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.
Researchers use CRISPR gene editing tool to analyze globin gene switching

Researchers use CRISPR gene editing tool to analyze globin gene switching

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

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

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

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

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

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

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

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