46 results about "Sickle cells" patented technology

The present invention provides a therapeutic method for treating an inflammatory response caused by a sickle cell crisis, comprising administration of an effective amount amount of an A_2A adenosine receptor agonist. Optionally, the method includes administration of a type IV PDE inhibitor (e.g., rolipram).

The present invention relates to methods of treating sickle cell disease comprising reducing, in a subject in need of such treatment, the adherence between sickle RBCs and leukocytes. It is based, at least in part, on the discovery that leukocytes play a direct role in the initiation of venular occlusion. The present invention further provides for methods for identifying agents which decrease SS-RBC/leukocyte adherence and for animal models which may be used to further elucidate the mechanism of vaso-occlusion in sickle cell crises.

Provided are methods for the detection and diagnosis of sickle cell. The methods are based on the discovery that abnormal levels of selected analytes in sample fluid, typically blood samples, of patients who are at risk are supportive of a diagnosis of sickle cell. At least two new biomarkers for sickle cell are thus disclosed, Eotaxin and Monocyte Chemotactic Protein-1. Altogether the concentrations of eleven analytes provide a sensitive and selective picture of the patient's condition, namely, whether the patient is suffering from sickle cell. Other important biomarkers for sickle cell are described, including but not limited to IL-12p40, SHBG, MMP-9, Adiponectin, Haptoglobin, FGF basic, IgM, Growth Hormone, Factor VII. Kits containing reagents to assist in the analysis of fluid samples are also described.

According to the present invention, there is provided a delivery vehicle including sickle red blood cells carrying a moiety. The moiety can be any type of diagnostic or therapeutic agent. The present invention further provides for a method of diagnosing systemic hypoxia, acidosis, or hypertonicity by administering sickle red blood cells to a patient and detecting the location of the sickle red blood cells. Additionally, the present invention provides for a method of therapeutic treatment of systemic hypoxia, acidosis, or hypertonicity by administering sickle red blood cells to a patient. Further, the present invention provides for a delivery vehicle that specifically localizes or concentrates at systemic hypoxia, acidosis, or hypertonicity areas. The delivery vehicle is used in diagnosing and therapeutically treating these areas of hypoxia, acidosis, or hypertonicity also. The present invention further provides for a method of making the delivery vehicle described herein.

A composition that includes a nanoparticle. The nanoparticle includes a shell which encapsulates sulfated non-anticoagulant heparin (SNACH). The shell includes poly L-arginine. The SNACH is ionically or covalently bonded to the poly L-arginine. A method for treating a disorder of a subject includes: administering to the subject a therapeutically effective amount of the composition for treating the disorder. The disorder is a vascular disorder, a complication of the vascular disorder, or a combination thereof.

The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic stem cell. Disclosed herein are methods and compositions for altering the expression or for correcting one or more genes encoding proteins involved in a genetic disease (e.g., producing proteins lacking, deficient or aberrant in the disease and/or proteins that regulate these proteins) such as sickle cell disease. The present invention describes compositions and methods for use in gene therapy and genome engineering.

The present invention provides compounds useful as medicaments for the treatment of sickle cell disease and diseases characterized by undesirable or abnormal cell proliferation. These compounds are substituted triarylmethane compounds or analogs thereof, wherein one or more aryl groups are partially substituted by heteroaryl, cycloalkyl or heterocycloalkyl groups and/or wherein the quaternary carbon is replaced by a different atom such as silicon, germanium , nitrogen or phosphorus substitution. They inhibit mammalian cell proliferation, inhibit the Gardos channel in cytoplasm, reduce sickle cell dehydration and/or delay red blood cell sickling and deformation.

A phytochemical composition for treatment of sickle cell anemia has been developed. The composition is a solid mixture of inorganic salts and various organic compounds of herbal origin and obtained by extracting a mixture of five herbs namely Piper guineenses seeds, Pterocarpus osum stem, Eugenia carophyllum fruit, Sorghum bicolor leaves and Curcuma longa tuber with aquous solution of sodium/potassium bicarbonate and sodium/potassium carbonate and then concentrating the extract to obtain a powder. Also described are methods of preparation of extraction product and method of its use for treatment of sickle cell disease afflicted patients.

The #β-hemoglobinopathies #β-thalassemia (BT) and sickle cell disease (SCD) are the most frequent genetic disorders worldwide. These diseases are caused by mutations causing reduced or abnormal synthesis of the β-globin chain of the adult hemoglobin (Hb) tetramer. Here, the inventors intend to improve HSC-based gene therapy for β-thalassemia and SCD by developing an innovative, highly infectious LV vector expressing a potent anti-sickling β-globin transgene and a second biological function either increasing fetal γ-globin expression (for β-thalassemia and SCD). More particularly, the inventors have designed a novel lentivirus (LV), which carry two different functions: βAS3 gene addition and gene silencing. This last strategy allows the re-expression of the fetal γ-globin genes (HBG1 and HBG2) and production of the endogenous fetal hemoglobin (HbF). Elevated levels of HbF and HbAS3 (Hb tetramer containing βAS3-globin) will benefit the β-hemoglobinopathy phenotype by increasing the total amount of β-like globin that will: (i) reduce the alpha precipitates and improve the alpha/non alpha ratio in β-thalassemia, and (ii) reduce the sickling in SCD. This combined strategy will improve the β-hemoglobinopathy phenotype at a lower vector copy number (VCN) per cell compared to a LV expressing the βAS3 alone.

The present invention provides a compound of Formula (I) and the pharmaceutically acceptable salts, esters, and prodrugs thereof, which are PRMT5 inhibitors. Also provided are methods of making compounds of Formula I, pharmaceutical compositions comprising compounds of Formula I, and methods of using these compounds to treat cancer, sickle cell, and hereditary persistence of foetal hemoglobin (HPFH) mutations.