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$2.5 million Defense Department grant funds gene therapy study for Lou Gehrig's disease
Date:4/1/2014

argely because the protein does not readily penetrate into the spinal cord. Regenerative Medicine Institute scientists previously showed that spinal transplantation of stem cells that were engineered to produce GDNF increased motor neuron survival, but this had no functional benefit because it did not prevent nerve cell deterioration at a critical site, the "neuromuscular junction" the point where nerve fibers connect with muscle fibers to stimulate muscle action.

Masatoshi Suzuki, PhD, DVM, assistant professor of comparative biosciences at the University of Wisconsin, Madison, who previously worked in the Svendsen Laboratory and remains a close collaborator, recently found that stem cells derived from human bone marrow and engineered to produce GDNF protected nerve cells, improved motor function and increased lifespan when transplanted into muscle groups of a rat model of ALS.

"It seems clear that GDNF has potent neuroprotective effects on motor neuron function when the protein is delivered at the level of the muscle, regardless of the delivery method. We think GDNF will be able to help maintain these connections in patients and thereby keep the motor neuron network functional," Suzuki said.

Svendsen's team now plans to directly deliver GDNF into muscle cells with AAV5, a "viral vector" a virus stripped of its harmful genes and engineered to carry a beneficial gene into targeted cells. This will be supplied by uniQure, a biotechnology company that is collaborating with several research centers to conduct gene therapy clinical trials targeting other diseases.

In the new lab rat studies, AAV5 will be engineered to deliver GDNF into muscle tissue of the leg and the diaphragm, addressing two major aspects of ALS.

"The disease's effects typically start in one limb, beginning with weakness and leading to paralysis, before moving to other limbs and to muscles throughout the body. The most common cause of death is respira
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Contact: Sandy Van
sandy@prpacific.com
808-526-1708
Cedars-Sinai Medical Center
Source:Eurekalert

Page: 1 2 3

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