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

Gene Therapy for Salivary Gland Shows Promise

Published: Tuesday, December 04, 2012
Last Updated: Tuesday, December 04, 2012
Bookmark and Share
An experimental trial showed that gene therapy can be performed safely in the human salivary gland.

The accomplishment may one day lead to treatments to help head and neck cancer survivors who battle with chronic dry mouth.

People with head and neck cancer often receive radiation therapy to shrink their tumors. The radiation can damage salivary glands, reducing their ability to secrete saliva into the mouth. Saliva is needed for taste, swallowing and speech. It also helps prevent infection and tooth decay. Salivary glands may partly recover after radiation therapy, but recovery is usually not complete. Doctors have limited options to offer most patients.

In the early 1990s, as the first gene therapy studies entered research clinics, Dr. Bruce Baum of NIH’s National Institute of Dental and Craniofacial Research (NIDCR) saw the potential of gene therapy to restore saliva secretion in salivary glands. He and his colleagues have been working for years to restore saliva secretion in animal models. By delivering the gene for a protein called aquaporin-1 into salivary gland cells, they restored saliva secretion in animal models. Aquaporin-1 forms pore-like water channels in cell membranes to help move fluid—such as when salivary gland cells secrete saliva into the mouth.

In 2008, the scientists treated the first patients in a small clinical trial designed to assess safety, work out dosage and identify side effects. The team included investigators from NIDCR, NIH's National Cancer Institute (NCI) and the NIH Clinical Center.

Eleven head and neck cancer survivors received a single-dose infusion directly into one of their 2 parotid salivary glands, the largest of the major salivary glands. The aquaporin-1 gene was packaged in a disabled adenovirus, which causes the common cold when intact. The disabled virus served as a delivery vehicle, or vector, entering cells that line the salivary gland and transferring the gene within. Once inside, the gene is turned on, or expressed, and directs the cells to make aquaporin-1.

The scientists reported on November 20, 2012, in Proceedings of the National Academy of Sciences that 6 of the 11 treated participants had increased levels of saliva secretion. Five also reported a renewed sense of moisture and lubrication in their mouths over the initial 42-day study period. There were no serious side effects.

The researchers tested 4 different doses of virus in the trial. Neither of the 2 people receiving the highest dose showed any benefit from the procedure. This and other observations suggest that higher doses of this virus may backfire, causing the immune system to launch an attack and prevent gene transfer.

“It is time to evaluate a different vector to deliver the aquaporin-1 gene, one that will cause only a minimal immune response,” Baum says.

Because of safety concerns, the researchers used a virus that causes only short-lived gene expression. Future research will be needed to develop methods that not only avoid an immune response but are also capable of longer-lived expression in salivary glands.

“These data will serve as stepping stones for other scientists to improve on this first attempt in the years ahead,” says Baum. “The future for applications of gene therapy in the salivary gland is bright.”


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 2,400+ scientific posters on ePosters
  • More than 3,700+ 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.

