Group

8
Gene Therapy for
chronic pain
WHEN: January 28, 2008
WHO: Andreas Beautler and
researchers at Mount Sinai
School of Medicine
The pain gate
This could bring relief to patients
suffering from chronic pain while
bypassing many of the debilitating
side effects associated with
traditional painkillers.
They injected a virus carrying the
gene for an endogenous opioid
directly into the spinal fluid of
rats.
It was targeted to the dorsal
root ganglia.
AAV- adeno associated virus
It creates pain relief without the
side effects such as constipation,
nausea, sedation and decreased
mental acuity.
It lasted as long as three months
from the single injection.
The technique effectively
restored the rats to a normal
level of pain sensitivity.
This approach could be tested
on humans in as few as three
years.
Gene Therapy for
Alcoholics
WHEN: January 09, 2008
WHO: Yedy Israel and his
colleagues at the University
of Chile
Alcoholic animals:
Researchers in Chile have
used gene therapy to
curb drinking in alcoholic
rats.
This therapy curbed the
activity in the liver of an
enzyme ALDEHYDE
DEHYDROGENASE.
It make the patient sick while
they drink.
The researchers started with
rats bred for their alcoholic
tendencies and offered them
limited quantities of diluted
ethanol.
Cut off animal access the animal
access to alcohol and injected a
virus containing a gene.
Those rats drink half as
much, on the average, as the
untreated animals.
The effect lasted throughout
the monthlong study,
If this gene therapy could be
applied to humans , it may be
a valuable addition to the
drugs and behavioral
approaches used to treat
alcoholism.
Gene therapy 'hope' on
impotence
 When is it discovered: June 3, 2007.
 Who discovered it: Scientists at
Wake Forest University, with the help
of Dr. Arnold Melman
 Who will benefit from it : Those who
are suffering from erectile
dysfunction or impotence.
 For erectile dysfunction.
 Treatment is well-tolerated
 Delivery-injection into the penis.
 The Wake Forest therapy works by
inserting small pieces of DNA into
cells to trigger the production of
proteins which, in turn, help smooth
muscle cells relax.
Gene Therapy Stops
Gum Disease
When it was discovered: December 17,
2008
Who discovered it:
Scientists at the University of Michigan
Who will benefit from it: People with
rheumatoid arthritis and gum disease
 used an inactivated virus to produce
more of a naturally-produced molecule
soluble TNF (tumor necrosis factor)
receptor
 Gene therapy can be used to
successfully stop the development of
periodontal disease, the leading cause
of tooth loss in adults.
Gene therapy to
grow new hair
cells and restore
hearing
When it was tested: February 14,
2005
Who discovered it:
Yehoash Raphael, Ph.D.
Masahiko Izumikawa, M.D.
Who will benefit from it: The future
researchers that will try to put this
gene therapy to humans.
Atoh1 Gene
- a key regulator of auditory
hair cell development
- pro-hair cell gene