Cooley's Anemia News and Research

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Cooley's anemia is a genetic blood disease that results in an inadequate production of hemoglobin, the essential oxygen-carrying substance in blood. This causes severe anemia that begins shortly after birth.

Base editing shows promise for treating sickle cell disease and beta thalassemia

Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease (SCD) and beta thalassemia.

3 Jul 2023

Scientists show how cellular hypoxia response increases fetal hemoglobin expression in adults

Scientists at St. Jude Children's Research Hospital have shown how a protein responsible for adapting to low oxygen conditions (hypoxia), causes increased expression of fetal hemoglobin (HbF) in adults.

12 Oct 2022

Researchers develop new strategy to treat sickle cell disease and beta thalassemia

Researchers at Dana-Farber/Boston Children's Cancer and Blood Disorders Center and the University of Massachusetts Medical School have developed a strategy to treat two of the most common inherited blood diseases -- sickle cell disease and beta thalassemia -- applying CRISPR-Cas9 gene editing to patients' own blood stem cells.

29 Mar 2019

People with iron overload disease are more vulnerable to Vibrio vulnificus infections

Every summer, the news reports on a bacterium called Vibrio vulnificus found in warm saltwater that causes people to get sick, or die, after they eat raw tainted shellfish or when an open wound comes in contact with seawater.

15 Jan 2015

Findings could lead to treatments for blood disorders associated with iron deficiencies and overloads

A UCLA research team has discovered a new hormone called erythroferrone, which regulates the iron supply needed for red blood-cell production.

2 Jun 2014

Atpif1 gene regulates heme synthesis in red blood cell formation

Scientists at the University of Georgia, Harvard Medical School and the University of Utah have discovered a new gene that regulates heme synthesis in red blood cell formation. Heme is the deep-red, iron-containing component of hemoglobin, the protein in red blood cells responsible for transporting oxygen in the blood.

14 Nov 2012

Atpif1 gene regulates hemoglobin synthesis during red blood cell formation

Researchers at Brigham and Women's Hospital (BWH) have discovered a new gene that regulates hemoglobin synthesis during red blood cell formation. The findings advance the biomedical community's understanding and treatment of human anemias and mitochondrial disorders.

7 Nov 2012

New gene transfer technique can treat beta-thalassemia, sickle cell anemia

A team of researchers led by scientists at Weill Cornell Medical College has designed what appears to be a powerful gene therapy strategy that can treat both beta-thalassemia disease and sickle cell anemia. They have also developed a test to predict patient response before treatment.

28 Mar 2012

Increasing TR2/TR4 expression can lead to higher fetal hemoglobin levels in sickle cell patients

A University of Michigan Health System laboratory study reveals a key trigger for producing normal red blood cells that could lead to a new treatment for those with sickle cell disease.

3 Nov 2011

EGT transfers clinical grade lentiviral vector to MSKCC

Errant Gene Therapeutics, LLC, a pioneering boutique drug development firm specializing in Rare Diseases, announced the transfer of its clinical grade lentiviral vector, TNS 9.55.3, to Memorial Sloan Kettering Cancer Center.

29 Jul 2011

Hepcidin hormone may be potential therapeutic tool for anemic blood disorders

A hormone made by the body may be a potential therapeutic tool for the treatment of two anemic blood disorders beta-thalassemia and hemochromatosis. The new research was led by scientists at Weill Cornell Medical College and published in the Journal of Clinical Investigation and the journal Blood.

24 Nov 2010

bluebird bio publishes LentiGlobin gene therapy clinical trial data results

bluebird bio an emerging leader in the development of innovative gene therapies for severe genetic disorders, today announced publication in the journal Nature of its promising Phase 1/2 data highlighting positive results of LentiGlobin gene therapy treatment in a young adult with severe beta-thalassemia, a blood disorder that is one of the most frequent inherited diseases.

16 Sep 2010

Researchers reveal findings on restricted lineage of iPS cells

Adult cells that have been reprogrammed into induced pluripotent stem cells (iPS cells) do not completely let go of their past, perhaps limiting their ability to function as a less controversial alternative to embryonic stem cells for basic research and cell replacement therapies, according to researchers at Children's Hospital Boston, John Hopkins University and their colleagues

20 Jul 2010

Gene therapy represents safe alternative to current cures for blood disorder β-thalassemia

Italian scientists pioneering a new gene transfer treatment for the blood disorder β-thalassemia have successfully completed preclinical trials, claiming they can correct the lack of beta-globin (β-globin) in patients' blood cells which causes the disease. The research, published in EMBO Molecular Medicine, reveals how gene therapy may represent a safe alternative to current cures that are limited to a minority of patients.

14 Jul 2010

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

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