What is the main idea employed in gene therapy?

Answer 1

Gene therapy involves artificially transferring genes from one organism to another. Gene therapy has been suggested as a corrective therapy to correct medical conditions characterized by the deficiency of essential proteins (hormones, enzymes) in the body.

What gene therapy essentially does is to supplement the genome of the patient with genes that will then code for the missing proteins, thus restoring balance and curing the medical condition.

Although the approach seems fairly straight forward, researchers have encountered several obstacles (both scientific and ethical) along the way. Currently, gene therapy has taken a back seat with the advent of cell replacement therapy as an alternative cure for diseases like type 2 Diabetes.

Answer 2

In a recent news update, a team of Irish gene therapy researchers announced a breakthrough in the search for a cure for one of the worst strands of the incredibly painful skin disease, EB (epidermolysis bullosa). According to the researchers,the incredibly painful genetic condition, which affects approximately 300 people in Ireland, is caused by a fault in the gene that makes a vital protein responsible for binding our layers of skin together. This results in painful blisters – both internal and external from the slightest knock or bumps – for thousands of people worldwide.

Answer 3

to be able to replace or supplement the defective gene with a normal allele

Answer 4

Gene therapy is the replacement of a defective or missing gene, or the addition of a new gene, into a human's genome to treat a genetic disease.

Answer 5

defective genes in an organism's DNA are replaced or altered to attempt to correct a heritable disease.

Answer 6

Gene therapy.