born with Hunter Syndrome. The 44 years-old man is now undergoing an in-body gene editing treatment that might help him get rid of the incurable disease.
On Monday, November 13, Brian Madeux received the first part of the treatment. This is a medical first since no doctor had previously attempted to use in-body gene-editing. Brian Madeux, the patient, mentioned that this revolutionary treatment doesn’t look as complicated as he initially believed it to be.
In order to start the treatment, the patient was hooked to an IV. This delivered a gene editor into his blood as well as other copies of a corrected gene. These latter are the ones that are going to replace the mutation Brian carries. Dr. Edward Conner, the maker of this new therapy, mentioned that the infusion only takes 2 or 3 hours. He also added that both he and his fellow colleagues are very hopeful.
Hunter Syndrome and How a Gene Editing Treatment Might Fix It
People who suffer from Hunter syndrome are missing an enzyme that is responsible for breaking down mucopolysaccharides. These are sugar molecules which are used by the body to build tissues. This enzyme is very important because the tissues it creates are for the heart, the brain, and other vital organs.
Doctor Connors mentioned that the cell needs to be seen like a house in which trash accumulates. Enzymes are the ones that clean out the cell and help it build new tissues. When a person is born with Hunter’s, the tissues from their organs start accumulating that ‘trash’.
Some of this condition’s most common symptoms include joint malformations, heart problems, breathing difficulties, and hearing problems. The only treatment for Hunter syndrome consists of enzyme replacement therapies. Even the patients who receive this therapy still have progressive damage to their bones, lungs, and hearts.
If the editing gene treatment is successful, this might be the only currently available chance for people with Hunter syndrome to get cured of the disease.
The gene editing treatment is in its first trial, and Madeux is the first person to receive it. This gene editing treatment may have a chance to be approved by the FDA if this on-going trial returns positive results.
Image Source: Pixabay