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:8/1/2015)... ... ... and Lita Hart, icons of the network marketing industry and founders of the Healthy ... Movement of EvolvHealth. In what has been a long-term relationship between the companies for ... of purpose together to change the course of the network marketing industry. , “This ...
(Date:8/1/2015)... ... ... Lume Wellness creators Alex, Robert, and Sam have announced update 3.0.1 to this ... The app is designed to track various metrics of wellness, including physical activity, diet, ... and use this information for positive behavior modification. Lume Wellness keeps track of both ...
(Date:8/1/2015)... Hartford, CT (PRWEB) , ... August 01, 2015 , ... On March 19, 2015 Phil ... 2,175 mile trek of the Appalachian Trail by stepping off Springer Mountain and ... , On July 26th Phil reached Connecticut and was greeted by friends and family. ...
(Date:7/31/2015)... ... July 31, 2015 , ... When the weather turned ... debuted their new type of sod called Leisure Time(TM) zoysia . It's nicknamed ... , To commemorate introduction of this new type of sod, Super-Sod is giving away ...
(Date:7/31/2015)... ... 31, 2015 , ... Ticket Down is a reliable source for authentic tickets ... Grant Park. The festival will end on Sunday, August 2nd. , ... annual Lollapalooza. This unique and unbelievably popular festival attracts the best in entertainment ...
Breaking Medicine News(10 mins):Health News:Looking for HOPE? Healthy Home Has Evolv’d 2Health News:Looking for HOPE? Healthy Home Has Evolv’d 3Health News:Lume Wellness Launches Health and Fitness Tracker iPhone App 2Health News:Connecticut Executive Hikes Appalachian Trail to Raise Awareness About Recovery. 2Health News:Super-Sod Introduces Leisure Time(TM) Zoysia Grass with an Adirondack Chair Promotion 2Health News:Cheap 2015 Lollapalooza Tickets: Ticket Down Slashes Ticket Prices on Lollapalooza Tickets at Grant Park in Chicago for Saturday, August 1st and Sunday, August 2nd 2
(Date:7/31/2015)... Ill. , July 31, 2015 ... ® (NABP ® ) issued a report ... ensuring that action is taken against websites illegally ... Internet Drug Outlet Identification Program Progress Report ... , thousands of websites illegally distribute ...
(Date:7/31/2015)... CHICAGO , July 31, 2015 ... Market by Product (Moist Heat, Dry Heat, Ethylene Oxide, Filtration, ... & End User (Hospitals, Pharmaceuticals) - Global Forecast to 2020", ... reach around USD 6.1 Billion by 2020, at a CAGR ... 242 market data T ables and ...
(Date:7/31/2015)... 31, 2015  Semler Scientific, Inc. (Nasdaq: SMLR ... to healthcare insurers and physician groups, today reported financial ... "In the second quarter of 2015, ... growth of 54%, quarter over quarter revenue growth of ... base of FloChec® units of 18%," said Doug Murphy-Chutorian, ...
Breaking Medicine Technology:NABP Stresses Need for Accountability in Enforcing Internet Policies to Protect the Public From Illegal Online Drug Sellers 2NABP Stresses Need for Accountability in Enforcing Internet Policies to Protect the Public From Illegal Online Drug Sellers 3Sterilization Equipment Market is Expected to Reach USD 6.1 Billion by 2020 2Sterilization Equipment Market is Expected to Reach USD 6.1 Billion by 2020 3Sterilization Equipment Market is Expected to Reach USD 6.1 Billion by 2020 4Semler Reports Second Quarter 2015 Financial Results 2Semler Reports Second Quarter 2015 Financial Results 3Semler Reports Second Quarter 2015 Financial Results 4Semler Reports Second Quarter 2015 Financial Results 5Semler Reports Second Quarter 2015 Financial Results 6
Cached News: