Featured Research

from universities, journals, and other organizations

Liver-cell transplants show promise in reversing genetic disease affecting liver and lungs

Date:
April 23, 2011
Source:
Albert Einstein College of Medicine
Summary:
Transplanting cells from healthy adult livers may work in treating a genetic liver-lung disorder that affects millions of people worldwide, according to a new animal study.

Transplanting cells from healthy adult livers may work in treating a genetic liver-lung disorder that affects millions of people worldwide, according to an animal study in the April 18 online edition of the Journal of Clinical Investigation. Jayanta Roy-Chowdhury, M.D. , professor of medicine and of genetics at Albert Einstein College of Medicine of Yeshiva University, is the study's senior author.

Related Articles


The genetic disorder, alpha-1 antitrypsin (AAT) deficiency, is the most common potentially lethal hereditary disease among Caucasians, affecting an estimated 100,000 people in the United States and 3.4 million people worldwide. AAT is a protein made by the liver that is essential for lung health. In AAT deficiency, the liver produces a misshapen form of AAT that cannot enter the bloodstream and instead gets stuck inside liver cells, causing two major problems:

  • AAT accumulates in the liver, leading to fibrosis (development of scar tissue) and liver failure;
  • Too little AAT reaches the lungs, where it's needed to rein in elastase, an enzyme produced by white blood cells. Elastase helps kill bacteria in the lungs, but uncontrolled elastase activity can damage lung tissue and lead to severe emphysema (chronic obstructive pulmonary disease).

In the study, Dr. Roy-Chowdhury and his colleagues tested cell therapy on transgenic mice whose liver cells (hepatocytes) had been engineered to produce mutant human AAT, resulting in liver fibrosis. When the mice were given infusions of hepatocytes harvested from the livers of healthy mice, the transplanted cells proliferated in the host liver, progressively replacing diseased hepatocytes. Most importantly, said Dr. Roy-Chowdhury, the transplanted cells reversed the fibrosis that had developed.

Current therapy for AAT deficiency consists of life-long injections of a genetically engineered version of AAT called Prolastin. "This very expensive therapy slows progression of the lung disease in some patients but does not have any beneficial effect on the liver disease," said Dr. Roy-Chowdhury. The only other therapy for AAT deficiency is combined lung-liver transplantation, which is reserved for the sickest patients.

"These promising results in animals indicate that it may be worthwhile to investigate the usefulness of hepatocyte transplantation for AAT deficiency as well as a variety of other inherited liver-based disorders," said Dr. Roy-Chowdhury.

The title of the paper is "Spontaneous hepatic repopulation in transgenic mice expressing mutant human alpha 1-anti-trypsin by wildtype donor hepatocytes." Other Einstein researchers involved in the study are Jianqiang Ding, M.D., Ph.D., Namita Roy-Chowdhury, Ph.D., Yesim Avsar, M.D., and Chandan Guha, M.B., B.S., Ph.D. The research was supported by the National Institutes of Health, the National Institute of Diabetes, Digestive and Kidney Diseases, the New York Stem Cell Foundation, the Oxalosis and Hyperoxaluria Foundation, and the United States Department of Defense.


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.


Journal Reference:

  1. Jianqiang Ding, Govardhana R. Yannam, Namita Roy-Chowdhury, Tunda Hidvegi, Hesham Basma, Stephen I. Rennard, Ronald J. Wong, Yesim Avsar, Chandan Guha, David H. Perlmutter, Ira J. Fox, Jayanta Roy-Chowdhury. Spontaneous hepatic repopulation in transgenic mice expressing mutant human α1-antitrypsin by wild-type donor hepatocytes. Journal of Clinical Investigation, 2011; DOI: 10.1172/JCI45260

Cite This Page:

Albert Einstein College of Medicine. "Liver-cell transplants show promise in reversing genetic disease affecting liver and lungs." ScienceDaily. ScienceDaily, 23 April 2011. <www.sciencedaily.com/releases/2011/04/110422115848.htm>.
Albert Einstein College of Medicine. (2011, April 23). Liver-cell transplants show promise in reversing genetic disease affecting liver and lungs. ScienceDaily. Retrieved April 25, 2015 from www.sciencedaily.com/releases/2011/04/110422115848.htm
Albert Einstein College of Medicine. "Liver-cell transplants show promise in reversing genetic disease affecting liver and lungs." ScienceDaily. www.sciencedaily.com/releases/2011/04/110422115848.htm (accessed April 25, 2015).

Share This


More From ScienceDaily



More Health & Medicine News

Saturday, April 25, 2015

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

85 Killed in Niger by Meningitis Since Start of Year

85 Killed in Niger by Meningitis Since Start of Year

AFP (Apr. 24, 2015) A meningitis outbreak in Niger has killed 85 people since the start of the year prompting authorities to close schools in the capital Niamey until Monday. Video provided by AFP
Powered by NewsLook.com
C-Section Births a Trend in Brazil

C-Section Births a Trend in Brazil

AFP (Apr. 24, 2015) More than half of Brazil&apos;s babies are born via cesarean section, as mothers and doctors opt for a faster and less painful experience despite the health risks. Duration: 02:02 Video provided by AFP
Powered by NewsLook.com
Anti-Malaria Jab Hope

Anti-Malaria Jab Hope

Reuters - News Video Online (Apr. 24, 2015) The world&apos;s first anti-malaria vaccine could get the go-ahead for use in Africa from October if approved by international regulators. Paul Chapman reports. Video provided by Reuters
Powered by NewsLook.com
3D Food Printing: The Meal of the Future?

3D Food Printing: The Meal of the Future?

AP (Apr. 23, 2015) Developers of 3D food printing hope the culinary technology will revolutionize the way we cook and eat. (April 23) Video provided by AP
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