Gene Therapy Helps Patients with Hemophilia

A single dose of an experimental gene therapy boosted production of a missing blood-clotting factor in people with hemophilia, a new study shows. The therapy might give patients a long-term solution for preventing dangerous bleeding episodes.

Hemophilia is a rare, inherited disorder in which blood is unable to clot normally. As a result, people with hemophilia tend to bleed more than others after injury. They may also bleed without warning inside their bodies. This bleeding can damage organs and tissues and may be life threatening.

The main treatment, called replacement therapy, involves infusing missing clotting factor proteins into the patient's bloodstream. These proteins help to restore normal blood clotting. But replacement therapy often must be repeated regularly, and it carries other risks.

To find an alternative, researchers from the University College London and St. Jude Children's Research Hospital led a team that investigated a potential gene therapy approach. The research, funded in part by NIH's National Heart, Lung and Blood Institute (NHLBI), focused on hemophilia B. This uncommon form of the disease affects about 1 in 5 patients with hemophilia. Hemophilia B is caused by defects in the gene that codes for human clotting factor IX.

Scientists packaged a normal factor IX gene into a modified adeno-associated virus that targets liver cells. The liver is the only site that can produce a form of factor IX needed for the clotting process. The virus—acting as a delivery vehicle, or vector—was designed to transport the normal gene into liver cells and launch production of factor IX.


 Get The Latest By Email

Weekly Magazine Daily Inspiration

6 men with severe hemophilia B received one-time intravenous infusions of the gene vector at varying doses. Prior to the study, the men were producing clotting factor IX at less than 1% of normal levels. They had been receiving the standard treatment for their condition: infusions of manufactured factor IX protein several times a month.

After gene therapy, each patient generated factor IX at between 2% and 11% of normal levels. In the short-term follow-up period (6 to 16 months), 4 of the 6 men no longer needed factor IX infusions for routine bleeding.

Hemophilia has long been one of the disorders thought most likely to be correctible with gene therapy, but previous approaches to deliver the gene have been disappointing,” says NHLBI Acting Director Dr. Susan B. Shurin. Results from this study represent a promising step toward making gene therapy a viable treatment option for hemophilia B. If future studies support these findings, it would bring a significant improvement in the quality of life for those living with the disease.

  • What is Hemophilia?
    http://www. nhlbi. nih.

  • http://ghr. nlm. nih.

  • http://clinicaltrials. gov/ct2/show/NCT00979238?

Article Source:
 http://www.nih.gov/researchmatters/december2011/12192011hemophilia.htm

AVAILABLE LANGUAGES

English Afrikaans Arabic Chinese (Simplified) Chinese (Traditional) Danish Dutch Filipino Finnish French German Greek Hebrew Hindi Hungarian Indonesian Italian Japanese Korean Malay Norwegian Persian Polish Portuguese Romanian Russian Spanish Swahili Swedish Thai Turkish Ukrainian Urdu Vietnamese

follow InnerSelf on

facebook icontwitter iconyoutube iconinstagram iconpintrest iconrss icon

 Get The Latest By Email

Weekly Magazine Daily Inspiration

Tuesday, 27 April 2021 08:56

Peas, lentils, chickpeas, beans and peanuts: if it comes in a pod then chances are it’s a legume. These unassuming food crops have a special ability that makes them fairly unique in the plant...

Wednesday, 28 April 2021 08:51

Insects are attracted to landscapes where flowering plants of the same species are grouped together and create big blocks of color, according to new research.

Thursday, 01 April 2021 16:24

  Flamenco dancing is a delight to watch. A good flamenco dancer exudes an exuberant self-confidence that we, the audience, absorb. The whole dance has a quality of proud self-assurance and...

Thursday, 15 April 2021 13:22

Skin brushing is a highly effective technique for cleansing the lymphatic system. Topics covered in this article: Benefits of Skin Brushing; What type of skin brush is the best to use; How to Brush...

Wednesday, 28 April 2021 08:57

Replenishing antioxidants in the body may help protect against oxidative stress and lower the risk of cancer

Saturday, 01 May 2021 08:12

High-intensity interval training (HIIT) workouts have become popular in recent years for a number of reasons. They don’t require as much time as a regular workout (some can take as little as 10...

New Attitudes - New Possibilities

InnerSelf.comClimateImpactNews.com | InnerPower.net
MightyNatural.com | WholisticPolitics.com | InnerSelf Market
Copyright ©1985 - 2021 InnerSelf Publications. All Rights Reserved.