CRISPR Therapeutics AG
MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES

Abstract:

Materials and methods for treating a patient with a hemoglobinopathy, both ex vivo and in vivo, and materials and methods for creating permanent changes to the genome that can result in at least one deletion, insertion, modulation, or inactivation of a transcriptional control sequence of a BCL11A gene in a cell by genome editing.