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

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
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 2,600+ scientific posters on ePosters
  • More than 3,800+ 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 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
New Gene Therapy for Vision Loss From a Mitochondrial Disease
NIH-funded study shows success in targeting mitochondrial DNA in mice.
Predictive Model for Breast Cancer Progression
Biomedical engineers have demonstrated a proof-of-principle technique that could give women and their oncologists more personalized information to help them choose options for treating breast cancer.
Specific Variations in RNA Splicing Linked to Breast Cancer
Researchers have identified cellular changes that may play a role in converting normal breast cells into tumors. Targeting these changes could potentially lead to therapies for some forms of breast cancer.
Gene Expression: A Snapshot of Stem Cell Development
New genes found that regulate development of stem cells.
Assessing Cancer Patient Survival and Drug Sensitivity
RNA editing events another way to investigate biomarkers and therapy targets.
Editing Genes to Create HIV Killers
Seattle scientists have managed to genetically transform human cells in the lab from HIV targets to HIV killers, and the technique could have implications for cancer and other diseases.
Researchers Disguise Drugs As Platelets to Target Cancer
Researchers have for the first time developed a technique that coats anticancer drugs in membranes made from a patient’s own platelets.
A New Single-Molecule Tool to Observe Enzymes at Work
A team of scientists at the University of Washington and the biotechnology company Illumina have created an innovative tool to directly detect the delicate, single-molecule interactions between DNA and enzymatic proteins.
Milestone Single-Biomolecule Imaging Technique May Advance Drug Design
The first nanometer resolved image of individual tobacco mosaic virions shows the potential of low-energy electron holography for imaging biomolecules at a single particle level; a milestone in structural biology and a potential new tool for drug design.
Opening the Door to Safer, More Precise Cancer Therapies
New method regulates when, and how strongly, cancer-killing therapeutic T cells are activated.

Skyscraper Banner
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
2,600+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
3,800+ scientific videos