gene therapy tagged posts

Last time, I wrote about how gene therapy is being used to fix certain kinds of errors in DNA, and so cure or significantly reduce certain kinds of cancers. I asked whether you would accept such a treatment for yourself.

It’s one thing to accept the risks that are associated with these still new and experimental treatments, but would you make that decision for a sick child—one of your children?

© Suthisa Kaewkajang | Dreamstime.com

Current Treatments

I didn’t make a point of it last time, but two of the diseases for which this kind of gene therapy is now available, B-cell acute lymphoblastic leukemia (ALL) and junctional epidermolysis bullosa (“butterfly skin”), are both childhood diseases...

Late last year, Science News magazine published an article about how scientists used genetic modification to save the life of a young boy. He had a skin condition in which the upper layer of his skin would blister and separate from the layers underneath if he was just touched or rubbed gently. Children with this condition are called “butterfly children” because their skin “is as fragile as butterfly wings.” At the time of his treatment, the boy had lost 80% of his skin and was close to death.

Photo by Andrianocz, via Dreamstime.com

People with this condition have a mutation in one of three genes. The doctors identified which one was the problem for this child, took a section of good skin, and used a retrovirus to insert good copies of the bad gene into the skin cells...