Vertex Pharmaceuticals Incorporated
CRISPR-CAS9 MODIFIED CD34+ HUMAN HEMATOPOIETIC STEM AND PROGENITOR CELLS AND USES THEREOF

Abstract:

Provided herein, in some embodiments, are methods and compositions for treatment of subjects with .beta.-thalassemia and subjects with severe sickle cell disease using autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells.