Thalassemia

Thalassemia is a group of genetic blood disorders that affect approximately 1,000 individuals in the United States. The most severe of these disorders is Cooley's Anemia.

People with thalassemia have a genetic defect of their red blood cells that affects the cells' ability to produce normal hemoglobin. Red blood cells use hemoglobin to carry oxygen to tissues. As a result of the defect, most forms of thalassemia produce a chronic, lifelong anemia that begins in early childhood and often must be treated with frequent transfusions.
What is Thalassemia?

Beta thalassemia describes a condition of the blood characterized by abnormal hemoglobin in the red blood cells. This abnormal hemoglobin is reduced in its capacity to transport oxygen around the body, which leads to anemia, fatigue, weakness and shortness of breath.

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Latest Thalassemia News and Research

Efficacy of vaccination against SARS-CoV-2 Omicron BA.1 and BA.2 sublineages in Sweden

Efficacy of vaccination against SARS-CoV-2 Omicron BA.1 and BA.2 sublineages in Sweden

UNSW research improves fundamental understanding of the mechanism behind sickle cell disease

UNSW research improves fundamental understanding of the mechanism behind sickle cell disease

Gene therapy leads to durable transfusion independence in youngest patients with thalassemia

Gene therapy leads to durable transfusion independence in youngest patients with thalassemia

Gene therapy may be a curative treatment option for patients with transfusion-dependent beta-thalassemia

Gene therapy may be a curative treatment option for patients with transfusion-dependent beta-thalassemia

Hydroxyurea therapy can improve or reverse cardiac complications in sickle cell anemia patients

Hydroxyurea therapy can improve or reverse cardiac complications in sickle cell anemia patients

Iron chelating properties of eltrombopag in pediatric acute myeloid leukemia

Iron chelating properties of eltrombopag in pediatric acute myeloid leukemia

A path to treat currently untreatable cases of cystic fibrosis is clearly achievable, study suggests

A path to treat currently untreatable cases of cystic fibrosis is clearly achievable, study suggests

Study examines safety of COVID-19 vaccination during lactation

Study examines safety of COVID-19 vaccination during lactation

UCSF launches new initiative to propel the development of living therapeutics

UCSF launches new initiative to propel the development of living therapeutics

Researchers develop proof-of-concept treatment for blood disorders

Researchers develop proof-of-concept treatment for blood disorders

High-throughput system can regulate gene expression for treating sickle cell disease, beta thalassemia

High-throughput system can regulate gene expression for treating sickle cell disease, beta thalassemia

Toward a transformative therapy for sickle cell patients

Toward a transformative therapy for sickle cell patients

PNA-based gene editing technique can advance gene discovery

PNA-based gene editing technique can advance gene discovery

Research provides a new cornerstone of malaria's evolution with humans

Research provides a new cornerstone of malaria's evolution with humans

Combined liver-cytokine humanization enhances human red blood cell production and survival

Combined liver-cytokine humanization enhances human red blood cell production and survival

Study: TALEN is five times more efficient than CRISPR-Cas9 in tightly packed DNA

Study: TALEN is five times more efficient than CRISPR-Cas9 in tightly packed DNA

New taxonomic classification of non-skeletal rare congenital disorders with impact on bone physiology

New taxonomic classification of non-skeletal rare congenital disorders with impact on bone physiology

Researchers develop telomerase gene therapy for treating different pathologies

Researchers develop telomerase gene therapy for treating different pathologies

Majority of U.S. COVID-19 fatalities had underlying medical conditions

Majority of U.S. COVID-19 fatalities had underlying medical conditions

Umbilical cord blood safely and effectively treats children with rare genetic disorders

Umbilical cord blood safely and effectively treats children with rare genetic disorders