HOME >> MEDICINE >> NEWS
Novel gene therapy may lead to cure in hemophilia A patients

A discovery by Medical College of Wisconsin and BloodCenter of Wisconsin researchers in Milwaukee may be a key to a permanent genetic cure for hemophilia A patients, including a subset who do not respond to conventional blood transfusions. The study of genetically altered hemophilia mice is published in the July 2006 edition of The Journal of Clinical Investigation.

Hemophilia A affects about one in 5,000 males who lack the hereditary blood clotting protein, Factor VIII (FVIII). Traditional treatment requires infusion of synthetic FVIII two to three times a week to control bleeding episodes. However, about 30 percent of these patients develop antibodies to FVIII, selectively inactivating its clotting properties and negating its therapeutic role. Treatment for adults who have these inhibitory antibodies can cost over $1 million annually if there is a major bleeding episode.

"We developed a Trojan horse approach cloaking FVIII in a platelet so that it is undetected by the antibodies and its clotting properties are preserved until the platelet sticks to a damaged blood vessel and releases its stored protein which now includes FVIII.," explains senior author Robert R. Montgomery, M.D., senior investigator at the BloodCenter of Wisconsin and professor of pediatric hematology at the Medical College. He is also affiliated with the Children's Research Institute.

"This is truly a landmark development for hemophilia A patients," says hematologist, Joan Gill, M.D., professor of pediatric hematology at the Medical College, and director of the Comprehensive Center for Bleeding Disorders at the BloodCenter and Children's Hospital of Wisconsin. "We look forward to the day when basic research is completed and clinical trials can begin in patients."

Normally in an injury, platelets circulating in plasma - the liquid portion of the blood - stick to the site of the blood vessel wound, activating its surface and rapidly stoppi
'"/>

Contact: Toranj Marphetia
toranj@mcw.edu
414-456-4744
Medical College of Wisconsin
3-Jul-2006


Page: 1 2 3

Related medicine news :

1. Novel candidate biomarker for heart failure also strongly predicts risk of death
2. Novel treatments may help alleviate constipation, IBS in women
3. Novel catheter technique successfully patches holes in the heart
4. Novel anti-oxidant and anti-inflammatory agent shows effectiveness on key endpoints in trial
5. Novel Ames Lab composite may replace depleted uranium
6. Novel computed imaging technique uses blurry images to enhance view
7. Novel brain areas associated with the recognition of gender, ethnicity and the identity of faces
8. Novel program enhances dementia caregivers quality of life
9. Novel therapy combinations gain ground in treating hepatitis
10. Novel stem cell technology leads to better spinal cord repair
11. Novel virus entry mechanism could lead to new drugs against poxviruses

Post Your Comments:
*Name:
*Comment:
*Email:
TAG: Novel gene therapy may lead cure hemophilia patients

(Date:5/24/2015)... 2015 The federal court overseeing ... has scheduled its next monthly Case Management Conference ... According to court documents filed in the U.S. ... will be convened on June 2, 2015, at ... Liability Litigation - MDL No. 2545) , “Our ...
(Date:5/24/2015)... May 24, 2015 ... CEO - The students were ... at the fourth annual A Day ... at Geneva National in Lake Geneva. ...     -Badger High School,         Jonathan Alfonso,         Bridget ...
(Date:5/23/2015)... May 23, 2015 Dr. David Benvenuti, an ... just posted a new article to his website ... Dr Benvenuti explained in the fascinating blog, he ... in their earlobes. , Known as “gauging,” the technique involves ... which Dr. Benvenuti said will eventually stretch the holes out ...
(Date:5/23/2015)... Manufacturers are now required to produce water heaters ... Experts in the field agree that the regulation is aimed ... score is what miles per gallon is to your car. ... the minimum HSPF score has been raised to 8.2, technology ... north of 12. Indeed, the regulation was welcomed by environmentalist, ...
(Date:5/23/2015)... May 23, 2015 Injury lawyer Steven H. ... been passed by the Maryland legislature but which has not ... General Assembly passed HB 449 with overwhelming bipartisan ... not signed it into law. This bill ensures that fracking ... growing that demonstrates the danger of fracking to our water ...
Breaking Medicine News(10 mins):Health News:Testosterone Lawsuit Attorneys at Bernstein Liebhard LLP Look Ahead to June Conference in Federal Multidistrict Litigation 2Health News:Testosterone Lawsuit Attorneys at Bernstein Liebhard LLP Look Ahead to June Conference in Federal Multidistrict Litigation 3Health News:Mercy Foundation Honors 16 Students for Making a Difference - Mercy Health System Javon Bea 2Health News:Dr. Benvenuti, a Plastic Surgeon in Newport Beach, CA, Posts New Article about Repairing Ear Gauges 2Health News:Parker & Sons Welcomes the Department of Energy’s Water Heater Regulation 2Health News:Baltimore Attorney Urges Governor Hogan to Sign the Fracking Moratorium Bill 2Health News:Baltimore Attorney Urges Governor Hogan to Sign the Fracking Moratorium Bill 3
(Date:5/21/2015)... DUBLIN , May 21, 2015 /PRNewswire/ ... http://www.researchandmarkets.com/research/5x7nlz/arthroscopy ) has announced the addition of ... report to their offering. ,     (Logo: http://photos.prnewswire.com/prnh/20130307/600769) ... to analyze and size the world market ... players and identifies trends impacting the market ...
(Date:5/21/2015)... , May 21, 2015  (MUSE Booth # ... Health Record (EHR) is invaluable in any hospital ... are now a requirement and mainstream in healthcare, ... into the EHR is gaining traction in MEDITECH ... Holmes, RN, Site Project Manager & Clinical Analyst ...
(Date:5/21/2015)... England , May 21, 2015 ... Bekämpfung von Arzneimittelfälschungen - Zwecks Leistungsvergleich ... Arzneimittelprüfdienstes wird auf EG-Mitgliedstaaten verwiesen, in ... betrieben werden.  Wählen Sie ein Aegate-Land: ... der Richtlinie zur Bekämpfung von Arzneimittelfälschungen ...
Breaking Medicine Technology:Analysing Arthroscopy Devices Markets Worldwide 2014-2020 2Analysing Arthroscopy Devices Markets Worldwide 2014-2020 3Hospital Streamlines ICU Efficiency with Timely Data Integration 2Hospital Streamlines ICU Efficiency with Timely Data Integration 3EU-Arzneimittel-Richtlinie für gefälschte Medikamente; Aegate-Zusammenfassung als reale Bewertung aus Belgien 2EU-Arzneimittel-Richtlinie für gefälschte Medikamente; Aegate-Zusammenfassung als reale Bewertung aus Belgien 3
Cached News: