Abstract: Methods and compositions disclosed herein generally relates to methods of determining minimum hematopoietic stem cell (HSC) chimerism and gene dosage for correction of a hematopoietic disease; in particular, in in vivo models. The invention also relates to modified lentiviral expression vectors for increase a viral titer and various methods for increasing such titers as well as expression vectors capable of enhancing such titers. The invention also relates to CHS4 chromatin insulator-derived functional insulator sequences. The invention further relates to methods for genetic correction of diseases or reducing symptoms thereof, such as sickle cell anemia, a lysosomal storage disease. The invention further relates to a method of improving and/or correcting one or more central nervous system (CNS) abnormalities caused by one or more lysosomal storage disease.

Abstract: Compositions and methods for delivering a protein of interest such as a cell homing molecule (e.g., CXCR4) into host cells (e.g., to hematopoietic cells). The compositions and methods provided herein may be used to enhance homing and long-term engraftment of hematopoietic cells post transplantation.

Abstract: A strong insulator fragment from foamy virus, which can be used to insulate expression of a transgene and reduce genotoxicity of integrating vectors comprising such. The insulator fragment can also be used in gene targeting constructs in gene editing.

Abstract: Provided herein are methods for preparing hematopoietic stem cells (HSCs) having enhanced engraftment activity, for example, by contacting HSCs in the presence of a p38 MAPK inhibitor and a HIF-1a stabilizer.

Abstract: Methods of producing and manufacturing retroviral particles. Such methods may involve the use of an ion-exchange column with an elution buffer comprising one or more salts, wherein the elution buffer has a low total salt concentration (e.g., 400 mM to 800 mM) relative to conventional practice. In some embodiments, the retroviral particles can be generated by host cells transfected with retroviral vectors using polyethylenimine (PEI).

Abstract: Methods and compositions disclosed herein generally relates to methods of determining minimum hematopoietic stem cell (HSC) chimerism and gene dosage for correction of a hematopoietic disease; in particular, in in vivo models. The invention also relates to modified lentiviral expression vectors for increasing a viral titer and various methods for increasing such titers as well as expression vectors capable of enhancing such titers. The invention also relates to CHS4 chromatin insulator-derived functional insulator sequences. The invention also relates to methods for genetic correction of diseases or reducing symptoms thereof, such as sickle cell anemia or ?-thalassemia.

Abstract: Disclosed are methods of preparing FV vector particles. In some aspects, the disclosed methods may include the steps of transfecting a population of eukaryotic cells by contacting said population of eukaryotic cells with one or more transfection reagents to form a transfection mixture, and incubating the transfection mixture to form a transfected cell population; harvesting the FV vector particles from said transfected cell population; purifying the FV vector particles; and concentrating the FV vector particles.

Abstract: A method of conditioning a subject for hematopoietic cell transplantation, wherein the method involves the use of a nitrogen mustard alkylating agent such as melphalan in an amount to achieve reduced-intensity conditioning.

Abstract: The invention relates to methods of improving titer in transfection-based bioreactor culture production or transfection-based production systems using eukaryotic cells.

Abstract: Provided herein are compositions and methods related to conditioning a subject for a hematopoietic stem cell (HSC) transplantation using an inhibitor of a stem cell growth factor receptor (KIT). The compositions and methods described herein are particularly useful for a subject who is in need of a HSC transplantation and who is hypersensitive to at least one DNA damaging agent, e.g., a subject with fanconi anemia. Compositions and methods related to conditioning a fanconi anemia subject for HSC transplantation using an inhibitor of KIT are also provided herein.

Abstract: A strong insulator fragment from foamy virus, which can be used to insulate expression of a transgene and reduce genotoxicity of integrating vectors comprising such. The insulator fragment can also be used in gene targeting constructs in gene editing.

