Question

Lipoprotein lipase deficiency (LPLD)is a genetic disorder in which a person has a defective gene for lipase. This leads to high triglycerides, stomach pain, and fat deposits under the skin. It may eventually affect the liver, pancreas and may also cause diabetes. The disorder occurs if a child acquires defective genes from both parents (autosomal recessive). ERT (enzyme replacement treatment) is one of the treatments offered to patients with LPLD.

1. 1. What procedure is followed in ERT?
   2. What could be one possible drawback of ERT?
2. How can LPLD be treated using Biotechnology? Elaborate.

Answer 1

1. 1. Functional enzyme lipase is given to the patient by injection.
   2. This procedure is not completely curative.
2. 1. The disease can be treated by using Gene therapy. 
   2. Gene therapy is a collection of methods that allows the correction of a gene defect that has been diagnosed in a child/embryo.
   3. Here, genes are inserted into a person’s cells and tissues to treat a disease. Correction of a genetic defect involves the delivery of a normal gene into the individual or embryo to take over the function of and compensate for the non-functional gene.