Beta Thalassemia News and Research

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Beta-thalassemia (ß-thalassemia) is a form of thalassemia due to mutations in the HBB gene on chromosome 11 , inherited in an autosomal recessive fashion.

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

Base editing shows promise for treating sickle cell disease and beta thalassemia

Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease (SCD) and beta thalassemia.

3 Jul 2023

Many genetic disorders respond well to specialized gene therapy

When the normal DNA sequence in an individual changes or mutates it can cause disease. The mutation may involve multiple genes and may be caused by environmental factors, or there may be chromosomal defects that are harmful at birth, in infancy or environment at a later stage.

12 Dec 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

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 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

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

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

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

Researchers used single-molecule imaging to compare the genome-editing tools CRISPR-Cas9 and TALEN.

27 Jan 2021

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

Thanks to major progress in the understanding and management of rare congenital diseases and syndromes, many patients with these rare disorders are now living longer lives.

13 Jan 2021

Rice University wins prestigious grant to develop new therapies for sickle cell disease

A Rice University lab has won a prestigious National Institutes of Health grant to pursue gene-editing research it hopes will lead to a cure for sickle cell disease (SCD).

25 Apr 2020

Ancient DNA analysis provides insight into genetic history of Sardinia

A new study of the genetic history of Sardinia, a Mediterranean island off the western coast of Italy, tells how genetic ancestry on the island was relatively stable through the end of the Bronze Age, even as mainland Europe saw new ancestries arrive.

24 Feb 2020

Horizon Discovery to offer novel base editing technology for clinical research

Horizon Discovery today announced that it will provide access to a novel base editing technology licensed from Rutgers for exclusive use in therapeutic, diagnostic and services applications.

From Horizon Discovery 14 Jan 2020

Mystery surrounds gene edited babies one year on

A year back Chinese scientist He Jiankui was in the news for all the wrong reasons. He had helped create the world’s first genetically edited human babies. After a year the world is still in dark about the whereabouts and health of the babies he had created and he himself has been not seen since January this year.

27 Nov 2019