Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:


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


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:

More articles from Health and Medicine:

nachricht Resolving the mystery of preeclampsia
21.10.2016 | Universitätsklinikum Magdeburg

nachricht New potential cancer treatment using microwaves to target deep tumors
12.10.2016 | University of Texas at Arlington

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: New 3-D wiring technique brings scalable quantum computers closer to reality

Researchers from the Institute for Quantum Computing (IQC) at the University of Waterloo led the development of a new extensible wiring technique capable of controlling superconducting quantum bits, representing a significant step towards to the realization of a scalable quantum computer.

"The quantum socket is a wiring method that uses three-dimensional wires based on spring-loaded pins to address individual qubits," said Jeremy Béjanin, a PhD...

Im Focus: Scientists develop a semiconductor nanocomposite material that moves in response to light

In a paper in Scientific Reports, a research team at Worcester Polytechnic Institute describes a novel light-activated phenomenon that could become the basis for applications as diverse as microscopic robotic grippers and more efficient solar cells.

A research team at Worcester Polytechnic Institute (WPI) has developed a revolutionary, light-activated semiconductor nanocomposite material that can be used...

Im Focus: Diamonds aren't forever: Sandia, Harvard team create first quantum computer bridge

By forcefully embedding two silicon atoms in a diamond matrix, Sandia researchers have demonstrated for the first time on a single chip all the components needed to create a quantum bridge to link quantum computers together.

"People have already built small quantum computers," says Sandia researcher Ryan Camacho. "Maybe the first useful one won't be a single giant quantum computer...

Im Focus: New Products - Highlights of COMPAMED 2016

COMPAMED has become the leading international marketplace for suppliers of medical manufacturing. The trade fair, which takes place every November and is co-located to MEDICA in Dusseldorf, has been steadily growing over the past years and shows that medical technology remains a rapidly growing market.

In 2016, the joint pavilion by the IVAM Microtechnology Network, the Product Market “High-tech for Medical Devices”, will be located in Hall 8a again and will...

Im Focus: Ultra-thin ferroelectric material for next-generation electronics

'Ferroelectric' materials can switch between different states of electrical polarization in response to an external electric field. This flexibility means they show promise for many applications, for example in electronic devices and computer memory. Current ferroelectric materials are highly valued for their thermal and chemical stability and rapid electro-mechanical responses, but creating a material that is scalable down to the tiny sizes needed for technologies like silicon-based semiconductors (Si-based CMOS) has proven challenging.

Now, Hiroshi Funakubo and co-workers at the Tokyo Institute of Technology, in collaboration with researchers across Japan, have conducted experiments to...

All Focus news of the innovation-report >>>



Event News

#IC2S2: When Social Science meets Computer Science - GESIS will host the IC2S2 conference 2017

14.10.2016 | Event News

Agricultural Trade Developments and Potentials in Central Asia and the South Caucasus

14.10.2016 | Event News

World Health Summit – Day Three: A Call to Action

12.10.2016 | Event News

Latest News

Resolving the mystery of preeclampsia

21.10.2016 | Health and Medicine

Stanford researchers create new special-purpose computer that may someday save us billions

21.10.2016 | Information Technology

From ancient fossils to future cars

21.10.2016 | Materials Sciences

More VideoLinks >>>