It's like the garbage man of the cell is not able to do its job
The trash keeps getting backed up inside the cell until the cells can no longer function properly and then eventually die throughout the entire brain.
The virus is used as a Trojan horse that houses and then delivers a healthy, functional gene into the cells of the brain ... The genes are incorporated within the genetic material of the cells, which are then able to produce a protein that is deficient in Batten disease.
Before now, we had no hope of a therapy for Batten disease, but today, we can say that there is some hope ... These results are not just promising for sufferers of the disease, but suggest that gene therapy can work and should be studied for other neurological disorders. Each gene in our body has the potential to become a target to study for human disease.
The primary variable was a clinical rating scale based on the number of seizures, language skills, motor skills and so on
This is potentially exciting, but it's also potentially not going to hold up
Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis with CNS Administration of a Serotype 2 Adeno-associated virus expressing the CLN2 cDNA
This clinical trial is an important step toward the development of treatments for this group of underserved inherited neurodegenerative disorders
You can't give back more brain cells. You can't improve it. All you can hope to do is to delay progression