Thalassemia News and Research

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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.

Telomerase's Double-Edged Sword: Preventing DNA Repair Mishaps

The natural ends of chromosomes appear alarmingly like broken DNA, much as a snapped spaghetti strand is difficult to distinguish from its intact counterparts.

16 Feb 2024

New Proof-of-Concept Model for Delivering Gene Editing Tools to Treat 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.

28 Jul 2023

Researchers use CRISPR gene editing tool to analyze globin gene switching

CRISPR gene editing—a form of “molecular scissors”—was used by UNSW researchers to investigate how deletions in one section of the genome might impact the expression of surrounding genes.

From The University of New South Wales (UNSW) 19 Apr 2022

Highly efficient method to identify the genetic “switches” that control gene expression

Scientists have created a high-throughput method to help explain and regulate gene expression for the treatment of diseases like beta-thalassemia and sickle cell disease.

7 May 2021

PNA-based method is a key component of the gene editing toolkit

The Somatic Cell Gene Editing Consortium has given an update on the status of their initiative to design safer techniques to edit the genes of disease-relevant somatic cells.

8 Apr 2021

Study shows TALEN is five times more efficient than CRISPR-Cas9 tool

Using single-molecule imaging, scientists have compared the CRISPR-Cas9 and TALEN genome editing tools.

28 Jan 2021

Umbilical cord blood treats children born with non-cancerous genetic disorders

Researchers at UPMC Children's Hospital of Pittsburgh found that infusing umbilical cord blood -- a readily available source of stem cells -- safely and effectively treated 44 children born with various non-cancerous genetic disorders, including sickle cell, thalassemia, Hunter syndrome, Krabbe disease, metachromatic leukodystrophy (MLD) and an array of immune deficiencies.

15 Jul 2020