bluebird bio, Inc.
BRUTON'S TYROSINE KINASE HOMING ENDONUCLEASE VARIANTS, COMPOSITIONS, AND METHODS OF USE

Abstract:

The present disclosure provides improved genome editing compositions and methods for editing a human BTK gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of X-linked agammaglobulinemia (XLA).