The presently disclosed subject matter provides for expression cassettes that allow for expression of a globin gene or a functional portion thereof, vectors comprising thereof, and cells transduced with such expression cassettes and vectors. The presently disclosed subject matter further provides methods for treating a hemoglobinopathy in a subject comprising administering an effective amount of such transduced cells to the subject.

Described herein are heme-binding compositions and methods relating to their use, for example methods of treatment of sepsis and rhabdomyolysis.

Described herein are heme-binding compositions and methods relating to their use, for example methods of treatment of sepsis and rhabdomyolysis.

Abstract The disclosure provides a reagent comprising a leuco dye and a compound represented by Formula (I): ##STR00001## where R represents a hydrocarbon chain having 8 to 17 carbon atoms, the reagent for measuring glycoprotein, a kit comprising the reagent and a second reagent, and methods of measuring hemoglobin A1c using the reagent.

Abstract The disclosure provides a reagent comprising a leuco dye and a compound represented by Formula (I): ##STR00001## where R represents a hydrocarbon chain having 8 to 17 carbon atoms, the reagent for measuring glycoprotein, a kit comprising the reagent and a second reagent, and methods of measuring hemoglobin A1c using the reagent.

The invention provides compositions comprising novel adenosine base editors (e.g., ABE8) that have increased efficiency and methods of using these adenosine deaminase variants for editing a target sequence and methods of using same to treat genetical disorder or conditions, e.g. sickle cell disease, with engraftment.

The invention provides compositions comprising novel adenosine base editors (e.g., ABE8) that have increased efficiency and methods of using these adenosine deaminase variants for editing a target sequence and methods of using same to treat genetical disorder or conditions, e.g. sickle cell disease, with engraftment.

The present invention relates to compositions and methods of increasing levels of fetal hemoglobin (HbF) in cells. The present invention further relates to methods for treating patients suffering from blood cell diseases, including those associated with reduced amounts of functional adult hemoglobin (HbA), such as sickle cell disease and β-thalassemias.

The present disclosure provides expression vectors comprising at least two nucleic acid sequences, namely a nucleic acid sequence encoding an anti-HPRT RNAi, and a nucleic acid sequence encoding a gamma globin gene. In some embodiments, the viral vector is a self-inactivating lentiviral vector. In some embodiments, the gamma-globin gene is used to genetically correct sickle cell disease or β-thalassemia or to reduce symptoms thereof.

The present disclosure provides expression vectors comprising at least two nucleic acid sequences, namely a nucleic acid sequence encoding an anti-HPRT RNAi, and a nucleic acid sequence encoding a gamma globin gene. In some embodiments, the viral vector is a self-inactivating lentiviral vector. In some embodiments, the gamma-globin gene is used to genetically correct sickle cell disease or β-thalassemia or to reduce symptoms thereof.