Cooley's Anemia News and Research RSS Feed - Cooley's Anemia News and Research

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

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. [More]
Atpif1 gene regulates heme synthesis in red blood cell formation

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. [More]
Atpif1 gene regulates hemoglobin synthesis during red blood cell formation

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. [More]
New gene transfer technique can treat beta-thalassemia, sickle cell anemia

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

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. [More]
EGT transfers clinical grade lentiviral vector to MSKCC

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. [More]
Hepcidin hormone may be potential therapeutic tool for anemic blood disorders

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. [More]
bluebird bio publishes LentiGlobin gene therapy clinical trial data results

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. [More]
Researchers reveal findings on restricted lineage of iPS cells

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

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. [More]
Avoiding spleen removal for Cooley's anemia sufferers

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. [More]
Mechanism driving iron overload in thalassemia identified

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. [More]