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

Gene Therapy Restores Sense of Smell in Mice

Published: Tuesday, October 02, 2012
Last Updated: Tuesday, October 02, 2012
Bookmark and Share
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.

The approach might one day lead to treatments for related human genetic disorders.

Cilia are antenna-like projections on cells that help them sense their environment. Genetic disorders of the cilia, known as ciliopathies, include diseases as diverse as polycystic kidney disease and retinitis pigmentosa—an inherited, degenerative eye disease that causes severe vision impairment and blindness. In the olfactory system, multiple cilia project from olfactory sensory neurons, cells high up in the nasal cavity. These cilia have receptors on their surfaces that bind odorants. A loss of these cilia results in a loss of the ability to smell, which is called anosmia.

A team of researchers, led by Drs. Jeremy C. McIntyre and Jeffrey R. Martens at the University of Michigan, Ann Arbor, has been studying mice carrying a mutation in the IFT88 gene. The mutation causes a decrease in the IFT88 protein, which leads to a dramatic reduction in cilia function in several different organ systems, including the olfactory system. To see if the gene might play a role in human disease, the scientists examined the IFT88 genes of over 200 people with severe ciliopathies. Their work was funded by 4 NIH components, led by the National Institute on Deafness and Other Communications Disorders (NIDCD).

As reported online on September 2, 2012, in Nature Medicine, the scientists detected IFT88 mutations in several people with severe ciliopathies. Experiments in zebrafish, a common laboratory model for development, confirmed that mutant IFT88 genes can cause developmental defects.

The researchers next used a harmless virus to introduce a healthy copy of IFT88 into the mice with the mutant version. For 3 consecutive days, the mice received intranasal doses of the virus. They were then given 10 days for infected sensory neurons to express the IFT88 protein. After this period, the mice were tested with an odorant called amyl acetate. The researchers found that the mice had regained olfactory function.

In many mammals, including humans, the urge to eat is driven by smell. The mice the scientists studied are born underweight, and their anosmia interferes with their motivation to eat. The body weight of mice treated with the gene therapy was 60% higher than that of untreated mice, showing that restored olfactory function motivated feeding.

This study shows that gene therapy can be used to restore functional cilia in established cells. “These results could lead to one of the first therapeutic options for treating people with congenital anosmia,” says NIDCD Director Dr. James F. Battey, Jr. “They also set the stage for therapeutic approaches to treating diseases that involve cilia dysfunction in other organ systems, many of which can be fatal if left untreated.”

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 2,800+ scientific posters on ePosters
  • More Than 4,000+ 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.

Related Content

Test Reliably Detects Inherited Immune Deficiency in Newborns
NIH-supported study suggests that early diagnosis of severe combined immunodeficiency leads to high survival rates.
Wednesday, August 20, 2014
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
Investigational Malaria Vaccine Found Safe and Protective
An investigational malaria vaccine has been found to be safe, to generate an immune system response, and to offer protection against malaria infection in healthy adults.
Friday, August 09, 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
New NIH funding for two Autism Centers of Excellence
A total of 11 centers now funded for up to five years.
Wednesday, April 03, 2013
Modelling Dynamics in Protein Crystal Structures by Ensemble Refinement
Detailed information about the dynamic behaviour of proteins is essential for a proper understanding of a variety of processes, including catalysis, ligand binding and protein–protein interactions.
Monday, January 28, 2013
Gene Therapy for Salivary Gland Shows Promise
An experimental trial showed that gene therapy can be performed safely in the human salivary gland.
Tuesday, December 04, 2012
Therapy Repairs Ravaged Immune System
Gene therapy can safely restore immune function in children with severe combined immunodeficiency and allow some to stop taking painful weekly injections.
Tuesday, October 02, 2012
Researchers Identify Protein Essential for Embryo Implantation
NIH researchers discovery shows how the hormone progesterone suppresses the growth of the uterus's lining so that a fertilized egg can implant in the uterus.
Tuesday, February 22, 2011
The National Database for Autism Research Announces its First Data Release
Autism Spectrum Disorder researchers now can use data from over 10,000 participants enrolled in ASD studies.
Wednesday, December 01, 2010
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
Scientific News
High Throughput Mass Spectrometry-Based Screening Assay Trends
Dr John Comley provides an insight into HT MS-based screening with a focus on future user requirements and preferences.
How a Genetic Locus Protects Adult Blood-Forming Stem Cells
Mammalian imprinted Gtl2 protects adult hematopoietic stem cells by restricting metabolic activity in the cells' mitochondria.
Genetic Basis of Fatal Flu Side Effect Discovered
A group of people with fatal H1N1 flu died after their viral infections triggered a deadly hyperinflammatory disorder in susceptible individuals with gene mutations linked to the overactive immune response, according to a recent study.
New Tech Vastly Improves CRISPR/Cas9 Accuracy
A new CRISPR/Cas9 technology developed by scientists at UMass Medical School is precise enough to surgically edit DNA at nearly any genomic location, while avoiding potentially harmful off-target changes typically seen in standard CRISPR gene editing techniques.
The MaxSignal Colistin ELISA Test Kit from Bioo Scientific
Kit can help prevent the antibiotic apocalypse by keeping last resort drugs out of the food supply.
"Good" Mozzie Virus Might Hold Key to Fighting Human Disease
Australian scientists have discovered a new virus carried by one of the country’s most common pest mosquitoes.
Non-Disease Proteins Kill Brain Cells
Scientists at the forefront of cutting-edge research into neurodegenerative diseases such as Alzheimer’s and Parkinson’s have shown that the mere presence of protein aggregates may be as important as their form and identity in inducing cell death in brain tissue.
Closing the Loop on an HIV Escape Mechanism
Research team finds that protein motions regulate virus infectivity.
New Class of RNA Tumor Suppressors Identified
Two short, “housekeeping” RNA molecules block cancer growth by binding to an important cancer-associated protein called KRAS. More than a quarter of all human cancers are missing these RNAs.
Potential Treatment for Life-Threatening Viral Infections Revealed
The findings point to new therapies for Dengue, West Nile and Ebola.
Scroll Up
Scroll Down
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
2,800+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
4,000+ scientific videos