Sangeetha Pulapaka

Gene therapy which was first conceived in the mid 1970's aims to insert working copies of genes into cells containing dysfunctional ones, It treats, cures and ultimately prevents disease by changing the expression of a person's genes

Human Genome Project: A genome is all the DNA in an organism, including its genes. Genes carry information for making all the proteins required by all organisms. Variations in structure of person's genes collectively helps define us as individuals.

Which cells are targeted?

Somatic geme therapy the recipitent's genome is changed, but the change is not passed along to the next generation. Gremile gene therapy, the sex cells are changed with the goal of passing these changes to their offspring. But, this is not being actively investigated, at least not in humans, although a lot of discussion is being conducted about its valud and desirability.

Risks with GT trials:

  1. Viruses can usually infect more than one type of cell
  2. When DNA is injected directly into a tumor, ther is a slight chance that this DNA could unintentionally be introduced into reproductive cells
  3. Other concerns include the possibility that transferred genes could be overexpressed


Gene therapy is still in the experimental stage. Scientists know little about function of thousands of genes. But still, this primarily helps in addressiong diseases such as sickle cell anemia.

Also, high costs associated with developing this technology and regulations that needed to be implied on it with experimentations are great hurdles for experiments in this field.

Researchers are working to improve the body's natual ability to fight the disease or to make the cancer cells more sensitive to other kinds of treatment such as chemotherapy. In this, injection of cancer cells with a gene that makes them more sensitive to treatment with an anticancer drug. Also the MDR gene is used to make the stem cells more resistant to the side effects of the high doses of anticancer drugs.

Not only Gene therapy help with cancer, it helps in fighting the world's other powerful disease AIDS.

For more than a decade, the strongest aids drugs could not fully control Matt Chappell's HIV infection. Now his body controls it by itself, and researchers are trying to perfect the gene editing that made this possible. 

Scientists removed some of his blood cells, disabled a gene to help them resist HIV, and returned these “edited” cells to him in 2014. So far, it has given the San Francisco man the next best thing to a cure. 

“I've been off medications for three and a half years,” he said. He even was able to keep the virus in check despite cancer treatments last year that taxed his immune system.