West Penn Allegheny Health System Scientists Explore Novel Approach to Gene Therapy for Sjögren’s Syndrome

Scientists at West Penn Allegheny Health System’s Allegheny Singer Research Institute (ASRI) have been awarded a grant from the Sjögren’s Syndrome Foundation to investigate an innovative technique for delivering gene drugs to the salivary glands to potentially block the development and progression of Sjögren’s syndrome.

Sjögren’s syndrome is a chronic autoimmune disease in which people’s white blood cells attack their moisture-producing glands. An estimated four million Americans are living with this disease which causes dry eyes and mouth, fatigue and joint and muscle pain. Sjögren’s also may lead to dysfunction of the kidneys, digestive tract, blood vessels, lungs, liver, pancreas and central, peripheral and autonomic nervous systems.

ASRI’s Gene Therapy Program, based at Allegheny General Hospital, received the Sjögren’s Syndrome Foundation’s (SSF) highest grant award, the Innovative Concept Grant. The $100,000 two-year grant will fund a project to determine the effectiveness of ultrasound-assisted gene transfer to the salivary glands in a mouse model of Sjögren’s.

“This is an interesting and imaginative approach which could move the possibility of a gene therapy option closer to reality for Sjögren’s patients,” said Steve Taylor, Chief Executive Officer of the Sjögren’s Syndrome Foundation.

Michael J. Passineau, PhD, principal investigator for the study and Director of the Gene Therapy Program for ASRI, is leading a burgeoning research program exploring cutting-edge therapies based upon genetic medicines for a variety of conditions including pulmonary vascular disease and lysosomal storage diseases. For Sjögren’s syndrome, gene therapy shows special promise.

“Gene therapy is a particularly exciting approach to this difficult disease because it can directly target the salivary gland with a gene drug,” Dr. Passineau said. “With traditional pharmacology, you take a pill like aspirin or get an injection, but only a very small amount of the medicine reaches its target—the rest is absorbed throughout the rest of the body, which leads to major systemic side-effects.”

The project builds on prior work in the Sjögren’s syndrome field, which has shown that certain gene drugs can block the progression of the disease in mouse models. Prior studies relied on modified versions of naturally occurring viruses to carry the drug into cells, but these viral “vectors” induce inflammation in the salivary gland and actually make the disease worse.

The potentially paradigm-shifting approach being pioneered by the ASRI team uses safe, clinical-grade ultrasonic energy to poke microscopic holes in the membranes of salivary gland cells, allowing the gene drug to slip through. These holes remain open for just seconds and don’t harm the cell. Once inside the salivary gland cells, the gene drug cannot be distinguished from the patient’s own DNA and the immune system ignores it.

“We are proud to have been recognized by the Sjögren’s Syndrome Foundation for our inventive research efforts in gene therapy,” said Joseph Ahearn, MD, Chief Scientific Officer and Vice President, Allegheny Singer Research Institute. “We look forward to furthering the knowledge of Sjögren’s and collaborating with the SSF to work toward a cure.”

Well-respected nationally for its participation in leading medical research and contributions to the advancement of medicine, ASRI is the research arm of West Penn Allegheny Health System and has been managing and conducting research for 34 years.

Founded in 1983, the Sjögren’s Syndrome Foundation provides patients with practical information and coping strategies that minimize the effects of Sjögren’s syndrome and works to increase awareness of the disease and encourage research that may lead to a cure.


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