Editas Medicine, Inc.
TARGETED INTEGRATION SYSTEMS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES

Abstract:

Genome editing systems, guide RNAs, DNA donor templates, and CRISPR-mediated methods are provided for altering a .beta.-globin gene to alter a genotype, e.g., by correcting or partially correcting, a genotype associated with thalassemia or sickle cell disease.