49 results about "Sickle cell anemia" patented technology

Methods are provided for treating hematological disorders by inhibition of DNA hypomethylation and histone deacetylase. Such disorders include, for example, acute promyelocytic leukemia, acute lymphoblastic leukemia, chronic myelogenous leukemia, myelodysplastic syndromes, and sickle cell anemia. The methods comprise: administering to a patient suffering from the disease a therapeutically effective amount of a DNA methylation inhibitor such as a cysteine analog such as decitabine, in combination with an effective amount of histone deacetylase inhibitor such as hydroxamic acid, cyclic peptide, benzamide, butyrate, and depudecin.

The present invention relates to immunomodulatory compounds, in particular a class known as IMiD TM , more specifically the compounds 4-(amino)-2-(2,6-dioxo(3-piperidinyl))-isoindoline-1,3-dione and 3-(4- Use of amino-1-oxo-1,3-dihydroisoindol-2-yl)-piperidine-2,6-dione for use in CD34 + The expression of genes for fetal hemoglobin, genes essential for erythropoiesis, and genes encoding alpha hemoglobin stabilizing proteins is induced in the cell population. These compounds are useful in the treatment of hemoglobinopathy, such as sickle cell anemia or beta-thalassemia, or anemia caused by disease, surgery, accident, or the introduction or ingestion of toxins, poisons or drugs.

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

The present invention relates to compounds of formula (I) and (IV), and pharmaceutically acceptable salts, tautomers, stereoisomers thereof, which are inhibitors of histone deacetylase (HDAC). The compounds of the present invention are for use in the treatment of disorders that are ameliorated by inhibition of HDAC such as cancer and hemoglobinopathies like β-thalassemia or sickle cell anemia.

The instant invention provides methods and compositions for the treatment, prevention and diagnosis of for example, platelet aggregation or clot formation in a subject. The invention inhibits the activity of decreases the amount of neutrophils in the subject by inhibiting the activity or production of IL-6, interferon-gamma, STAT1, or cathepsin G. The invention addresses decreasing the amount of neutrophils in an attempt to treat subjects that have or are at risk of developing a vascular occlusive disease, an ischemia or reperfusion injury, an acute or chronic inflammatory state, autoimmune disease, myelodysplastic syndrome, tissue injury from surgery or accidental trauma, acute bacterial or viral infection, has undergone a microvascular surgical reconstructive procedure, is receiving granulocyte colony stimulating factor therapy, receiving stem cell therapy, or has sickle cell anemia.

The combination of nitric oxide generating compounds which are not dependent upon aldehyde dehydrogenase for bioactivation, or are specifically targeted to nNOS or the sarcoplasmic reticulum of cardiac muscle cells, and xanthine oxidase inhibitors are effective in the treatment of heart disease, specifically congestive heart failure and ischemic coronary disease. This treatment is particularly effective in patients who have particularly heavy oxidative burdens, e.g. diabetics, patients with lung disorders, patients with sickle cell anemia and patients of Asian descent.

The invention provides methods of treating hyperglycemia induced Red Blood Cell Disease / Dysfunction (RBCD) caused by generation of early and late glycation end products. The invention provides a method of treating hemolytic anemia, smoldering hemolytic anemia, sickle cell anemia, hemorrhagic diseases, hemorrhagic stroke, hemorrhagic bleeding. Method of treating RBCD to prevent progression to diabetes associated vascular complications is referred to as Syndrome X, particularly to prevent progression to chronic kidney disease, coronary vascular disease, and peripheral vascular disease.

Disclosed are methods of treating pain in a mammal, which may include the step of administering human growth hormone to a mammal in need thereof. The pain treated by the disclosed methods may be of a type caused by inflammation induced mechanical and / or thermal hypersensitivity, and may include, for example, a pain type resulting from one or more conditions selected from peripheral injury pain, post-operative pain, cutaneous inflammation, cutaneous incision, muscle incision, or chronic pain. Disease states in which the disclosed methods may be used include fibromyalgia, sickle cell anemia, epidermolysis bullosa, erythromelalgia, complex regional pain syndrome, or generalized muscle pain.

Disclosed are methods of treating pain in a mammal, which may include the step of administering human growth hormone to a mammal in need thereof. The pain treated by the disclosed methods may be of a type caused by inflammation induced mechanical and / or thermal hypersensitivity, and may include, for example, a pain type resulting from one or more conditions selected from peripheral injury pain, post-operative pain, cutaneous inflammation, cutaneous incision, muscle incision, or chronic pain. Disease states in which the disclosed methods may be used include fibromyalgia, sickle cell anemia, epidermolysis bullosa, erythromelalgia, complex regional pain syndrome, or generalized muscle pain.