cancer Cancer Discovery Cancer screening Clinical Trials

Early Clinical Trial Data Show Gene Therapy Reversing Sickle Cell Anemia

After over a decade of preclinical research and development, a new gene therapy treatment for Sickle Cell Anemia (SCA) is reversing disease symptoms in two adults and showing early potential for transportability to resource-challenged parts of the world where SCA is most common.  Preliminary data from a pilot Phase 1-2 clinical trial testing the gene-addition therapy were presented Dec. 3 at the American Society of Hematology’s (ASH) annual meeting in San
Gene Therapy Genetic Disorders Genomes

Genome Engineering Paves The Way For Sickle Cell Cure

A team of physicians and laboratory scientists has taken a key step toward a cure for sickle cell disease, using CRISPR-Cas9 gene editing to fix the mutated gene responsible for the disease in stem cells from the blood of affected patients. For the first time, they have corrected the mutation in a proportion of stem cells that is high enough to produce a substantial benefit in sickle cell patients. The