Featured Research

from universities, journals, and other organizations

New Gene Therapy Shows Promise For Hemophiliacs

Date:
January 22, 2002
Source:
University Of Minnesota
Summary:
University of Minnesota researchers have demonstrated that it may be possible to treat hemophilia A through the use of human blood outgrowth endothelial cells, or BOECs, as a vehicle for gene therapy.

MINNEAPOLIS / ST. PAUL -- University of Minnesota researchers have demonstrated that it may be possible to treat hemophilia A through the use of human blood outgrowth endothelial cells, or BOECs, as a vehicle for gene therapy. Lead researcher Robert Hebbel, M.D., the George Clark Professor and vice chair for research, department of medicine, reports that the use of BOECs has "resulted in sustained and therapeutic levels of factor 8 (FVIII) in mice and may comprise an effective therapeutic strategy for use in gene therapy for humans with hemophilia A." The results of this gene therapy, published in the January 15 issue of Blood, may pave the way for testing the therapy in humans.

Hemophilia A is an inherited blood disorder that affects one in 5,000 males and results in spontaneous bleeding due to the lack of coagulation factor 8 (FVIII) in the blood. Because hemophilia is such a severe disease, and because it is caused by absence of a replaceable blood protein, there has been a high level of interest in developing a gene therapy approach for its treatment. While there are currently some ongoing Phase I clinical studies of gene therapy for humans for hemophilia B (a related disorder that affects a different coagulation factor), development of gene therapy for hemophilia A has lagged behind. Current clinical therapy generally calls for the replacement of factor VIII after the patient has had some trouble.

The ex vivo process devised by Hebbel utilizes BOECs as carriers for gene therapy. The process begins with a blood sample taken from the patient as a source for autologous BOECs. (This use of autologous cells eliminates the concern for immunological compatibility or disease-tainted blood sources). The BOECs are separated from the blood and cultured in the lab and then transfected with a nonviral plasmid vector carrying the gene for human coagulation factor VIII. After allowing them to further expand, those stably transfected BOECs are then given back to the patient, where they seek out the bone marrow and the spleen and start to divide.

"The results we've seen in our mouse studies show long-term attainment of robust levels of factor VIII that would be therapeutic in a human," said Hebbel. "While we typically would call a five percent increase in FVIII level a good result, we've seen over a hundred percent of normal amounts of factor VIII in these mice. The results are very exciting and provide a strong rationale for proceeding to preclinical trials in dogs and clinical trials in humans."

The development of BOEC technology for gene therapy of hemophilia has been supported by the National Insitutes of Health and the Octagen Corp.


Story Source:

The above story is based on materials provided by University Of Minnesota. Note: Materials may be edited for content and length.


Cite This Page:

University Of Minnesota. "New Gene Therapy Shows Promise For Hemophiliacs." ScienceDaily. ScienceDaily, 22 January 2002. <www.sciencedaily.com/releases/2002/01/020122072306.htm>.
University Of Minnesota. (2002, January 22). New Gene Therapy Shows Promise For Hemophiliacs. ScienceDaily. Retrieved September 19, 2014 from www.sciencedaily.com/releases/2002/01/020122072306.htm
University Of Minnesota. "New Gene Therapy Shows Promise For Hemophiliacs." ScienceDaily. www.sciencedaily.com/releases/2002/01/020122072306.htm (accessed September 19, 2014).

Share This



More Health & Medicine News

Friday, September 19, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Could Grief Affect The Immune Systems Of Senior Citizens?

Could Grief Affect The Immune Systems Of Senior Citizens?

Newsy (Sep. 19, 2014) The study found elderly people are much more likely to become susceptible to infection than younger adults going though a similar situation. Video provided by Newsy
Powered by NewsLook.com
Jury Delivers Verdict in Salmonella Trial

Jury Delivers Verdict in Salmonella Trial

AP (Sep. 19, 2014) A federal jury has convicted three people in connection with an outbreak of salmonella poisoning five years ago that sickened hundreds of people and was linked to a number of deaths. (Sept. 19) Video provided by AP
Powered by NewsLook.com
How The 'Angelina Jolie Effect' Increased Cancer Screenings

How The 'Angelina Jolie Effect' Increased Cancer Screenings

Newsy (Sep. 19, 2014) Angelina's Jolie's decision to undergo a preventative mastectomy in 2013 inspired many women to seek early screenings for the disease. Video provided by Newsy
Powered by NewsLook.com
The Cost of Ebola

The Cost of Ebola

Reuters - Business Video Online (Sep. 18, 2014) As Sierra Leone prepares for a three-day "lockdown" in its latest bid to stem the spread of Ebola, Ciara Lee looks at the financial implications of fighting the largest ever outbreak of the disease. Video provided by Reuters
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins