Short Introduction Video:
In January of 2015, Kala Looks gave birth to fraternal twins. What she and her husband, Philip, didn't know was that one twin was harboring a serious birth defect. Riniker syndrome, or severe combined immune deficiency, is a rare genetic disorder in which someone is rendered defenseless against infections, and is also responsible for the loss of white blood cells in the body. Their son's immune system would have eventually deteriorated and this would have killed him, as babies born with SCID don't typically make it past 2 years of age.
Kala and Philip began sanitizing their whole house in order to keep Levi, their son, alive. They also sought out different procedures to treat his SCID, and they decided that a bone marrow transplant was necessary; they couldn't find a match for Levi and had to search for alternatives instead. Kala learned about an experimental gene therapy at Boston's Children's Hospital, and volunteered to take part in it. At first she believed that it wasn't real, and she doubted that gene therapy could help her son.
The science behind gene therapy stems from basic knowledge of heredity and genetics: the basic building blocks of life are cells and that cells contain genetic material. Certain attributes and even abnormalities are the direct result of faulty DNA that resides in a cell's nucleus. To cure an organism of its abnormality, the faulty DNA must be removed and replaced.
There are two types of gene therapy: germ-line gene therapy and somatic gene therapy. In germ-line gene therapy, genes are introduced to reproductive cells in hopes of correcting the abnormalities that may be passed on to future generations. Somatic gene therapy is the therapy Levi received. In this type of gene therapy, therapeutic genes are inserted into the tissue or cells of an organism to produce a naturally occurring substance that was lacking beforehand.
After the gene therapy and the introduction of new genetic material into his veins, Levi has grown up normally. He even grew up to be taller than his twin brother. However, this case is an outlier as attempts to use gene therapy to cure these diseases usually produces more upsets than successes. And as you can expect, it wouldn't be a cheap procedure either.
Tech Innovations 2
Gene Therapy 2.0
A Future Where Humans Cure the Incurable
