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:


(Date:10/13/2017)... ... ... On The Brink”: the Christian history of the United States and the loss of ... William Nowers. Captain Nowers and his wife, Millie, have six children, ten grandchildren, ... Navy. Following his career as a naval aviator and carrier pilot, he spent ...
(Date:10/12/2017)... ... October 12, 2017 , ... Planet Fitness, one of the largest and ... plans to open a flagship location in Covington, LA at 401 N. U.S. Highway ... store next to Office Depot in the Holiday Square shopping center. Its location allows ...
(Date:10/12/2017)... ... 2017 , ... The American College of Medical Informatics (ACMI) will present the ... Opening Session of AMIA’s Annual Symposium in Washington, D.C. AMIA’s Annual Symposium ... a pioneer in the field of medical informatics, this prestigious award is presented to ...
(Date:10/12/2017)... ... October 12, 2017 , ... Dr. Parsa Mohebi, the Los ... article to the newly revamped Cosmetic Town journal section, featuring articles ... procedure known as Follicular Unit Extraction (FUE). , Dr. Mohebi says ...
(Date:10/12/2017)... FL (PRWEB) , ... October 12, 2017 , ... On ... holding a treadmill relay – Miles by Moonlight to raise money for the American ... $300 or more. , Teams will work together to keep their treadmills moving ...
Breaking Medicine News(10 mins):
(Date:10/2/2017)... announces the European launch of their new low volume, high throughput ... Cambridge, U.K on October 4th. The new ... unprecedented speed and sensitivity while using far less sample volume through ... ... ...
(Date:9/28/2017)... -- Hill-Rom Holdings, Inc. (NYSE: HRC), will host its ... on Friday, November 3, 2017, beginning at 7:00 a.m. ... 8:30 a.m. (CDT) / 9:30 a.m. (EDT). ... and guidance for 2018, Hill-Rom executives will also highlight ... and long-range financial outlook through 2020. ...
(Date:9/27/2017)... MIAMI , Sept. 27, 2017  Commended for their ... recent notable awards. Ranked as number one in the South ... ninth time in Inc. 5000 yearly list, the national specialty ... CEO, Armando Bardisa will soon be honored by ... Set to receive his award ...
Breaking Medicine Technology:
Cached News: