Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Protein discovered that keeps hemoglobin in balance

13.06.2002


Research at The Children’s Hospital of Philadelphia may advance treatment of the blood disease thalassemia



Hematology researchers at The Children’s Hospital of Philadelphia have discovered a gene and its associated protein that may have major implications for red blood cell formation, specifically for hemoglobin, which carries oxygen in red blood cells. Understanding how this protein functions may eventually lead to novel treatments for the hemoglobin-related blood disease, thalassemia.

Thalassemia is a group of related inherited disorders that together are the most common single-gene disease known. The most severe form of the disease, beta thalassemia major, affects 300,000 patients worldwide.


Thalassemia results from an imbalance between two proteins in hemoglobin, called alpha and beta globin. An excess of either type of protein is toxic, causing thalassemia symptoms including poor growth, fatigue, bone damage, or skin ulcers. The newly found protein, alpha hemoglobin stabilizing protein (AHSP), binds to free alpha globin and prevents it from forming a precipitate that damages red blood cells.

“AHSP acts as a chaperone molecule – a chemical that helps another protein to fold or unfold,” said Mitchell Weiss, M.D., Ph.D., a pediatric hematologist at The Children’s Hospital of Philadelphia, and senior author of the paper, published in the June 13 issue of Nature. “Here it makes free alpha globin stable and prevents its deleterious effects.”

Dr. Weiss’ team suspected that AHSP, by preventing free alpha hemoglobin from precipitating within red blood cells, protects the cells from injury. To test that hypothesis, the researchers developed knockout mice, animals genetically engineered to lack the gene that produces AHSP. Those mice showed blood abnormalities similar to those found in mice with thalassemia.

AHSP’s protective role could explain how some patients who carry the genetic trait for beta thalassemia have mild disease and few symptoms even though their bodies produce more alpha than beta globin. By binding to free alpha globin, AHSP may protect the body from a dangerous accumulation of that protein. However, if AHSP does not function properly, the excess alpha globin precipitation may change milder or intermediate thalassemia into more severe disease.

This current research suggests that if physicians can deliver AHSP or a similar agent to patients with thalassemia, they may produce a new treatment for the disease. Severe cases are now treated with frequent blood transfusions that carry their own serious complications, such as excess iron. “If we can reduce the buildup of free alpha globin we may be able to lower the dose of transfusion needed, and improve patients’ quality of life,” added Dr. Weiss.


In addition to Dr. Weiss, co-authors of the article are Anthony J. Kihm, Ph.D., Yi Kong, Wei Hong, Ph.D., J. Eric Russell, M.D., Susan Rouda, Kazuhiko Adachi, Ph.D., and Gerd A. Blobel, M.D., Ph. D., all of Children’s Hospital’s Division of Hematology, and M. Celeste Simon, Ph.D., of the Howard Hughes Medical Institute and the University of Pennsylvania. The research was funded by the Cooley’s Anemia Foundation, the Unico Foundation and the National Institutes of Health.

Founded in 1855 as the nation’s first pediatric hospital, The Children’s Hospital of Philadelphia is ranked today as the best pediatric hospital in the nation by a comprehensive Child magazine survey. Its pediatric research program is among the largest in the country, ranking second in National Institutes of Health funding.

John Ascenzi | EurekAlert

More articles from Health and Medicine:

nachricht Tracking movement of immune cells identifies key first steps in inflammatory arthritis
23.01.2017 | Massachusetts General Hospital

nachricht Team discovers how bacteria exploit a chink in the body's armor
20.01.2017 | University of Illinois at Urbana-Champaign

All articles from Health and Medicine >>>

The most recent press releases about innovation >>>

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

Im Focus: Quantum optical sensor for the first time tested in space – with a laser system from Berlin

For the first time ever, a cloud of ultra-cold atoms has been successfully created in space on board of a sounding rocket. The MAIUS mission demonstrates that quantum optical sensors can be operated even in harsh environments like space – a prerequi-site for finding answers to the most challenging questions of fundamental physics and an important innovation driver for everyday applications.

According to Albert Einstein's Equivalence Principle, all bodies are accelerated at the same rate by the Earth's gravity, regardless of their properties. This...

Im Focus: Traffic jam in empty space

New success for Konstanz physicists in studying the quantum vacuum

An important step towards a completely new experimental access to quantum physics has been made at University of Konstanz. The team of scientists headed by...

Im Focus: How gut bacteria can make us ill

HZI researchers decipher infection mechanisms of Yersinia and immune responses of the host

Yersiniae cause severe intestinal infections. Studies using Yersinia pseudotuberculosis as a model organism aim to elucidate the infection mechanisms of these...

Im Focus: Interfacial Superconductivity: Magnetic and superconducting order revealed simultaneously

Researchers from the University of Hamburg in Germany, in collaboration with colleagues from the University of Aarhus in Denmark, have synthesized a new superconducting material by growing a few layers of an antiferromagnetic transition-metal chalcogenide on a bismuth-based topological insulator, both being non-superconducting materials.

While superconductivity and magnetism are generally believed to be mutually exclusive, surprisingly, in this new material, superconducting correlations...

Im Focus: Studying fundamental particles in materials

Laser-driving of semimetals allows creating novel quasiparticle states within condensed matter systems and switching between different states on ultrafast time scales

Studying properties of fundamental particles in condensed matter systems is a promising approach to quantum field theory. Quasiparticles offer the opportunity...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

Sustainable Water use in Agriculture in Eastern Europe and Central Asia

19.01.2017 | Event News

12V, 48V, high-voltage – trends in E/E automotive architecture

10.01.2017 | Event News

2nd Conference on Non-Textual Information on 10 and 11 May 2017 in Hannover

09.01.2017 | Event News

 
Latest News

Tracking movement of immune cells identifies key first steps in inflammatory arthritis

23.01.2017 | Health and Medicine

Electrocatalysis can advance green transition

23.01.2017 | Physics and Astronomy

New technology for mass-production of complex molded composite components

23.01.2017 | Process Engineering

VideoLinks
B2B-VideoLinks
More VideoLinks >>>