Corporate Banner
Satellite Banner
Immunology
Scientific Community
 
Become a Member | Sign in
Home>News>This Article
  News
Return

Researchers Identify Way to Increase Gene Therapy Success

Published: Thursday, October 31, 2013
Last Updated: Thursday, October 31, 2013
Bookmark and Share
Scientists have found a way to keep the immune system from neutralizing a therapeutic virus before it can deliver its genetic payload.

In a study published recently in Molecular Therapy, researchers found that giving subjects a treatment to temporarily rid the body of antibodies provides the virus safe passage to targeted cells, allowing it to release a corrective or replacement gene to treat disease.

Gene therapy is among the most promising treatment options for such genetic disorders as muscular dystrophy, congenital blindness and hemophilia. Scientists also are investigating gene therapy as a cure for some cancers, neurodegenerative diseases, viral infections and other acquired illnesses. To get the therapeutic gene into cells, researchers have turned to viruses, which deliver their genetic material into cells as part of their normal replication process. Time and time again, these efforts have been thwarted by the body’s own immune system, which attacks the viral vector. The therapeutic genes aren’t delivered and disease rages on.

Now, a team led by Louis G. Chicoine, MD, Louise Rodino-Klapac, PhD, and Jerry R. Mendell, MD, principal investigators in the Center for Gene Therapy at Nationwide Children’s, has shown for the first time that using a process called plasmapheresis just before delivering a virus-packed gene therapy protects the virus long enough for it to enter the cell and deliver the gene.

Plasmapheresis, widely used to treat patients with autoimmune disorders, removes blood from the body, separates the plasma and cells, filters out antibodies, and returns the blood to the patient. The antibody loss is temporary; the body begins producing new antibodies within a few hours of the procedure.

In a study of a gene therapy designed to treat Duchenne muscular dystrophy (DMD), Drs. Chicoine and Rodino-Klapac used plasmapheresis in a large animal model, then injected a virus packed with a micro-dystrophin gene. When they examined the levels of micro-dystrophin gene expression in the animals, they found a 500 percent percent increase over gene expression in animals that did not receive plasmapheresis. Dr. Mendell, director of the Center for Gene Therapy, helped conceive of this treatment for DMD patients based on experience with autoimmune diseases such as myasthenia gravis and inflammatory nerve diseases.

“Right now, gene therapy seems to work best in patients who have no antibodies for the virus being used to deliver the gene,” Dr. Mendell says. “That limits the number of patients who can benefit from gene therapy.”

Using plasmapheresis would increase the potential for gene therapy, Dr. Chicoine adds, by eliminating one obstacle of immune reaction.

“As gene therapy becomes more prevalent, patients may need to receive more than one treatment,” Dr. Rodino-Klapac says. “The problem is that when they get the first treatment, their body will develop antibodies to the virus used to deliver the gene. Using plasmapheresis on someone who previously received gene therapy could allow them to be treated again.”


Further Information

Join For Free

Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 3,100+ scientific posters on ePosters
  • More than 4,500+ scientific videos on LabTube
  • 35 community eNewsletters


Sign In



Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into TechnologyNetworks.com you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.


Scientific News
Alzheimer’s Protein Serves as Natural Antibiotic
Alzheimer's-associated amyloid plaques may be part of natural process to trap microbes, findings suggest new therapeutic strategies.
Slime Mold Reveals Clues to Immune Cells’ Directional Abilities
Study from UC San Diego identifies a protein involved in the directional ability of a slime mold.
Supressing Intenstinal Analphylaxis in Peanut Allergy
Study from National Jewish Health shows that blockade of histamine receptors suppresses intestinal anaphylaxis in peanut allergy.
Getting a Better Look at How HIV Infects and Takes Over its Host Cells
A new approach, developed by a team of researchers led by The Rockefeller University and The Aaron Diamond AIDS Research Center (ADARC), offers an unprecedented view of how a virus infects and appropriates a host cell, step by step.
Untangling Disease-Related Protein Misfolding
Work advances understanding of genetic forms of thrombosis, emphysema, cirrhosis of the liver, neurodegenerative diseases and inflammation, among others.
Developing a More Precise Seasonal Flu Vaccine
During the 2014-15 flu season, the poor match between the virus used to make the world’s vaccine stocks and the circulating seasonal virus yielded a vaccine that was less than 20 percent effective.
Fighting Cancer with Borrowed Immunity
A new step in cancer immunotherapy: researchers from the Netherlands Cancer Institute and University of Oslo/Oslo University Hospital show that even if one's own immune cells cannot recognize and fight their tumors, someone else's immune cells might.
Loss Of Y Chromosome Increases Risk Of Alzheimer’s
Men with blood cells that do not carry the Y chromosome are at greater risk of being diagnosed with Alzheimer’s disease. This is in addition to an increased risk of death from other causes, including many cancers. These new findings by researchers at Uppsala University could lead to a simple test to identify those at risk of developing Alzheimer’s disease.
Immune Cells Remember Their First Meal
Scientists at the University of Bristol have identified the trigger for immune cells' inflammatory response – a discovery that may pave the way for new treatments for many human diseases.
"Sunscreen" Gene May Guard Against Melanoma
USC-led study reveals that melanoma patients with deficient or mutant copies of the gene are less protected from harmful ultraviolet rays.
SELECTBIO

SELECTBIO Market Reports
Go to LabTube
Go to eposters
 
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
3,100+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
4,500+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FOR FREE!