Abstract: Methods and compositions for producing fetal hemoglobin and treating a hemoglobinopathy or thalassemia are disclosed. Specifically, the disclosure provides methods of increasing the level of human fetal hemoglobin and/or gamma-globin in a cell or subject, the method comprising administering at least one zinc finger protein 410 (ZNF410) inhibitor to the cell or subject, wherein the subject has a beta-chain hemoglobinopathy, thalassemia, and/or sickle cell disease.

Abstract: Methods and compositions for producing fetal hemoglobin and treating a hemoglobinopathy or thalassemia are disclosed.

Abstract: Provided are compositions and methods for inducing expression of human beta-globin in erythrocytes for use in prophylaxis and/or therapy of a hemoglobinopathy in an individual. The method generally entails introducing into CD34+ cells a polynucleotide encoding: i) a 5? long terminal repeat (LTR) and a self-inactivating 3? LTR; ii) at least one polyadenylation signal; iii) at least one promoter; iv) a globin gene locus control region (LCR); v) an ankyrin insulator element (Ank); vi) a Woodchuck Post-Regulatory Element (WPRE) configured such that the WPRE does not integrate into a target genome; and vii) a sequence that is a reverse complement of a sequence encoding human beta-globin, and can include beta-globin that has a ?T87Q mutation. Intron 2 of the beta globin gene can be a complete intron. Modified erythrocyte progenitor cells, recombinant vectors and virions comprising recombinant polynucleotides, and methods of making the vectors and virions are included.

Abstract: Provided are compositions and methods for inducing expression of human beta-globin in erythrocytes for use in prophylaxis and/or therapy of a hemoglobinopathy in an individual. The method generally entails introducing into CD34+ cells a poly-nucleotide encoding: i) a 5? long terminal repeat (LTR) and a self-inactivating 3? LTR; ii) at least one polyadenylation signal; iii) at least one promoter; iv) a globin gene locus control region (LCR); v) an ankyrin insulator element (Ank); vi) a Woodchuck Post-Regulatory Element (WPRE) configured such that the WPRE does not integrate into a target genome; and vii) a sequence that is a reverse complement of a sequence encoding human beta-globin, and can include beta-globin that has a PT87Q mutation. Intron 2 of the beta globin gene can be a complete intron. Modified erythrocyte progenitor cells, recombinant vectors and virions comprising recombinant polynucleotides, and methods of making the vectors and virions are included.

Abstract: Methods and compositions for producing fetal hemoglobin and treating a hemoglobinopathy or thalassemia are disclosed.

Abstract: Methods and compositions for producing fetal hemoglobin and treating a hemoglobinopathy or thalassemia are disclosed.

Abstract: Methods and compositions for producing fetal hemoglobin and treating a hemoglobinopathy or thalassemia are disclosed.

Abstract: Methods and compositions for controlling gene expression are disclosed.

Abstract: Methods and compositions for controlling gene expression are disclosed.

Abstract: Provided are compositions and methods for inducing expression of human beta-globin in erythrocytes for use in prophylaxis and/or therapy of a hemoglobinopathy in an individual. The method generally entails introducing into CD34+ cells a polynucleotide encoding: i) a 5? long terminal repeat (LTR) and a self-inactivating 3? LTR; ii) at least one polyadenylation signal; iii) at least one promoter; iv) a globin gene locus control region (LCR); v) an ankyrin insulator element (Ank); vi) a Woodchuck Post-Regulatory Element (WPRE) configured such that the WPRE does not integrate into a target genome; and vii) a sequence that is a reverse complement of a sequence encoding human beta-globin, and can include beta-globin that has a PT87Q mutation. Intron 2 of the beta globin gene can be a complete intron. Modified erythrocyte progenitor cells, recombinant vectors and virions comprising recombinant polynucleotides, and methods of making the vectors and virions are included.

Abstract: Methods and compositions for controlling gene expression are disclosed.

Abstract: Compositions and methods for the treatment of HIV are provided.

Abstract: Methods and compositions for controlling gene expression are disclosed.

Abstract: Compositions and methods for the treatment of HIV are provided.

Abstract: A transgenic non-human animal with alterations in the Alpha Hemoglobin Stabilizing Protein (AHSP) gene is prepared by introduction of a gene encoding an altered Alpha Hemoglobin Stabilizing Protein (AHSP) protein into a host non-human animal. Methods for using transgenic mice so generated to screen for agents that effect Alpha Hemoglobin Stabilizing Protein (AHSP)'s hemoglobin binding activity are also provided.