Featured Research

from universities, journals, and other organizations

Gene Therapy Trial Approved For Rare Anemia

Date:
January 25, 1999
Source:
University Of North Carolina At Chapel Hill
Summary:
A gene therapy clinical trial for Fanconi anemia, a fatal inherited blood disease, is set to begin at the University of North Carolina at Chapel Hill. Currently, the best survival hope for Fanconi patients is a bone marrow transplant from a donor with a perfect tissue match. However, for most patients, a suitable donor cannot be found in time.

CHAPEL HILL, N.C. -- A gene therapy clinical trial for Fanconi anemia, a fatal inherited blood disease, is set to begin at the University of North Carolina at Chapel Hill.

Related Articles


Currently, the best survival hope for Fanconi patients is a bone marrow transplant from a donor with a perfect tissue match. However, for most patients, a suitable donor cannot be found in time.

"In people with Fanconi anemia, the bone marrow fails. It's a failure state like liver failure or heart failure," says Dr. Christopher E. Walsh, assistant professor of medicine at UNC-CH School of Medicine and clinical director of the university's Gene Therapy Center. "The bone marrow failure associated with this anemia leads to severe reductions in the numbers of circulating blood cells, including red cells, all types of white cells and platelets, which are crucial to clotting."

According to Walsh, the majority of individuals with Fanconi anemia are unaware they have the disease. "By and large, most patients come to medical attention because they have something wrong with their blood cell counts," he says. However, he notes that in some people with low cell counts, certain physical characteristics may raise medical suspicion of Fanconi. These can include short stature, finger deformities, and hyperpigmentation of the skin -- so-called "café au lait" spots.

The phase I clinical trial will test the safety and feasibility of correcting the blood disorder by transferring a cloned corrective gene called FAA into a patient's very primitive bone marrow cells - "progenitor" stem cells from which all blood cells are formed. These progenitor cells, which have been removed from bone marrow for the gene transfer, are then re-infused into the patient. Compelling evidence from prior laboratory studies indicate that the corrective gene, when packaged with a retrovirus, can enter the cell nucleus and enable formation of the millions of healthy blood cells that are needed every day.

Fanconi anemia affects an estimated 2,000 to 3,000 people in the United States and Europe. Given these relatively small numbers, Walsh says he hopes to enroll at least 10 patients for the trial.

"Fanconi anemia is a perfect candidate disease for the use of gene therapy," Walsh says. "What we learn from here can be applied to other disorders involving a single gene."


Story Source:

The above story is based on materials provided by University Of North Carolina At Chapel Hill. Note: Materials may be edited for content and length.


Cite This Page:

University Of North Carolina At Chapel Hill. "Gene Therapy Trial Approved For Rare Anemia." ScienceDaily. ScienceDaily, 25 January 1999. <www.sciencedaily.com/releases/1999/01/990125073403.htm>.
University Of North Carolina At Chapel Hill. (1999, January 25). Gene Therapy Trial Approved For Rare Anemia. ScienceDaily. Retrieved December 21, 2014 from www.sciencedaily.com/releases/1999/01/990125073403.htm
University Of North Carolina At Chapel Hill. "Gene Therapy Trial Approved For Rare Anemia." ScienceDaily. www.sciencedaily.com/releases/1999/01/990125073403.htm (accessed December 21, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Sunday, December 21, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

The Best Tips to Curb Holiday Carbs

The Best Tips to Curb Holiday Carbs

Buzz60 (Dec. 19, 2014) — It's hard to resist those delicious but fattening carbs we all crave during the winter months, but there are some ways to stay satisfied without consuming the extra calories. Vanessa Freeman (@VanessaFreeTV) has the details. Video provided by Buzz60
Powered by NewsLook.com
Sierra Leone Bikers Spread the Message to Fight Ebola

Sierra Leone Bikers Spread the Message to Fight Ebola

AFP (Dec. 19, 2014) — More than 100 motorcyclists hit the road to spread awareness messages about Ebola. Nearly 7,000 people have now died from the virus, almost all of them in west Africa, according to the World Health Organization. Video provided by AFP
Powered by NewsLook.com
Researchers Test Colombian Village With High Alzheimer's Rates

Researchers Test Colombian Village With High Alzheimer's Rates

AFP (Dec. 19, 2014) — In Yarumal, a village in N. Colombia, Alzheimer's has ravaged a disproportionately large number of families. A genetic "curse" that may pave the way for research on how to treat the disease that claims a new victim every four seconds. Duration: 02:42 Video provided by AFP
Powered by NewsLook.com
The Best Protein-Filled Foods to Energize You for the New Year

The Best Protein-Filled Foods to Energize You for the New Year

Buzz60 (Dec. 19, 2014) — The new year is coming and nothing will energize you more for 2015 than protein-filled foods. Fitness and nutrition expert John Basedow (@JohnBasedow) gives his favorite high protein foods that will help you build muscle, lose fat and have endless energy. Video provided by Buzz60
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:

Strange & Offbeat Stories

 

Health & Medicine

Mind & Brain

Living & Well

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