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)... ... 2017 , ... Lori R. Somekh, founder of the Law Office of ... elder law and special needs planning attorneys. “Membership in ElderCounsel helps our office remain ... to network with elder law attorneys nationwide,” said Somekh. , ElderCounsel ...
(Date:10/13/2017)... ... 2017 , ... Many families have long-term insurance that covers care for a ... waiver for care if the client has a cognitive impairment diagnosis. , “What ... is often waived, so the benefits from their insurance start immediately,” said Mechell Vieira, ...
(Date:10/13/2017)... ... October 13, 2017 , ... Ellevate Network, the leading network for professional ... action towards gender equality at their inaugural Summit in New York City in June. ... a social audience of over 3 million. To watch the Mobilize Women video, ...
(Date:10/13/2017)... ... ... Yisrayl Hawkins, Pastor and Overseer at The House of Yahweh, has released ... understood books in the Holy Scriptures, Revelation. The Book of Revelation paints a picture ... Many have tossed it off as mere rubbish, but Yisrayl Hawkins says that is ...
(Date:10/13/2017)... VA (PRWEB) , ... October 13, 2017 , ... ... of DevOps and Agile Software Development, has been awarded a contract by the ... Blanket Purchase Agreement (BPA) aims to accelerate the enterprise use of Agile methodologies ...
Breaking Medicine News(10 mins):
(Date:10/12/2017)... 2017 West Pharmaceutical Services, Inc. ... injectable drug administration, today announced that it will release ... Thursday, October 26, 2017, and will follow with a ... at 9:00 a.m. Eastern Time. To participate on the ... conference ID is 94093362. ...
(Date:10/10/2017)... received FDA 510(k) clearance in May 2017 for its highly anticipated ... for endoscopy environments. An innovative secondary monitor solution, ZeroWire Mobile ... the improvement of patient outcomes, procedural efficiency, and the lowering of ... ... By ...
(Date:10/4/2017)... 4, 2017  According to the Centers for Disease Control and Prevention ... . PhysicianOne Urgent Care is helping communities across Massachusetts ... , by offering no-cost* flu shots through the end of the month. ... health insurance regulations. ... to get a flu shot is by the end of October, according ...
Breaking Medicine Technology:
Cached News: