Featured Research

from universities, journals, and other organizations

Transplanted Cells May Hold The Key To Curing Hemophilia A

Date:
February 19, 2008
Source:
Albert Einstein College of Medicine
Summary:
Transplanted cells can cure hemophilia A (the most common form of the disease) in an animal model, new research suggests. Researchers transplanted healthy liver endothelial cells from donor mice into a mouse model of the disease. Their findings also overturned conventional wisdom regarding which cells produce factor VIII, the crucial clotting protein that people with type A hemophilia lack.

Scientists at the Albert Einstein College of Medicine of Yeshiva University have shown for the first time that transplanted cells can cure hemophilia A (the most common form of the disease) in an animal model. Hemophilia A affects one in 10,000 males and is characterized by recurrent, spontaneous bleeding that can cause disability or death. It has, most famously, affected British and Russian nobility, along with countless others.

The Einstein team, led by Dr. Sanjeev Gupta, cured hemophilia A by transplanting healthy liver endothelial cells from donor mice into a mouse model of the disease. In curing these mice, the Einstein researchers also overturned conventional wisdom regarding which cells produce factor VIII, the crucial clotting protein that is lacking in people with type A hemophilia.

"We've known for three decades that factor VIII is produced in the liver, but precisely where has been controversial," says Dr. Gupta, a professor in the departments of medicine and pathology at Einstein. "It was assumed that factor VIII was made by the hepatocytes--cells that perform many of the liver's functions and comprise most of its bulk. But our research had suggested that the primary sources were special endothelial cells that line the sinusoids, the liver's blood-filled spaces. We did this study to confirm the role of liver endothelial cells in producing factor VIII and to see if transplanting them from a healthy donor liver could correct hemophilia A in an animal model."

The donor mice used by Dr. Gupta's team had been genetically engineered so that their endothelial cells expressed the gene for green fluorescent protein. This allowed the researchers to see whether the liver endothelial cells--isolated from donor livers and then injected into a vein leading into the liver of recipient hemophilia A mice--could successfully engraft within the recipients' livers. Before transplantation, to "make room" for the donated cells to engraft and proliferate, the researchers gave recipient mice a dose of the toxic chemical monocrotaline to deplete their native liver endothelial cells.

"Three months after transplantation, when we examined the livers of recipient mice, we found that the transplanted cells had engrafted and increased in number," says Dr. Antonia Follenzi, an instructor in pathology at Einstein and the lead author of the study. "Even more important, those mice with the most transplanted cells in their livers produced factor VIII in sufficient amounts to completely correct their hemophilia A."

Efforts up until now to cure hemophilia--ranging from faith healing by the Russian monk Rasputin early in the 20th century to clinical trials in the U.S. using gene therapy--have had limited or no success. While the Einstein study marks a significant advance against hemophilia A, Dr. Gupta cautions that more work is necessary before liver-cell transplants can be attempted on people. "An important need," he says, "is for safer alternatives to monocrotaline, the chemical we gave to recipient mice to create space for healthy donor liver cells. Fortunately, several drugs already in clinical use hold promise in that regard."

The principle established by this study--selectively injuring native endothelial cells and replacing them with healthy donor endothelial cells--might also help in treating hemophilia B (the less common form of the disorder) and, more broadly, aid in "tissue engineering" in the many other areas of the body where endothelial cells are found, says Dr. Gupta. For example, he notes that endothelial cells isolated from blood vessels and transplanted into the heart could help in treating cardiovascular disease. In addition, he says that it may be possible to deliver therapeutic substances into the liver and other organs by combining gene therapy with the cell-transplantation approach used in this study.

The study appears online February 14 in the Journal of Clinical Investigation. Daniel Benten and Phyllis Novikoff of Einstein were also involved in this study. Other collaborators were Louisa Faulkner and Sanj Raut of the National Institute for Biological Standards and Control, Hertfordshire, United Kingdom. The study was funded in part by The National Institute of Diabetes and Digestive and Kidney Diseases.


Story Source:

The above story is based on materials provided by Albert Einstein College of Medicine. Note: Materials may be edited for content and length.


Cite This Page:

Albert Einstein College of Medicine. "Transplanted Cells May Hold The Key To Curing Hemophilia A." ScienceDaily. ScienceDaily, 19 February 2008. <www.sciencedaily.com/releases/2008/02/080215103319.htm>.
Albert Einstein College of Medicine. (2008, February 19). Transplanted Cells May Hold The Key To Curing Hemophilia A. ScienceDaily. Retrieved October 23, 2014 from www.sciencedaily.com/releases/2008/02/080215103319.htm
Albert Einstein College of Medicine. "Transplanted Cells May Hold The Key To Curing Hemophilia A." ScienceDaily. www.sciencedaily.com/releases/2008/02/080215103319.htm (accessed October 23, 2014).

Share This



More Health & Medicine News

Thursday, October 23, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Ebola Fears Keep Guinea Hospitals Empty

Ebola Fears Keep Guinea Hospitals Empty

AP (Oct. 23, 2014) Fears of Ebola are keeping doctors and patients alike away from hospitals in the West African nation of Guinea. (Oct. 23) Video provided by AP
Powered by NewsLook.com
Orthodontist Mom Jennifer Salzer on the Best Time for Braces

Orthodontist Mom Jennifer Salzer on the Best Time for Braces

Working Mother (Oct. 22, 2014) Is your child ready? Video provided by Working Mother
Powered by NewsLook.com
U.S. Issues Ebola Travel Restrictions, Are Visa Bans Next?

U.S. Issues Ebola Travel Restrictions, Are Visa Bans Next?

Newsy (Oct. 22, 2014) Now that the U.S. is restricting travel from West Africa, some are dropping questions about a travel ban and instead asking about visa bans. Video provided by Newsy
Powered by NewsLook.com
More People Diagnosed With TB In 2013, But There's Good News

More People Diagnosed With TB In 2013, But There's Good News

Newsy (Oct. 22, 2014) The World Health Organizations says TB numbers rose in 2013, but it's partly due to better detection and more survivors. Video provided by Newsy
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