Abstract: Disclosed are methods of preparing FV vector particles. In some aspects, the disclosed methods may include the steps of transfecting a population of eukaryotic cells by contacting said population of eukaryotic cells with one or more transfection reagents to form a transfection mixture, and incubating the transfection mixture to form a transfected cell population; harvesting the FV vector particles from said transfected cell population; purifying the FV vector particles; and concentrating the FV vector particles.

Abstract: Provided herein are methods for preparing hematopoietic stem cells (HSCs) having enhanced engraftment activity, for example, by contacting HSCs in the presence of a p38 MAPK inhibitor and a HIF-1a stabilizer.

Abstract: Methods and compositions disclosed herein generally relates to methods of determining minimum hematopoietic stem cell (HSC) chimerism and gene dosage for correction of a hematopoietic disease; in particular, in in vivo models. The invention also relates to modified lentiviral expression vectors for increase a viral titer and various methods for increasing such titers as well as expression vectors capable of enhancing such titers. The invention also relates to CHS4 chromatin insulator-derived functional insulator sequences. The invention further relates to methods for genetic correction of diseases or reducing symptoms thereof, such as sickle cell anemia, a lysosomal storage disease. The invention futher relates to a method of improving and/or correcting one or more central nervous system (CNS) abnormalities caused by one or more lysosomal storage disease.

Abstract: Methods and compositions disclosed herein generally relates to methods of determining minimum hematopoietic stem cell (HSC) chimerism and gene dosage for correction of a hematopoietic disease; in particular, in in vivo models. The invention also relates to modified lentiviral expression vectors for increasing a viral titer and various methods for increasing such titers as well as expression vectors capable of enhancing such titers. The invention also relates to CHS4 chromatin insulator-derived functional insulator sequences. The invention also relates to methods for genetic correction of diseases or reducing symptoms thereof, such as sickle cell anemia or ?-thalassemia.

Abstract: Provided herein are methods for preparing hematopoietic stem cells (HSCs), for example, human HSCs suitable for engraftment in the presence of a p38 MAPK inhibitor. Methods for assessing engraftment of HSCs (e.g., human HSCs) are also provided.

Abstract: Methods and compositions disclosed herein generally relates to methods of determining minimum hematopoietic stem cell (HSC) chimerism and gene dosage for correction of a hematopoietic disease; in particular, in in vivo models. The invention also relates to modified lentiviral expression vectors for increasing a viral titer and various methods for increasing such titers as well as expression vectors capable of enhancing such titers. The invention also relates to CHS4 chromatin insulator-derived functional insulator sequences. The invention also relates to methods for genetic correction of diseases or reducing symptoms thereof, such as sickle cell anemia or ?-thalassemia.

Abstract: Provided herein are compositions and methods related to conditioning a subject for a hematopoietic stem cell (HSC) transplantation using an inhibitor of a stem cell growth factor receptor (KIT). The compositions and methods described herein are particularly useful for a subject who is in need of a HSC transplantation and who is hypersensitive to at least one DNA damaging agent, e.g., a subject with fanconi anemia. Compositions and methods related to conditioning a fanconi anemia subject for HSC transplantation using an inhibitor of KIT are also provided herein.

Abstract: This invention relates to a new use of compounds that are angiotensin II receptor agonists, specifically agonists of the angiotensin II type 2 receptor (the AT2 receptor), and especially agonists that bind selectively to the AT2 receptor, in the treatment of sickle cell disease.

Abstract: Methods and compositions disclosed herein generally relates to methods of determining minimum hematopoietic stem cell (HSC) chimerism and gene dosage for correction of a hematopoietic disease; in particular, in in vivo models. The invention also relates to modified lentiviral expression vectors for increasing a viral titer and various methods for increasing such titers as well as expression vectors capable of enhancing such titers. The invention also relates to CHS4 chromatin insulator-derived functional insulator sequences. The invention also relates to methods for genetic correction of diseases or reducing symptoms thereof, such as sickle cell anemia or ?-thalassemia.