Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

New gene therapy tool successfully treats mice with hemophilia A

10.06.2002


Scientists at the University of North Carolina have successfully treated mice with hemophilia A using a new approach to gene therapy - RNA trans-splicing. The experimental procedure repairs a mutated section of the gene responsible for hemophilia A, a hereditary bleeding disorder.



Dr. Hengjun Chao, a research assistant professor at the UNC School of Medicine, Gene Therapy Center will present the new research Saturday June 8 in Boston at the Presidential Symposium of the American Society of Gene Therapy Annual Meeting.

Hemophilia A is a sex-linked congenital disease, occurring in one out of 5,000 to 10,000 live males in all populations and is caused by a defect in coagulation factor VIII. The mutation renders the factor VIII gene non-functional resulting in recurrent, non-predictable, spontaneous bleeding into major joints and soft tissues. Currently, the disorder is treated with injections of factor VIII protein in response to bleeding incidents. Conventional approaches to gene therapy have not proven successful against hemophilia A, partially due to difficulties involved in packaging and delivering the large factor VIII gene.


This new study in mice with hemophilia A was conducted in collaboration with scientists from Intronn Inc., Rockville, Maryland, where RNA trans-splicing was pioneered. A "pre-trans-splicing molecule" (PTM) was injected into some of the mice. The molecule, or "cassette," is designed to produce RNA that binds and splices onto the existing faulty RNA, correcting it. The corrected RNA then encodes for the normal factor VIII protein.

"Preliminary data using the hemophilia A mice is very encouraging," said Dr. Hengjun Chao, "After injecting hemophilia A mice with the PTM cassette, factor VIII levels in the blood rose from lower than 1% to a maximum of 20 % of normal factor VIII activity. These levels of activity corrected the bleeding tendency of the hemophilia A mice, thus protecting the mice from a trauma challenge, which is usually lethal to untreated hemophilia A mice.

"If the technology is proven effective in humans, it would provide a more permanent treatment for hemophilia A."

According to Dr. Christopher Walsh, Assistant Professor of Medicine at UNC and principal investigator of the study, RNA trans-splicing offers several advantages over conventional DNA gene therapy. "Among these, only a mutated segment of the gene is repaired rather than the entire gene. Also, very large pieces of DNA cannot be effectively packaged and delivered using conventional DNA viral vector therapy. This new gene therapy tool will help treat hemophilia A as well as a host of other genetic diseases such as cystic fibrosis, sickle cell anemia, muscular dystrophy and some forms of cancer."



Note: Contact Chao at (919) 966-9117, hchao@med.unc.edu. From June 6-9, contact Chao at the Boston Marriott Copley Place, 617-236-5800.

School of Medicine contact: Les Lang at (919) 843-9687, llang@med.unc.edu

Leslie H. Lang | EurekAlert

More articles from Health and Medicine:

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

nachricht Flexible sensors can detect movement in GI tract
11.10.2017 | Massachusetts Institute of Technology

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

Ocean atmosphere rife with microbes

17.10.2017 | Life Sciences

Neutrons observe vitamin B6-dependent enzyme activity useful for drug development

17.10.2017 | Life Sciences

NASA finds newly formed tropical storm lan over open waters

17.10.2017 | Earth Sciences

VideoLinks
B2B-VideoLinks
More VideoLinks >>>