I have two grandchildren in college. I have told them that working on genes and gene therapy is the future of medicine.
My grandson is entering his third year in biomedical engineering. My granddaughter is entering her second year in genetics.
Because of that I was pleasantly surprised by a headline I saw recently. The headline involved the genetic treatment of sickle cell disease.
There are many conditions similar to sickle cell disease. A gene in the body is missing, which means a certain protein cannot be made. This results in a medical problem.
In sickle cell disease it is a protein that makes red blood cells. People with sickle cell disease make brittle blood cells that are easily destroyed. The complications are many.
The sickle cell gene is a very common one. That is related to evolution. A gene missing on only one chromosome produces the sickle cell trait. This is protective against malaria. Someone with the sickle cell trait can survive better in areas where malaria is common. That is why over the course of history the sickle cell trait became common in those areas.
When the gene is missing on both chromosomes, it causes sickle cell disease. Since it involves our genetic structure, there is no treatment.
However, science has changed that recently through a new technology called CRISPR – clustered regularly interspaced short palindromic repeats. Try saying that three times fast.
They are pieces of a chromosome. The piece may contain a gene. That gene can then produce the desired protein. An enzyme is used to separate that piece of gene from a full chromosome.
In the recent case, researchers took bone marrow cells from the patient. They then used the CRISPR technology to attach the missing gene to those cells. The gene will be able to create the missing protein. They then gave the cells back to the patient.
It is too early to tell if it will work. It is also too early to tell how long it will take to work and whether or not there will be complications.
This is only the first step in what will be a future that will focus on gene therapy for the group of disorders that occur because of a missing protein.