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.

Thalassemia Treatment

Thalassemia Prevalence

Thalassemia Benefits

Thalassemia Pathophysiology

Thalassemia Genetic Prevalence

Latest Thalassemia News and Research

New gene therapy strategy reactivates dormant genes to treat blood disorders

Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers.

19 Jun 2025

Customized CRISPR gene editing therapy successfully treats infant with rare disease

In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of Philadelphia (CHOP) and Penn Medicine.

15 May 2025

CRISPR tool unveils genetic drivers of blood cell maturation

Collaborative research led by investigators at Dana-Farber/Boston Children's Cancer and Blood Disorders Center defines a novel approach to understanding how certain proteins called transcription factors determine which genetic programs will drive cell growth and maturation.

4 Apr 2025

In the Thymus We Trust: ProTcell Therapy & the Future of Allogeneic Immunotherapy

In this interview, News Medical speaks with Olivier Negre, Chief Scientific Officer at Smart Immune, about how immunotherapy is being revolutionized.

From Hanson Wade Group - 7th Allogeneic Cell Therapies Summit 18 Mar 2025

Scientists discover key molecular switch for blood stem cell regeneration

A single molecular switch is essential for blood stem cells to enter an activated, regenerative state in which they produce new blood cells, according to a preclinical study led by Weill Cornell Medicine investigators.

10 Mar 2025

Iron dysregulation linked to long COVID development

Problems with iron levels in the blood and the body's ability to regulate this important nutrient as a result of SARS-CoV-2 infection could be a key trigger for long COVID, new research has discovered.

5 Mar 2025

JRC releases a new CRM to aid the fight against beta-thalassemia

Our blood is a window to our health. Doctors examine biomarkers in our blood to determine our health status.

12 Oct 2024

SCD health disparities tied to community factors

A new study finds that preschool-aged children with sickle cell disease (SCD) who live in food deserts and have limited access to transportation are at greater risk for acute complications and hospitalizations, despite receiving free evidence-based therapy and social support, according to results published today in Blood Advances.

8 Oct 2024

WHO coordinates large-scale medical evacuation from Gaza to UAE

Today, 85 sick and severely injured patients from Gaza were evacuated to Abu Dhabi, United Arab Emirates (UAE), for specialized care.

31 Jul 2024

Understanding iron overload in β-thalassemia: Mechanisms, diagnosis, and treatments

β-Thalassemia is a genetic disorder characterized by reduced or absent synthesis of the beta chains of hemoglobin, leading to ineffective erythropoiesis and severe anemia.

29 Jul 2024

Comprehensive cost and utilization of commercial CAR-T therapy in pediatric B-ALL patients

A new editorial paper was published in Oncoscience (Volume 11) on April 25, 2024, entitled, "The price of hope: CAR-T therapy in pediatric leukemia."

17 May 2024

CASGEVY gene therapy eliminates vaso-occlusive crises in sickle cell patients

In a landmark study, an international consortium led by researchers at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).

24 Apr 2024

Breakthrough in CRISPR delivery promises safer gene editing

Recent research in PNAS highlights advancements in non-viral, cell-specific delivery methods for CRISPR-Cas systems, emphasizing their potential to enhance research and gene therapy applications by minimizing risks associated with off-target effects and improving targeted delivery.

7 Mar 2024

Iron dysregulation identified as potential trigger for long COVID

4 Mar 2024

Breakthrough gene-editing therapy transforms lives of patients with a hereditary disorder

A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher.

2 Feb 2024

Study: Two-thirds of babies with sickle cell disease are born to mothers living in disadvantaged areas

As if starting life with a potentially disabling genetic blood disease wasn't enough, a study shows that almost two-thirds of babies born with sickle cell disease are born to mothers who live in disadvantaged areas.

4 Jan 2024

Fetal hormone GDF15 linked to nausea and vomiting in pregnancy

Study finds that high levels of the fetal hormone GDF15 in mothers are linked to an increased risk of nausea, vomiting, and hyperemesis gravidarum in pregnancy, offering new insights into the condition's etiology.

14 Dec 2023

Shining a light on thalassemia: Global report unveils knowledge gaps and opportunities

Thalassemia, a hereditary hemoglobinopathy, occurs in 4.4 out of every 10,000 live births and is prevalent in Mediterranean coastal areas, Africa, the Middle East, Southeast Asia, and southern China.

4 Dec 2023

New CRISPR approach shows promise for treating glioblastoma

The gene-editing technology CRISPR shows early promise as a therapeutic strategy for the aggressive and difficult-to-treat brain cancer known as primary glioblastoma, according to findings of a new study from Gladstone Institutes.

27 Nov 2023

BGI Genomics report uncovers knowledge gaps and barriers to thalassemia screening

According to Thailand's Ministry of Public Health, approximately 18-24 million or 30-40 percent of the Thai population carries the thalassemia gene, with moderately severe thalassemia patients requiring regular treatment, including blood transfusion and chelation therapy to remove excess iron from the blood.

20 Nov 2023