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

Abstract: The present invention relates to the identification, the isolation and the use of molecules interfering with the function(s) mediated by the poliovirus receptor (PVR) on cells. The molecules can be used for the treatment of cells having a metastatic potential, metastasis and cancer. Further methods are provided that are useful for identifying and isolating molecules which have the capacity to modulate PVR mediated adhesion or invasion potential of cells.

Abstract: The present invention relates to molecules interfering with the function of neuropilin-1 in the context of angiogenesis and the treatment of cancer. Molecules, polypeptides, antibodies, compositions and methods are provided that are useful for reducing, inhibiting or treating angiogenesis, the invasion of blood vessels into tumors, and/or the invasion or the metastatic potential of specific tumor cells. Additionally, a method is provided that allows identifying molecules, which interfere with the functionality of neuropilin-1. Furthermore, a method is provided that allows determining whether a naturally occuring tumor cell depends on functional neuropilin-1 for its invasiveness and/or metastatic potential.

Abstract: The present invention describes inhibitors of extracellular Hsp90. The inhibition of extracellular Hsp90 leads to a reduction of the invasiveness of the tumor cells. Furthermore, the invention relates to the use of molecules inhibiting extracellular Hsp90 function for the manufacture of a medicament for the treatment or prevention of invasion and/or metastatic potential of cancer cells.