Abstract: Provided are compositions and methods for the treatment of hemoglobinopathies such as thalassemias and sickle cell disease. Compositions and methods include one or more endonuclease(s) or endonuclease fusion protein(s), including one or more homing endonuclease(s) and/or homing endonuclease fusion protein(s) and/or CRISPR endonuclease(s) and/or CRISPR endonuclease fusion protein(s): (a) to disrupt a Bcl11a coding region; (b) to disrupt a Bcl11a gene regulatory region; (c) to modify an adult human ?-globin locus; (d) to disrupt a HbF silencing DNA regulatory element or pathway, such as a Bcl11a-regulated HbF silencing region; (e) to mutate one or more ?-globin gene promoter(s) to achieve increased expression of a ?-globin gene; (f) to mutate one or more ?-globin gene promoter(s) to achieve increased expression of a ?-globin gene; and/or (g) to correct one or more ?-globin gene mutation(s).

Abstract: Provided are compositions and methods for the treatment of hemoglobinopathies such as thalassemias and sickle cell disease. Compositions and methods include one or more endonuclease(s) or endonuclease fusion protein(s), including one or more homing endonuclease(s) and/or homing endonuclease fusion protein(s) and/or CRISPR endonuclease(s) and/or CRISPR endonuclease fusion protein(s): (a) to disrupt a Bcl11a coding region; (b) to disrupt a Bcl11a gene regulatory region; (c) to modify an adult human ?-globin locus; (d) to disrupt a HbF silencing DNA regulatory element or pathway, such as a Bcl11a-regulated HbF silencing region; (e) to mutate one or more ?-globin gene promoter(s) to achieve increased expression of a ?-globin gene; (f) to mutate one or more ?-globin gene promoter(s) to achieve increased expression of a ?-globin gene; and/or (g) to correct one or more ?-globin gene mutation(s).

Abstract: Provided are compositions and methods for the treatment of hemoglobinopathies such as thalassemias and sickle cell disease. Compositions and methods include one or more endonuclease(s) or endonuclease fusion protein(s), including one or more homing endonuclease(s) and/or homing endonuclease fusion protein(s) and/or CRISPR endonuclease(s) ad/or CRISPR endonuclease fusion protein(s): (a) to disrupt a Bcl11a coding region; (b) to disrupt a Bcl11a gene regulatory region; (c) to modify an adult human ?-globin locus; (d) to disrupt a HbP silencing DNA regulatory element or pathway, such as a Bcl11a-regulated HbP silencing region; (e) to mutate one or more ?-globin gene promoter(s) to achieve increased expression of a ?-globin gene; (f) to mutate one or more ?-globin gene promoter(s) to achieve increased expression of a ?-globin gene; and/or (g) to correct one or more ?-globin gene mutation(s).

Abstract: Boron containing compounds are targeted to hormonally responsive cells by contacting the cells with a conjugate of a boron containing moiety conjugated to a ligand having binding specificity for an intracellular receptor of the cell. The conjugates are useful for imaging or killing hormonally responsive cells, such as hormonally responsive tumor cells. For target cell killing, the cells are contacted with a .sup.10 B containing moiety conjugated to a ligand having binding specificity for an intracellular hormone receptor of the cells. The .sup.10 B containing moiety becomes associated with the hormone receptor of the cells, which may then be irradiated with neutrons to kill the cells by boron neutron capture. For target cell imaging, the cells are contacted with a .sup.

Abstract: Human checkpoint huCDC34, huRAD9.sub.compA, and huRAD9.sub.compB cDNAs shown in FIGS. 1, 2, and 3.

Abstract: Human checkpoint huCDC34, huRAD9.sub.compA, and huRAD9.sub.compB cDNAs shown in SEQ ID Nos:7-9.

Abstract: An avian/mammalian microcell hybrid immortalized pre B cell line containing a mammalian chromosome that carries a selectable marker has been produced. The avian/mammalian microcell hybrid immortalized pre B cell line can be used for introducing predetermined point mutations into specific mammalian chromosomal loci via high frequency homologous recombination.