Gene Therapy

DNA in Medicine

I got the idea for gene therapy from the book. It said "gene therapy", I read about one sentence and thought it sounded interesting.

1. Searched "gene therapy"

2. o4uiovzQBoCD9rw_wcB

3. Here I found a program for the study of neurodevelopment in rare disorders, or the NDRD. Here's some things about them:

1. They help raise awareness and give an understanding of these rare diseases

2. They've had patients from 47 of 50 states and from 27 different countries

3. Their goal is to improve the quality of kids with these diseases through education, research and clinical interventions

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Well that first website wasn't much help....

1. Stayed on the website and searched "gene therapy" again


3. JDRF is looking at gene therapy for type 1 diabetes. They'd use it to improve islet cell transplantation for insulin replacement and neurological complications. The goal of this is to have a cure to diabetes.

1. Searched "What is gene therapy"


3. Gene therapy is a relatively new and experimental technique to treat or even prevent diseases. Researchers are using three different methods to gene therapy:

1. Replacement: replacing the mutated gene with a healthy one

2. "Knocking out" the mutated gene

3. Introducing a new gene into the body to help fight the disease

The main diseases people are using gene therapy for are diseases that have no other cures. This is because it's new and still considered risky. There is testing and experiments going on to make sure the procedure is safe and effective.

1. Searched "gene therapy for type 1 diabetes"


3. So, this is still experimental. Researchers are currently using diabetic rats. So far, they have injected the rats with an insulin producing gene. The gene works and even the researchers are surprised. Unfortunately, it only lasts for up to six weeks. If you think about it though once every six weeks is much better than every day. A weird thing about this is that diabetes affects the pancreas, but the therapy targets liver cells. Researchers chose to do it this way because of the livers regenerating ability. This therapy has also kept rats free from side effects of type 1 diabetes if the disease is not properly cared for like blindness, kidney failure and amputations. Dr. Hans Sollinger and his colleagues have named the injection Insulete, for insulin obsolete. His current research is to get the injection time from 6 weeks to 6 months.

4. A couple statistics:

A: 1-1.5 million people in the US have been diagnosed with T1DM

B: It costs the US $8-14 billion each year to care for these people