HOME >> BIOLOGY >> NEWS
Scientists bypass major hurdle to hemophilia gene therapy

Scientists bypass major hurdle to hemophilia gene therapy; animal studies yield high levels of clotting protein

CHAPEL HILL - For the first time, scientists at the University of North Carolina at Chapel Hill have used a gene-therapy technique in animals to continually produce very high amounts of a clotting protein similar to that lacking in people with hemophilia.

A lack of this protein, known as factor IX, occurs in hemophilia type B. If human gene therapy studies could yield sustained factor IX production in high amounts, then hemophilia patients would no longer need daily injections of the protein. The new research suggests this might someday prove feasible.

Moreover, the new findings also indicate that the gene-therapy method used in the study may be applied to hemophilia A, the more common form of the disease. And a report published Dec. 4 in the journal Molecular Therapy concludes that the approach "may be useful for the treatment of a wide variety of inherited diseases."

In animal experiments at Carolina and elsewhere in recent years, the method involved a genetically engineered virus called AAV to infect cells and thereby deliver a cloned gene into an animal's body. Previous studies used only type 2 of six known AAV serotypes, each of which differ in their protein wrapper. This time, however, the Carolina researchers tried five of the six, comparing factor IX production of AAV types 1, 3, 4 and 5 with that of type 2. The results were startling.

Surprisingly and unexpectedly, we found the mice were producing amounts of this factor 100 to a thousand times more than weve observed before, said senior study author Dr. Christopher E. Walsh, assistant professor of medicine at the UNC-CH School of Medicine and clinical director of the universitys Gene Therapy Center.

"The purpose of this experiment was to see if we could generate very high protein production long-term," he added. "These mice are now six months out
'"/>

Contact: Leslie Lang
LLANG@MED.UNC.EDU
919-843-9687
University of North Carolina School of Medicine
3-Dec-2000


Page: 1 2 3

Related biology news :

1. Scientists to prototype cyberinfrastructure for research and education access to ocean observatories
2. Scientists sequence genome of kind of organism central to biospheres carbon cycle
3. Scientists find nanowires capable of detecting individual viruses
4. Scientists discover potential new way to control drug-resistant bacteria
5. Scientists explore genome of methane-breathing microbe
6. Scientists decipher genetic code of biothreat pathogen
7. Stuck on you: Scientists lay bare secrets of bacterial attachment proteins
8. Scientists discover proteins involved in spread of HIV-1 infection
9. Scientists fear new Ebola outbreak may explain sudden gorilla disappearance
10. Scientists reinvent DNA as template to produce organic molecules
11. Scientists visualise cellular handmaiden that restores shape to proteins

Post Your Comments:
*Name:
*Comment:
*Email:


(Date:4/5/2017)... -- Today HYPR Corp. , leading innovator in ... the HYPR platform is officially FIDO® Certified . ... that empowers biometric authentication across Fortune 500 enterprises and ... 15 million users across the financial services industry, however ... suites and physical access represent a growing portion of ...
(Date:3/30/2017)... HONG KONG , March 30, 2017 ... developed a system for three-dimensional (3D) fingerprint identification by adopting ground ... technology into a new realm of speed and accuracy for use ... applications at an affordable cost. ... ...
(Date:3/24/2017)... The Controller General of Immigration from Maldives Mr. Mohamed Anwar ... the prestigious international IAIR Award for the most innovative high security ePassport ... ... Maldives Immigration Controller General, Mr. ... on the right) have received the IAIR award for the "Most innovative ...
Breaking Biology News(10 mins):
(Date:10/12/2017)... (PRWEB) , ... October 12, 2017 , ... ... Sciences today announced the three Winners and six Finalists of the 2017 Blavatnik ... annually by the Blavatnik Family Foundation and administered by the New York Academy ...
(Date:10/12/2017)... (PRWEB) , ... October 12, 2017 , ... ... Surgical Wound Market with the addition of its newest module, US Hemostats & ... market for thrombin hemostats, absorbable hemostats, fibrin sealants, synthetic sealants and biologic sealants ...
(Date:10/11/2017)... (PRWEB) , ... October 11, 2017 , ... ... Foundation President Andi Purple announced Dr. Suneel I. Sheikh, the co-founder, CEO ... ASTER Labs ), Inc. has been selected for membership in ARCS Alumni ...
(Date:10/11/2017)... ... October 11, 2017 , ... ComplianceOnline’s Medical Device Summit is back ... 8th June 2018 in San Francisco, CA. The Summit brings together current and former ... CEOs, board directors and government officials from around the world to address key issues ...
Breaking Biology Technology:
Cached News: