Researcher Profiles – Dr. Jagdeep Walia

Researcher Profiles – Dr. Jagdeep Walia


My focus is on developing gene therapeutics for Neurodegenerative conditions and other genetic conditions The group of disorders in which I am working is called GM2 general psychosis. The example of the disease is tay-sachs disease and Sandhoff disease It is hereditary that means that The parents have to be carriers, and there is 1 in 4 chance of them having a child affected with this condition There is no treatment for these crypto disorders. So mostly these children: They are born, they regress and they die. What happens is that there is a deficiency of an enzyme which is used to break down This lipid called GM2 ganglioside. When the enzyme is missing the GM2 ganglioside starts accumulating in the brain. Once it reaches the toxic level it kills that cell, so since this lipid is mostly in the brain it Starts killing the brain cells. When a child is born the child is usually normal to appear. But within six months, the levels get accumulated very much to the level that the brain cells start dying, the child starts showing the signs of regression. Our strategy is that using vehicles or some kind of transport mechanism called viral vectors We give that gene which is not working Back to the body especially in the brain and the brain starts making that enzyme. It was very difficult before to take this enzyme and get it transported to the brain because there’s a blood-brain barrier Fortunately we got a viral vector called AV9 which is a kind of virus which can cause common cold, but it doesn’t cause any major disease to humans so that vector can take the gene into the brain and The cells will start making the protein It is a very new, cutting edge field. Trying to find the cure for genetic conditions that have no cure. I think genetics is going to have its role in almost every field of medicine or health care provision. People have been working for almost 10 years 10 maybe 15 years But we haven’t found a cure yet. if I can find a very long term treatment for Tay-sachs and Sandhoff group of disorders. I would say that we have achieved something

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