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