Novel therapies such as gene therapy represent an opportunity to fix
the faulty gene responsible and allow these children the chance to have a normal life.
Cinnamon's genetic layout — published in Genome Research — has already helped pin down
the faulty gene responsible for blindness caused by retinitis pigmentosa, a hereditary disease that causes blindness in kitties and in one in of every 3,500 people.
A therapy that replaces
the faulty gene responsible for cystic fibrosis in patients» lungs has produced encouraging results in a major UK trial.
Not exact matches
The treatment (which involves injecting people with modified viruses carrying the CRISPR technology that will go in and repair the
faulty DNA) can be injected directly into the retina and, in this case, used to delete the portion of the CEP290
gene that's
responsible for the disease.
Charlie inherited the
faulty RRM2B
gene from his parents, affecting the cells
responsible for energy production and respiration and leaving him unable to move or breathe without a ventilator.
Diabetes researchers have debated for years whether errant
genes or
faulty biology are
responsible for the disease.