Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:


New gene therapy tool successfully treats mice with hemophilia A


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, 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,

Leslie H. Lang | EurekAlert

More articles from Health and Medicine:

nachricht Indian roadside refuse fires produce toxic rainbow
26.10.2016 | Duke University

nachricht Inflammation Triggers Unsustainable Immune Response to Chronic Viral Infection
24.10.2016 | Universität Basel

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: Etching Microstructures with Lasers

Ultrafast lasers have introduced new possibilities in engraving ultrafine structures, and scientists are now also investigating how to use them to etch microstructures into thin glass. There are possible applications in analytics (lab on a chip) and especially in electronics and the consumer sector, where great interest has been shown.

This new method was born of a surprising phenomenon: irradiating glass in a particular way with an ultrafast laser has the effect of making the glass up to a...

Im Focus: Light-driven atomic rotations excite magnetic waves

Terahertz excitation of selected crystal vibrations leads to an effective magnetic field that drives coherent spin motion

Controlling functional properties by light is one of the grand goals in modern condensed matter physics and materials science. A new study now demonstrates how...

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...

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

3-D-printed structures shrink when heated

26.10.2016 | Materials Sciences

Indian roadside refuse fires produce toxic rainbow

26.10.2016 | Health and Medicine

First results of NSTX-U research operations

26.10.2016 | Physics and Astronomy

More VideoLinks >>>