Navigate Exhibit by Clicking here Genes and Drugs, Vaccines, and Enzyme Replacement Therapy
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Vaccines on a New Frontier


Vaccines expose you to the proteins of bacteria and viruses that cause disease. The vaccine stimulates an immune response "memory." This means that if you have been vaccinated against a disease and then are exposed to the disease, your body will fight the disease faster and better. To work, the vaccine must stimulate your immune system without making you sick. Photo of a child getting vaccinated - Courtesy of the National Institute for Diabetes and Digestive and Kidney Diseases
  Child getting vaccinated. Courtesy of the National Institute for Diabetes and Digestive and Kidney Diseases
 
Children receive vaccines to protect them from many dangerous and deadly diseases like whooping cough, mumps, measles, and diptheria. Scientists are working vaccines which will help to prevent several illnesses, including one of the most common in children -- ear infections. Adults and children can get a vaccine against the influenza virus every autumn. The influenza virus causes the flu. Recently, the National Institute of Allergy and Infectious Diseases (NIAID) ran a trial with a new influenza vaccine created with recombinant DNA. The recombinant DNA influenza vaccine caused fewer side effects and could be used at higher doses. Because the vaccine is not made using chicken eggs -- the way most viral vaccines are made -- people with egg allergies can take it.


Enzyme Replacement Therapy and Gaucher Disease
The 14-year old girl in this photo has Gaucher Disease (GD). GD is a genetic disease that affects the storage of fats in the body. A fatty molecule called "glucocerebroside" is normally broken down in the body by the enzyme "glucoerebrosidase." A mutation in the gene for the enzyme glucocerebrosidase, causes the enzyme to be less active than normal. Because it is not broken down, the fat accumulates in the spleen, liver, and bone marrow of the person with GD. People with GD suffer anemia, bone damage, swelling of the liver and spleen, and sometimes neurological damage. Photo of girl with Gaucher Disease - Courtesy of Dr. Roscoe Brady, National Institute of Neurological Disorders and Stroke
Girl with Gaucher Disease. Courtesy of Dr. Roscoe Brady, National Institute of Neurological Disorders and Stroke

Photo of girl at 17 with Dr. Roscoe Brady - Courtesy of Dr. Roscoe Brady, National Institute of Neurological Disorders and Stroke
Girl at 17 with Dr. Roscoe Brady. Courtesy of Dr. Roscoe Brady, National Institute of Neurological Disorders and Stroke
This is the same girl, after she received enzyme replacement therapy. She is standing with Dr. Roscoe Brady of the National Institute of Neurological Disorders and Stroke (NINDS). In 1991, twelve people with Gaucher Disease received the purified enzyme glucocerebrosidase, which had been isolated from human placental tissue. All responded dramatically. Dr. Roscoe Brady’s team had devoted 17 years to perfecting this therapy, in which the impaired enzyme was simply replaced. In 1995, a recombinant version of the enzyme was developed, which is as effective as the placental preparation used previously.

More information on Dr. Brady's work on Gaucher Disease please go to:
"Researching Disease: Dr. Roscoe Brady & Gaucher Disease".


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Revolution in Progress: Human Genetics and Medical Research/
National Institutes of Health