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A LOCAL RESEARCHER HELPS
FIND A CURE FOR A RARE DISEASE.
GENE THERAPY IS THE KEY
THAT UNLOCKED TREATMENT FOR A
DISORDER THAT ATTACKS THE
PANCREAS.
MARSHA BONHART TELLS US WHY
THIS IS SUCH A MEDICAL
BREAKTHROUGH...
NAT SOT THIS IS A LABORATORY
AT WRIGHT STATE UNIVERSITY.
THIS IS WHERE RESEARCH IS
HELPING PEOPLE WITH DISORDERS
LIVE A BETTER QUALITY OF LIFE.
NAT SOT W STUDENT IN LAB DR.
KATE EXCOFFON HAS BEEN WORKING
TO HELP SOME PATIENTS WHO HAVE
A RARE GASTROINTESTINAL
DISEASE THAT ATTACKS THE
PANCREAS.03:08"...THIS DISEASE
IS CALLED LIPOPROTEIN LIPASE.
SO ALL THE FAT WE TAKE IN,
WHETHER MCDONALD'S OR
SOMEWHERE ELSE, COMES IN
INTESTINE AND FATTY PARTICLES
ENTER BLOOD STREAM AND HAVE TO
BE DIGESTED...03:24 BECAUSE
THESE PATIENTS DON'T HAVE THE
GENE THAT DIGESTS FAT, DR.
EXCOFFON FOUND A WAY, THROUGH
GENE THERAPY, TO MAKE A
HEALTHY GENETIC COPY.SHE USES
A COMMON VIRUS, PLACES THE
GENE INSIDE AND INSERTS THAT
INTO MUSCLE TISSUE. ONCE
INSIDE THE MUSCLE CELLS, THE
PROTEIN IS CREATED. THAT
PROCESS DEVELOPED A DRUG THAT
HAS BEEN APPROVED IN EUROPE.
10:51"...I AM HOPING U-S, FDA,
WILL COME QUICKLY AND BE
AVAILABLE IN 5 YRS OR
SO..."11:02 PROVIDING LIFE
SAVING THERAPIES FOR PATIENTS
WITH ALL KINDS OF GENETIC
DISEASES. AT WRIGHT STATE
UNIVERSITY, MARSHA BONHART,
2NEWS.