Base editing—the genome editing technique that alters base pairs using a nuclease-deficient Cas9 fused to a deaminase—was found to restart fetal hemoglobin expression in sickle cell disease (SCD) ...
Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine A new chapter is unfolding for patients with sickle cell ...
AUGUSTA, Ga. (May 11, 2023) – A class of drugs used for their ability to stop tumor cells from dividing is now under study for their potential to reduce the pain and damage caused by sickle cell ...
PHILADELPHIA, June 1, 2021 /PRNewswire/ — Researchers at Children’s Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders like sickle cell disease and ...
Sickle cell disease, while rare, is the most common inherited blood disorder and affects over 100,000 people in the United States, more than 90% of whom are Black according to the Centers for Disease ...
Though the first CRISPR drug—Casgevy, the CRISPR-Cas9 therapy formerly known as exa-cel developed by Vertex Pharmaceuticals and CRISPR Therapeutics—was approved in the U.K. for the treatment of sickle ...
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 ...
Researchers 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 ...