Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

World-first gene therapy for cystic fibrosis targets lung stem cells?

21.10.2002


PhD student Maria Limberis inspects a CF mouse


The genetically – inherited disease cystic fibrosis causes severe, unrelenting lung disease in children and adults worldwide. Approximately 1 in 2,500 infants are born with this disease and only half survive past 30 years of age.

Now, researchers from the Women’s and Children’s Hospital, Adelaide have developed a novel system of gene therapy for lungs affected by cystic fibrosis, involving a natural compound found in our lungs which ’conditions’ lung airways to allow cells to take up the therapeutic gene.

Our lungs have developed highly effective ways to protect us from allergens, irritants, dust, viruses and other foreign particles. But according to principal medical scientist in Pulmonary Medicine, Dr David Parsons these defences also hinder effective gene therapy in our lungs.



"Using a mouse model of cystic fibrosis Maria Limberis, a PhD student in our lab, has helped us develop a system to briefly overcome these defences. As the cells lining the mouse nose behave in much the same way as human lung cells – this enables us to use the nose airways in mice to easily develop and test out gene therapy treatments.

"By instilling a single dose of a detergent found naturally in low amounts in our lungs, we are able to ’condition’ cells to take up the gene needed to treat cystic fibrosis.

" Viruses are very good at transferring their genetic material into cells and we make use of this by getting useful parts of an inactive and highly-modified human immunodeficiency virus type 1 (HIV 1) to safely transfer the cystic fibrosis gene into cells. We use this modified HIV because it is one of the few viruses that can give long-lasting gene transfer," Dr Parsons says.

Using this system, the research team has shown, for the first time in a living animal, that long-lived gene therapy for cystic fibrosis is possible. Not only do the airway cells take up the correcting gene, but these cells also show substantial recovery from the cystic fibrosis defect for, so far, at least 110 days.

"Airway cells are replaced every three months so our findings are particularly exciting because they imply we are in fact targeting airway stem cells through this approach - some of the therapeutic gene must have been passed on from these parent stem cells to their daughter cells for the effect to persist beyond three months," Dr Parsons says.

Research funding is now being sought to establish the most effective dose and timing for giving the detergent together with rigorous safety checks on the highly modified HIV-1 based virus particle used for the gene transfer.

Another senior member of the team, molecular biologist Dr Don Anson, explains, "Last year we published a method which vastly increases the safety with which HIV-1 can be modified and used to transfer genes without causing disease.

"We are now working to further improve on this method in order that patients and their families will feel totally confident to eventually take part in human trials of this gene therapy for cystic fibrosis," Dr Anson says.


The work from this research is to be published in Human Gene Therapy, volume 13 #16 on October 20.

Members of the Research Team are:
Dr David Parsons (Medical Scientist, Pulmonary Medicine)
Dr Don Anson (Senior Molecular Biologist, Chemical Pathology)
Dr Maria Fuller (Molecular Biologist, Chemical Pathology)
Ms Maria Limberis (PhD student, Pulmonary Medicine)

Dr Edna Bates | EurekAlert!
Further information:
http://www.wch.sa.gov.au/

More articles from Health and Medicine:

nachricht Researchers release the brakes on the immune system
18.10.2017 | Rheinische Friedrich-Wilhelms-Universität Bonn

nachricht Norovirus evades immune system by hiding out in rare gut cells
12.10.2017 | University of Pennsylvania School of Medicine

All articles from Health and Medicine >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Neutron star merger directly observed for the first time

University of Maryland researchers contribute to historic detection of gravitational waves and light created by event

On August 17, 2017, at 12:41:04 UTC, scientists made the first direct observation of a merger between two neutron stars--the dense, collapsed cores that remain...

Im Focus: Breaking: the first light from two neutron stars merging

Seven new papers describe the first-ever detection of light from a gravitational wave source. The event, caused by two neutron stars colliding and merging together, was dubbed GW170817 because it sent ripples through space-time that reached Earth on 2017 August 17. Around the world, hundreds of excited astronomers mobilized quickly and were able to observe the event using numerous telescopes, providing a wealth of new data.

Previous detections of gravitational waves have all involved the merger of two black holes, a feat that won the 2017 Nobel Prize in Physics earlier this month....

Im Focus: Smart sensors for efficient processes

Material defects in end products can quickly result in failures in many areas of industry, and have a massive impact on the safe use of their products. This is why, in the field of quality assurance, intelligent, nondestructive sensor systems play a key role. They allow testing components and parts in a rapid and cost-efficient manner without destroying the actual product or changing its surface. Experts from the Fraunhofer IZFP in Saarbrücken will be presenting two exhibits at the Blechexpo in Stuttgart from 7–10 November 2017 that allow fast, reliable, and automated characterization of materials and detection of defects (Hall 5, Booth 5306).

When quality testing uses time-consuming destructive test methods, it can result in enormous costs due to damaging or destroying the products. And given that...

Im Focus: Cold molecules on collision course

Using a new cooling technique MPQ scientists succeed at observing collisions in a dense beam of cold and slow dipolar molecules.

How do chemical reactions proceed at extremely low temperatures? The answer requires the investigation of molecular samples that are cold, dense, and slow at...

Im Focus: Shrinking the proton again!

Scientists from the Max Planck Institute of Quantum Optics, using high precision laser spectroscopy of atomic hydrogen, confirm the surprisingly small value of the proton radius determined from muonic hydrogen.

It was one of the breakthroughs of the year 2010: Laser spectroscopy of muonic hydrogen resulted in a value for the proton charge radius that was significantly...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

ASEAN Member States discuss the future role of renewable energy

17.10.2017 | Event News

World Health Summit 2017: International experts set the course for the future of Global Health

10.10.2017 | Event News

Climate Engineering Conference 2017 Opens in Berlin

10.10.2017 | Event News

 
Latest News

Terahertz spectroscopy goes nano

20.10.2017 | Information Technology

Strange but true: Turning a material upside down can sometimes make it softer

20.10.2017 | Materials Sciences

NRL clarifies valley polarization for electronic and optoelectronic technologies

20.10.2017 | Interdisciplinary Research

VideoLinks
B2B-VideoLinks
More VideoLinks >>>