Related Content

Drug Combination May be Highly Effective in Recurrent Ovarian Cancer
The drugs were tested in a phase I combination study followed by a randomized phase 2 trial.
Monday, June 02, 2014
TCGA Bladder Cancer Study Reveals Potential Drug Targets, Similarities to Several Cancers
Investigators have identified new potential therapeutic targets for a major form of bladder cancer, including important genes and pathways that are disrupted in the disease.
Thursday, January 30, 2014
Gene Variants Found Associated With Human Immune System, Autoimmune Disease
Numerous studies have reported that certain diseases are inherited. But genetics also plays a role in immune response, affecting our ability to stave off disease.
Friday, September 27, 2013
NIH Program Explores the Use of Genomic Sequencing in Newborn Healthcare
Can sequencing of newborns’ genomes provide useful medical information beyond what current newborn screening already provides?
Wednesday, September 04, 2013
Clues to Congenital Heart Disease
Non-inherited mutations in hundreds of genes together account for about 1 in 10 cases of severe congenital heart defects.
Wednesday, May 22, 2013
Gene Therapy Restores Sense of Smell in Mice
Mice that were unable to smell from birth gained the ability to smell when researchers used gene therapy to regrow structures called cilia on cells that detect odor.
Tuesday, October 02, 2012
Researchers Discover Key Mutation in Acute Myeloid Leukemia
Researchers have discovered mutations in a particular gene that affects the treatment prognosis for some patients with acute myeloid leukemia (AML), an aggressive blood cancer that kills 9,000 Americans annually.
Monday, November 15, 2010
NIH Launches Genotype-Tissue Expression Project
Project to chart influence of DNA changes on gene function in human tissues and organs.
Wednesday, October 13, 2010
Gene Variations that Alter Key Enzyme Linked to Prostate Cancer
Ties found previously for susceptibility to testicular cancer, adrenal gland tumors.
Monday, October 04, 2010
NCBI Launches the Database of Genomic Structural Variations
A new tool to aid in understanding the genetics of health and disease.
Friday, October 01, 2010
NIH Expands Network Focused on How Genes Affect Drug Responses
NIH plans to spend $161.3 million over the next five years to expand the Pharmacogenomics Research Network.
Thursday, September 09, 2010
Gene Scan Finds Link Across Array of Childhood Brain Disorders
Researchers found gene mutations through whole exome sequencing - a new gene scanning technology that cuts the cost and time of searching for rare mutations.
Friday, August 27, 2010
New Compound Improves Obesity-Related Health Complications in NIH-Led Study
A report of the study, which was conducted with obese mice, appears online in the Journal of Clinical Investigation.
Tuesday, July 27, 2010
Gene Variant that may Prevent African Sleeping Sickness also Leads to Increased Chance of Kidney Disease
NIH researchers found that gene variants in APOL1, more common in African Americans, come with both health risk and reward.
Monday, July 19, 2010
NIH Expands National Network for Transforming Clinical and Translational Research
Nine Institutions to receive $255 million over five years to help scientists bridge laboratory discoveries to patient treatments.
Thursday, July 15, 2010
Scientific News
RNAi Screening Trends
Understand current trends and learn which application areas are expected to gain in popularity over the next few years.
The Genetic Roots of Adolescent Scoliosis
Scientists at the RIKEN Center for Integrative Medical Sciences in collaboration with Keio University in Japan have discovered a gene that is linked to susceptibility of Scoliosis.
A Gene-Sequence Swap Using CRISPR to Cure Haemophilia
For the first time chromosomal defects responsible for hemophilia have been corrected in patient-specific iPSCs using CRISPR-Cas9 nucleases
New Tool Uses 'Drug Spillover' to Match Cancer Patients with Treatments
Researchers have developed a new tool that improves the ability to match drugs to disease: the Kinase Addiction Ranker (KAR) predicts what genetics are truly driving the cancer in any population of cells and chooses the best "kinase inhibitor" to silence these dangerous genetic causes of disease.
Understanding the Molecular Origin of Epigenetic Markers
Researchers at IRB Barcelona discover the molecular mechanism that determines how epigenetic markers influence gene expression.
New Tech Enables Epigenomic Analysis with a Mere 100 Cells
A new technology that will dramatically enhance investigations of epigenomes, the machinery that turns on and off genes and a very prominent field of study in diseases such as stem cell differentiation, inflammation and cancer has been developed by researchers at Virginia Tech.
Access Denied: Leukemia Thwarted by Cutting Off Link to Environmental Support
A new study reveals a protein’s critical – and previously unknown -- role in the development and progression of acute myeloid leukemia (AML), a fast-growing and extremely difficult-to-treat blood cancer.
New Weapon in the Fight Against Blood Cancer
This strategy, which uses patients’ own immune cells, genetically engineered to target tumors, has shown significant success against multiple myeloma, a cancer of the plasma cells that is largely incurable.
Toxin from Salmonid Fish has Potential to Treat Cancer
Researchers from the University of Freiburg decode molecular mechanism of fish pathogen.
Study Finds Non-Genetic Cancer Mechanism
Cancer can be caused solely by protein imbalances within cells, a study of ovarian cancer has found.
Skyscraper Banner

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
2,400+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
3,700+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FREE!