Wednesday, March 28, 2012

Possible Cure to Red Blood Cell Disorders

Just recently a group of scientists tested a type of gene therapy that can put an end to sickle cell anemia and beta thalassemia. The newly developed gene has a healthy beta globin gene that promotes a cell's hemoglobin production. With this new gene, the cells that were lacking this beta globin gene or have a non functioning can now hope to produce hemoglobin. And this type of therapy would bear few risks due to the fact that this new gene can be tested on a blood sample before it's actually given to you. By giving these new cells a functioning beta globin gene this can lead to the cure of beta thalassemia. The success rate in a process of gene therapy can be quite difficult though because when the gene is injected it will randomly place itself, meaning the gene could not be expressed, so an insulator is made to lure the gene into the genome area increasing the success rate of gene therapy.

With these new findings maybe it can be possible to master gene therapy and be able to replace any dysfunctional gene with a functional one. This can lead to the cure of many genetic disorders such as PKU.

Posted by Khoa Chu (A)


  1. The production of erythrocytes stems from red bone marrow so would this gene adjustment be a modification to the marrow its self? Or would this process be a series of injections that would be required over and over? If the gene could be implanted at the source, the production of the beneficial gene would be perpetual. That would eliminate the need for costly shots and medications that would have the same effect.

    Jeff Keating (2)

    1. Good point. Blood transfusions are almost like a gene therapy at this point, introducing healthy blood cells based on their genetic makeup....i.e. type of blood (A, B, O, AB and the fact that the blood transfused isn't blood from someone with a blood disease or other pathogens), so how would this gene therapy differ? I'm curious as well.

      Karen Melendez

  2. If they can figure out greater success rates, this sounds like a great treatment for those who have the disease. I think it is interesting how they can test the blood sample before actually giving it to you, so they know how you will react.

    Taylor Pirog (2)

  3. i just learned about sickle cell anemia in physiology. Apparently carriers where a evolutionary adaption to parasites in Africa. These parasites gave malaria, but they couldn't effect a person who carried to sickle cell. So they were protected from malaria, and not effected by being homozygous recessive. Still it's a nasty disease in a lot of other parts of the world. This treament might the right step.

    posted by Dorian Pillari(c)

  4. Very interesting! With further research increasing the success rate this procedure could be great curing so many people affected by these diseases. Hopefully with time the gene pleacement will become much easier and more efficient leading to the cure of additional genetic diseases.

  5. Sounds awesome! Transgenics is such a cool weird field, but unfortunately gets a bad rep for the whole genetically modified food disaster. If transgenics were mastered we would be able to create almost anything we want. Humanity playing god with food, animals, and ourselves would solve so many problems. Hopefully we get these methods down soon.

    Mike Selden (C)