Beta Thalassemia News and Research

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Beta-thalassemia (ß-thalassemia) is a form of thalassemia due to mutations in the HBB gene on chromosome 11 , inherited in an autosomal recessive fashion.

Promising results in mice could prevent fatal iron buildup in humans

A new study shows that a protein found in blood alleviates anemia, a condition in which the body's tissues don't get enough oxygen from the blood. In this animal study, injections of the protein, known as transferrin, also protected against potentially fatal iron overload in mice with thalassemia, a type of inherited anemia that affects millions of people worldwide.

27 Jan 2010

Simple, five minute saliva test to determine baby's risk for more than 100 life-threatening genetic diseases

Genetic diseases like those seen in the new Harrison Ford movie "Extraordinary Measures" can now be prevented with a simple saliva test which is free with insurance for more than 100 million Americans.

22 Jan 2010

Hemaquest Pharmaceuticals' HQK-1001 holds promise for sickle cell disease and beta thalassemia

HemaQuest Pharmaceuticals presented data from new preclinical studies and early results from clinical trials of its lead drug candidate, HQK-1001, in sickle cell disease and beta thalassemia at the annual meeting of the American Society of Hematology in New Orleans.

8 Dec 2009

New data demonstrating the strength of Novartis' hematology portfolio to be presented

Novartis announced today that new data, including a late-breaking presentation on Tasigna® (nilotinib) 200 mg capsules in a form of chronic myeloid leukemia, demonstrate the strength of the company's hematology portfolio in advancing the care of patients.

2 Dec 2009

Gene therapy saves two children with adrenoleukodystrophy

The ELA association and Zinedine Zidane, its emblematic ambassador, are proud to announce a world premiere: the results regaring the gene therapy in adrenoleukodystrophy conducted in France have just been published in the prestigious journal Science. Two children have been treated and their diseases have been halted. The children are doing well, which is unexpected for a disease destroying the brain in a few months. This discovery opens up treatment perspectives for numerous widespread diseases.

6 Nov 2009

Avoiding spleen removal for Cooley's anemia sufferers

Researchers from Weill Cornell Medical College may have discovered the precise role of a gene in one of the world's most common blood disorders, beta-thalassemia, commonly known as Cooley's anemia. Along with sickle-cell anemia, Cooley's anemia is the most commonly inherited disease in the world, affecting many people of Mediterranean descent, and 20 out of every 100,000 African-Americans. The World Health Organization estimates that between 50,000-100,000 children are born with the disease each year.

27 May 2008

Variation in the BCL11A gene linked to moderated symptoms of beta-thalassemia

Beta-thalassemia is a serious, potentially life-threatening disease that affects red blood cells, cells that carry oxygen via hemoglobin throughout the body.

4 Feb 2008

Discovery of protein that plays a key role in the recycling of iron from blood

Scientists working in the only lab at MIT doing hematology research have uncovered a protein that plays a key role in the recycling of iron from blood. Their work, described in the October 11 Journal of Clinical Investigation, could lead to new therapies for certain inherited blood disorders such as beta-thalassemia, a condition that causes chronic anemia.

12 Oct 2007

New research may lead to future gene therapies for patients with sickle cell anemia and beta-thalassemia

Virginia Commonwealth University researchers studying hemoglobin genes, mutations of which play a role in genetic blood disorders like sickle cell anemia and beta-thalassemia, have identified two proteins that are responsible for regulating overlapping groups of genes during the development of red blood cells.

7 Aug 2007

First case of successful ovarian tissue transplantation between two, nonidentical sisters

A woman, whose ovaries had failed due to damage caused by chemotherapy and radiotherapy, has received a successful ovarian transplant from her genetically non-identical sister.

3 Aug 2007

Woman has successful ovarian transplant from her sister

A woman whose ovaries were damaged by chemotherapy and radiotherapy, has received a successful ovarian transplant from her sister.

1 Aug 2007

Mechanism driving iron overload in thalassemia identified

Led by researchers at Weill Cornell Medical College in New York City, an international group of scientists has pinpointed a specific genetic relationship as the cause of dangerous iron overload in persons with a form of the inherited blood disease, beta-thalassemia.

14 Feb 2007

New drug may revolutionize how chronic iron overload is treated in chronic anemia patients

Those with severe chronic anemias need frequent blood transfusions to remain healthy, but such frequent transfusions can cause a potentially deadly buildup of iron in the body, leading to heart and liver failure. The traditional treatment to remove excess iron is so onerous that many patients choose to forgo it, putting their own lives at risk. The results of an international study on deferasirox, a new drug that may revolutionize the way chronic iron overload is treated, will be published in the May 1, 2006, issue of Blood, the official journal of the American Society of Hematology.

26 Apr 2006

Researchers identify the role of a protein in hemoglobin gene silencing

Virginia Commonwealth University Massey Cancer Center researchers have identified the role of a protein in hemoglobin gene silencing that may one day be a potential target for the treatment of genetic blood disorders like sickle-cell anemia and beta-thalassemia on the molecular level.

11 Apr 2006

DNA repair kit announced by scientists

A study by scientists at Sangamo BioSciences, Inc. has demonstrated the use of the Company's zinc finger DNA-binding protein (ZFP) technology to achieve highly efficient, permanent correction of a disease-causing gene in primary human cells.

5 Apr 2005

New simple and cost-effective method of diagnosing hereditary genetic disorders

A technique called size-fractionation performed on a sample of the mother's blood allows researchers to identify fetal DNA molecules separate from maternal DNA as a way to determine which pregnancies may be at risk for genetic disorders, according to a preliminary communication in the February 16 issue of JAMA, a theme issue on medical applications of biotechnology.

15 Feb 2005

New advances in sickle cell disease

Excruciating pain episodes, recurrent pneumonias, strokes, severe infections, chronic hemolytic anemia, and secondary pulmonary hypertension are common complications of sickle cell disease, a life-threatening inherited defect in blood that causes normally disc-shaped red blood cells to take on a sickle shape.

6 Dec 2004

Technique for genetically modifying blood stem cells brings cure for blood diseases closer

The condition of mice with a genetic blood disease called beta-thalassemia improved significantly following treatment of their blood forming cells with a gene that enabled them to produce the type of hemoglobin normally found only in the fetus.

12 Oct 2004