Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

New research demonstrates bone-marrow derived stem cells can reverse genetic kidney disease

26.04.2006
Animal study shows promise for treatment of Alport syndrome

The discovery that bone-marrow derived stem cells can regenerate damaged renal cells in an animal model of Alport syndrome provides a potential new strategy for managing this inherited kidney disease and offers the first example of how stem cells may be useful in repairing basement membrane matrix defects and restoring organ function.

Led by researchers at Beth Israel Deaconess Medical Center (BIDMC), the findings are described in the Proceedings of the National Academy of Sciences (PNAS), which appears on-line the week of April 24, 2006.

Symptoms of Alport syndrome, the second most common genetic cause of kidney failure, usually appear in children, affecting the kidneys’ filtration system and typically leading to end-stage renal disease in the patient’s teens, 20s or 30s. The disease additionally causes deafness in some patients.

"This is one of 31 human diseases that occur because of genetic defects in the body’s extracellular matrix and basement membrane proteins, " explains the study’s senior author Raghu Kalluri, PhD, chief of the division of matrix biology at BIDMC and associate professor of medicine at Harvard Medical School.

Present throughout the body, the extracellular matrix (ECM) is made of collagens, proteogylcans, noncollagenous glycoproteins and in some tissues, elastin fibers. The ECM serves a unique role by constructing a "scaffold" for cells, thereby maintaining the structural integrity of many tissues.

"In normal kidneys, a specialized form of extracellular matrix known as the glomerular basement membrane [GBM], composed primarily of type IV collagen, is the key component of the blood filtration apparatus," says Kalluri.

However, in patients with Alport syndrome, mutations in three different type IV collagen genes cause structural damage to the GBM, leading to a breakdown of the organ’s filtration system, and resulting in the seepage of blood and protein into the urine and eventual kidney failure.

"With no known cure, treatment options for Alport syndrome are limited to kidney transplantation or lifelong dialysis," he adds. "Our lab set out to determine if bone marrow-derived stem cell therapy might provide another treatment option."

Using a mouse model of the disease, in which COL4A3 – one of the three type IV collagen genes that is mutated in Alport patients -- had been removed, the researchers transplanted allogenic bone marrow into the knockout mouse.

According to Kalluri, within a period of about four weeks, the investigators found that approximately 10 percent of the transplanted cells had incorporated into the damaged regions of the kidney glomeruli in the knockout mouse, emerging as healthy renal cells (podocytes and mesangial cells). This led to clear improvement in the animal’s kidney function and repair of the glomerular architecture defects.

"These results offer a possible therapeutic opportunity for both children and adults with Alport syndrome," he says. In addition, the new findings demonstrate that bone-marrow-derived stem cells can be used to repair extracellular/basement membrane defects, and according to Kalluri, the researchers’ next step will be to see if the use of embryonic stem cells and circulating adult stem cells will achieve the same function.

"This new evidence offers hope that this is a treatment strategy that can be explored for other diseases that result from extracellular matrix defects," he notes.

Study coauthors include BIDMC researchers Hikaru Sugimoto, MD, PhD, Thomas Mundel, MD, PhD, Malin Sund, MD, PhD, and Liang Xie, PhD, and Dominic Cosgrove of Boystown National Research Hospital, Omaha, Nebraska.

Bonnie Prescott | EurekAlert!
Further information:
http://www.bidmc.harvard.edu

More articles from Life Sciences:

nachricht A Map of the Cell’s Power Station
18.08.2017 | Albert-Ludwigs-Universität Freiburg im Breisgau

nachricht On the way to developing a new active ingredient against chronic infections
18.08.2017 | Deutsches Zentrum für Infektionsforschung

All articles from Life Sciences >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Fizzy soda water could be key to clean manufacture of flat wonder material: Graphene

Whether you call it effervescent, fizzy, or sparkling, carbonated water is making a comeback as a beverage. Aside from quenching thirst, researchers at the University of Illinois at Urbana-Champaign have discovered a new use for these "bubbly" concoctions that will have major impact on the manufacturer of the world's thinnest, flattest, and one most useful materials -- graphene.

As graphene's popularity grows as an advanced "wonder" material, the speed and quality at which it can be manufactured will be paramount. With that in mind,...

Im Focus: Exotic quantum states made from light: Physicists create optical “wells” for a super-photon

Physicists at the University of Bonn have managed to create optical hollows and more complex patterns into which the light of a Bose-Einstein condensate flows. The creation of such highly low-loss structures for light is a prerequisite for complex light circuits, such as for quantum information processing for a new generation of computers. The researchers are now presenting their results in the journal Nature Photonics.

Light particles (photons) occur as tiny, indivisible portions. Many thousands of these light portions can be merged to form a single super-photon if they are...

Im Focus: Circular RNA linked to brain function

For the first time, scientists have shown that circular RNA is linked to brain function. When a RNA molecule called Cdr1as was deleted from the genome of mice, the animals had problems filtering out unnecessary information – like patients suffering from neuropsychiatric disorders.

While hundreds of circular RNAs (circRNAs) are abundant in mammalian brains, one big question has remained unanswered: What are they actually good for? In the...

Im Focus: RAVAN CubeSat measures Earth's outgoing energy

An experimental small satellite has successfully collected and delivered data on a key measurement for predicting changes in Earth's climate.

The Radiometer Assessment using Vertically Aligned Nanotubes (RAVAN) CubeSat was launched into low-Earth orbit on Nov. 11, 2016, in order to test new...

Im Focus: Scientists shine new light on the “other high temperature superconductor”

A study led by scientists of the Max Planck Institute for the Structure and Dynamics of Matter (MPSD) at the Center for Free-Electron Laser Science in Hamburg presents evidence of the coexistence of superconductivity and “charge-density-waves” in compounds of the poorly-studied family of bismuthates. This observation opens up new perspectives for a deeper understanding of the phenomenon of high-temperature superconductivity, a topic which is at the core of condensed matter research since more than 30 years. The paper by Nicoletti et al has been published in the PNAS.

Since the beginning of the 20th century, superconductivity had been observed in some metals at temperatures only a few degrees above the absolute zero (minus...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

Call for Papers – ICNFT 2018, 5th International Conference on New Forming Technology

16.08.2017 | Event News

Sustainability is the business model of tomorrow

04.08.2017 | Event News

Clash of Realities 2017: Registration now open. International Conference at TH Köln

26.07.2017 | Event News

 
Latest News

A Map of the Cell’s Power Station

18.08.2017 | Life Sciences

Engineering team images tiny quasicrystals as they form

18.08.2017 | Physics and Astronomy

Researchers printed graphene-like materials with inkjet

18.08.2017 | Materials Sciences

VideoLinks
B2B-VideoLinks
More VideoLinks >>>