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

Protection afforded by SARS-CoV-2 infection against reinfection when Omicron BA.5 was the dominating subvariant in Scania county, Sweden

Researchers from Sweden conducted a cohort study to evaluate the protection granted by previous SARS-CoV-2 Omicron subvariant infections against reinfections among individuals vaccinated between June and August 2022.

17 Nov 2022

First transfusions of lab-grown blood

Two patients in the United Kingdom received two small doses of lab-grown blood samples as part of the RESTORE trial.

9 Nov 2022

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

Azerbaijan Thalassemia Center and BGI partner to improve thalassemia screening in Azerbaijan

On 6th September 2022, the Azerbaijan Thalassemia Center and BGI held a virtual signing ceremony to seal a Collaboration Agreement. The partnership aims to improve thalassemia screening in Azerbaijan through genetic technology.

16 Sep 2022

Researchers explore disease severity post-infection with SARS-CoV-2 Omicron in hematologic patients

A new study has studied the clinical outcome and spectrum in 61 patients infected with the SARS-CoV-2 Omicron variant.

29 Jun 2022

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

Researchers compared the vaccine effectiveness against infections by SARS-CoV-2 variant of concern Omicron BA.1 and BA.2 sublineages in Sweden.

22 Apr 2022

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

UNSW researchers have used CRISPR gene editing – a type of 'molecular scissors' – to understand how deletions in one area of the genome can affect the expression of nearby genes.

8 Apr 2022

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

Over 90 percent of patients with transfusion-dependent thalassemia, an inherited blood disorder, no longer needed monthly blood transfusions years after receiving gene therapy, according to an international Phase 3 clinical trial that for the first time included children younger than 12 years of age.

22 Feb 2022

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

A novel gene therapy promoted transfusion independence in more than 90 percent of adult and pediatric patients with transfusion-dependent beta-thalassemia, according to a recent clinical trial published in The New England Journal of Medicine.

3 Feb 2022

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

Heart abnormalities are common complications of sickle cell anemia (SCA) that can contribute to severe disease symptoms and even death.

16 Sep 2021

Iron chelating properties of eltrombopag in pediatric acute myeloid leukemia

Oncotarget published "Eltrombopag and its iron chelating properties in pediatric acute myeloid leukemia" which reported that the standard treatment for pediatric AML currently consists in a combination of cytarabine and antracycline.

9 Aug 2021

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

An experimental drug reported in Nature Communications suggests that a "path is clearly achievable" to treat currently untreatable cases of cystic fibrosis disease caused by nonsense mutations.

19 Jul 2021

Study examines safety of COVID-19 vaccination during lactation

A new preprint, recently released on the medRxiv* server, reports from real life in a cohort of mother-child dyads in Singapore. The mothers were lactating after having received COVID-19 vaccination.

25 Jun 2021

UCSF launches new initiative to propel the development of living therapeutics

UC San Francisco is launching a new initiative to propel the development of living therapeutics - a category of treatments broadly defined as human and microbial living cells that are selected, modified, or engineered to treat or cure disease - and bring them quickly to patients.

14 Jun 2021

Researchers develop proof-of-concept treatment for blood disorders

Researchers at Children's Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders like sickle cell disease and beta-thalassemia that could raise hemoglobin levels by activating production of both fetal and adult hemoglobin.

1 Jun 2021

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

St. Jude Children's Research Hospital scientists have developed an integrated, high-throughput system to better understand and possibly manipulate gene expression for treatment of disorders such as sickle cell disease and beta thalassemia. The research appears today in the journal Nature Genetics.

7 May 2021

Toward a transformative therapy for sickle cell patients

For the millions of people worldwide who have sickle cell disease, there are only a few treatment options, which include risky bone marrow transplants, gene therapy or other treatments that address a subset of symptoms.

9 Apr 2021

PNA-based gene editing technique can advance gene discovery

In an article published in the April 8 issue of Nature, the National Institutes of Health's Somatic Cell Gene Editing Consortium provided a detailed update on the progress of their nationwide effort to develop safer and more effective methods to edit the genomes of disease-relevant somatic cells and reduce the burden of disease caused by genetic changes.

8 Apr 2021

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

New bioarchaeological research shows malaria has threatened human communities for more than 7000 years, earlier than when the onset of farming was thought to have sparked its devastating arrival.

13 Mar 2021

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

In a new study by the Yale Department of Immunobiology and Yale Cancer Center, researchers report combined liver and growth factor humanization enhances human red blood cell production and survival in circulation the immunodeficient murine host.

5 Mar 2021

Thalassemia

Overview

Thalassemia Genetic Prevalence

Thalassemia Prevalence

Thalassemia Pathophysiology

Feature Articles

Thalassemia Treatment

Thalassemia Benefits

Latest Thalassemia News and Research

Protection afforded by SARS-CoV-2 infection against reinfection when Omicron BA.5 was the dominating subvariant in Scania county, Sweden

First transfusions of lab-grown blood

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

Azerbaijan Thalassemia Center and BGI partner to improve thalassemia screening in Azerbaijan

Researchers explore disease severity post-infection with SARS-CoV-2 Omicron in hematologic patients

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

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

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

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

Study examines safety of COVID-19 vaccination during lactation

UCSF launches new initiative to propel the development of living therapeutics

Researchers develop proof-of-concept treatment for blood disorders

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

Toward a transformative therapy for sickle cell patients

PNA-based gene editing technique can advance gene discovery

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

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

Miracles Start in the Lab: the quest to find a vaccine to cure AIDS

Pioneering new medical imaging solutions

Pioneering new medical imaging solutions

Lab Diagnostics & Automation eBook

Thalassemia

Overview

Feature Articles

Thalassemia Treatment

Thalassemia Benefits

Latest Thalassemia News and Research

Miracles Start in the Lab: the quest to find a vaccine to cure AIDS

Pioneering new medical imaging solutions

Pioneering new medical imaging solutions

Lab Diagnostics & Automation eBook

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