Gene therapy has been there for decades. The idea is beautiful, one single treatment to get rid of horrific diseases forever. Before gene therapy was introduced diseases such as severe combined immune deficiency or SCID could only be treated with bone marrow transplant, which is a very pricey and delicate solution. Recently gene therapy has proven to work on this dreadful disease.
A three months old baby named Levi was diagnosed with SCID. This is a disease that causes immune cells in blood to gradually go from low to total loss. The patient's body is in an extremely vulnerable state and even a small infection can be fatal. Levi's parents were waiting for a match for bone marrow transplant, when his mother found out about an experimental treatment at Boston Children’s hospital involving gene therapy. The treatment consists of an infusion into the baby's body containing a virus that essentially finds the deficiency in the genes and “repairs" it. The therapy turned Levi into a perfectly healthy kid.
The CRISPR treatment (a method of gene therapy) is claimed to be cheap and very stable. While gene treatment can already handle up to 200 different disease, this is merely the beginning. In the US, fertility facilities already put gene therapy (PGD) of an embryo prior to transfer into a womb onto a price list. The list of diseases they are able to treat is long, but the prices of the procedure are less heart-warming.
Does this mean we will be able to cure cancer? Maybe. At the moment gene therapy covers diseases that are controlled by deficiencies in one gene, such as SCID. But Alzheimer's, diabetes and heart failures are harder to tackle, since these conditions are linked to multiple genes. And the exact genes involved can vary from person to person. For now, let's cheer for little Levi and the fact this therapy brought hope to more families.
Source: MIT Technology Review