Broad Institute of MIT and Harvard
Sickle cell disease (SCD) is the most common deadly genetic disorder, affecting more than 300,000 newborns worldwide each year. It leads to chronic pain, organ failure, and early death in patients. A team led by researchers at the Broad Institute of MIT and Harvard and St. Jude Children’s Research Hospital has now demonstrated a base editing approach that efficiently corrects the mutation underlying SCD in patient blood stem cells and in mice. This gene editing treatment rescued the disease symptoms in animal models, enabling the long-lasting production of healthy blood cells.
The root of SCD is two mutated copies of the hemoglobin gene, HBB, which cause red blood cells to transform from a circular disc into a sickle shape setting off a chain of events leading to organ damage, recurrent pain, and early mortality. In this study, the researchers used a molecular technology called base editing to directly convert a single letter of pathogenic DNA
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.
Researchers used the system to identify dozens of DNA regulatory elements that act together to orchestrate the switch from fetal to adult hemoglobin expression. The method can also be used to study other diseases that involve gene regulation.
Regulatory elements, also called genetic switches, are scattered throughout non-coding regions of DNA. These regions do not encode genes and make up about 98% of the genome. The elements have a variety of names enhancer, repressor, insulator and more but the specific genes they regulate, how the regulatory elements act together, and answers to ot
Date Time
Researchers speed identification of DNA regions that regulate gene expression
Corresponding author Yong Cheng, Ph.D., of the St. Jude Departments of Hematology and Computational Biology, helped develop a highly efficient method in identifying the genetic switches that regulate gene expression.
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.
Researchers used the system to identify dozens of DNA regulatory elements that act together to orchestrate the switch from fetal to adult hemoglobin expression. The method can also be used to study other diseases that involve gene regulation.
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