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 Genetic Prevalence

Thalassemia Prevalence

Thalassemia Pathophysiology

Thalassemia Treatment

Thalassemia Benefits

Latest Thalassemia News and Research

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

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.

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

New report highlights urgent need to improve thalassemia awareness among Azerbaijan women

5.2% of the global population carry hemoglobin abnormalities, resulting in 300,000 to 400,000 children born with severe hemoglobinopathies annually.

17 Nov 2023

Gene therapy's double-edged sword: Breakthrough treatments face manufacturing and efficacy

Research explores the advancements, challenges, and economic implications of gene therapy for various diseases, including inherited blood disorders, malignancies treated with CAR-T cells, and diseases targeted by in vivo AAV vectors. The review highlights the transformative potential of gene therapies while acknowledging the complexities in manufacturing, safety, efficacy, and long-term durability.

12 Nov 2023

NGS-based screening identifies rare and novel thalassemia mutations in Ganzhou, China

Around 5.2% of the global population carries abnormal hemoglobin genes. Each year, 300,000 to 500,000 children are born with severe hemoglobinopathies worldwide, with approximately 80% of these cases occurring in developing countries.

5 Sep 2023

New gene therapy offers hope for beta thalassemia patients

UCSF Benioff Children's Hospital Oakland provides new therapy to first patient since FDA approval.

31 Aug 2023

Research breaks ground on new ways of treating blood disorders with gene therapy

Research from experts at Michigan Medicine, the Children's Hospital of Philadelphia and Penn Medicine is breaking ground on new ways of treating blood disorders, such as sickle cell anemia, through gene therapy.

9 Aug 2023

New gene editing approach could revolutionize the treatment of blood disorders

In a step forward in the development of genetic medicines, researchers at Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.

27 Jul 2023

Thalassemia

Overview

Thalassemia Genetic Prevalence

Thalassemia Prevalence

Thalassemia Pathophysiology

Feature Articles

Thalassemia Treatment

Thalassemia Benefits

Latest Thalassemia News and Research

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

SCD health disparities tied to community factors

WHO coordinates large-scale medical evacuation from Gaza to UAE

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

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

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

Breakthrough in CRISPR delivery promises safer gene editing

Iron dysregulation identified as potential trigger for long COVID

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

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

Fetal hormone GDF15 linked to nausea and vomiting in pregnancy

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

New CRISPR approach shows promise for treating glioblastoma

BGI Genomics report uncovers knowledge gaps and barriers to thalassemia screening

New report highlights urgent need to improve thalassemia awareness among Azerbaijan women

Gene therapy's double-edged sword: Breakthrough treatments face manufacturing and efficacy

NGS-based screening identifies rare and novel thalassemia mutations in Ganzhou, China

New gene therapy offers hope for beta thalassemia patients

Research breaks ground on new ways of treating blood disorders with gene therapy

New gene editing approach could revolutionize the treatment of blood disorders

Small solutions for big problems in drug discovery and delivery

Professor Nancy Ip: Pioneering New Paths in Neurodegenerative Therapy

Utilizing process Raman in optimizing cultivated meat production

Thalassemia

Overview

Feature Articles

Thalassemia Treatment

Thalassemia Benefits

Latest Thalassemia News and Research

Small solutions for big problems in drug discovery and delivery

Professor Nancy Ip: Pioneering New Paths in Neurodegenerative Therapy

Utilizing process Raman in optimizing cultivated meat production

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