Harvard College

Children's Medical Center Corporation

Chad A Cowan

Jack L Strominger

Torsten B Meissner

Leonardo M R Ferreira

Derrick J Rossi

Kiran Musunuru

Leonardo Mr Ferreira

Kabungo Y Mulumba

Disclosed herein are universal donor stem cells and related methods of their use and production. The universal donor stem cells disclosed herein are useful for overcoming the immune rejection in cell-based transplantation therapies. In certain embodiments, the universal donor stem cells disclosed herein do not express one or more MHC-I and MHC-II human leukocyte antigens. Similarly, in certain embodiments, the universal donor stem cells disclosed herein do not express one or more human leukocyte antigens (e.g., HLA-A, HLA-B and/or HLA-C) corresponding to MHC-I and MHC-II human leukocyte antigens, thereby rendering such cells hypoimmunogenic.

Universal donor stem cells and related methods

Disclosed herein are cells and populations of cells comprising a genome in which the B2M gene has been edited to eliminate surface expression of MHC Class I protein in the cells or population of cells, and methods for allogeneic administration of such cells to reduce the likelihood that the cells will trigger a host immune response when the cells are administered to a subject in need of such cells.

Cells lacking B2M surface expression and methods for allogeneic administration of such cells

Disclosed herein are methods, compositions, and kits for high efficiency, site-specific genomic editing of cells for treating or preventing genetic blood disorders.

Therapeutic uses of genome editing with CRISPR/Cas systems

Disclosed herein are methods, compositions, and kits for high efficiency, site-specific genomic editing of cells.

Growing community of inventors

Boston, MA, United States of America

Chad A Cowan