49 results about "Sickle cell anemia" patented technology

The present invention is directed to the use of immunomodulatory compounds, particularly members of the class of compounds known as IMiDs™, and more specifically the compounds 4-(Amino)-2-(2,6-dioxo(3-piperidyl))-isoindoline-1,3-dione and 3-(4-amino-1-oxo-1,3-dihydroisoindol-2-yl)-piperidine-2,6-dione, to induce the expression of fetal hemoglobin genes, genes essential for erythropoiesis, and genes encoding alpha hemoglobin stabilizing protein, within a population of CD34+ cells. These compounds are used to treat hemoglobinopathies such as sickle cell anemia or β-thalassemia, or anemias 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.

A compound having the formula (I) or a pharmaceutically acceptable salt thereof where the variables are defined in the specification are useful in the treatment of sickle-cell anemia.

The present invention relates to antagonists of vertebrate growth hormones obtained by mutation of the third alpha helix of such proteins (especially bovine or human GHs). These mutants-have growth-inhibitory or other GH-antagonizing effects. These novel hormones may be administered exogenously to animals, or transgenic animals may be made that express the antagonist. Animals have been made which exhibited a reduced growth phenotype. The invention also describes methods of treating acromegaly, gigantism, cancer, diabetes, vascular eye diseases (diabetic retinopathy, retinopathy of prematurity, age-related macular degeneration, retinopathy of sickle-cell anemia, etc.) as well as nephropathy and other diseases, by administering an effective amount of a growth hormone antagonist. The invention also provides pharmaceutical formulations comprising one or more growth hormone antagonists.

A pharmaceutical composition is disclosed comprising sodium 4-phenylbutyrate, an effective amount of at least one aromatic flavoring agent, and an effective amount of at least one synthetic sweetening agent. Also disclosed is a method of treatment of a urea cycle deficiency or sickle-cell anaemia.

The present invention generally relates to compositions and methods for treatment of subjects having or at risk of arteriosclerosis, hypertension, sickle-cell anemia, or other conditions. In some cases, the composition may include nitric oxide. The nitric oxide may be present within a first phase comprising a lecithin, such as phosphatidylcholine. In certain embodiments, the lecithin is present in liposomes, micelles, or other vesicles containing nitric oxide. The composition can take the form of a gel, a cream, a lotion, an ointment, a solution, a solid “stick,” etc., that can be rubbed or sprayed onto the skin, e.g., onto a location with acne, or on another suitable portion of the skin. Other aspects of the present invention are generally directed to methods of making or using such compositions, methods of promoting such compositions, kits including such compositions, or the like.

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 invention describes methods for treating blood disorders or for treating the symptoms and / or complications associated with blood disorders by administering a therapeutically effective amount of at least one nitric oxide donor compound and optionally at least one antioxidant, or a pharmaceutically acceptable salt thereof, and / or at least one therapeutic agent. The antioxidant is preferably a hydralazine compound or a pharmaceutically acceptable salt thereof. The nitric oxide donor compound is preferably N-hydroxy-L-arginine and / or isosorbide dinitrate and / or isosorbide mononitrate. The blood disorder is preferably sickle cell anemia. The complication resulting from a blood disorder is preferably pulmonary hypertension.

A lymphocytotoxic, but hematopoietic stem cell-sparing, high-dose amount of an oxazaphosphorine drug such as, for example, cyclophosphamide, administered post-transplantation can be used to reduce transplant rejection, including graft-versus-host-disease (GVHD). In some embodiments, the transplants are bone marrow transplants or hematopoietic stem cell transplants carried out for the treatment of hematologic disorders, including hematologic malignancies and non-malignant hematologic disorders. In some embodiments, the transplants are carried out for the treatment of hereditary hemoglobinopathies, such as sickle cell anemia and thalassemia.

A nutritional supplement composition for treating nutritional deficiencies caused by a medical condition in subjects is disclosed. The present application further discloses a method of using a nutritional supplements composition for treating a subject with complications resulting from sickle cell anemia. The method comprises administering to a subject an effective amount of the nutritional supplement.

The invention discloses a lentiviral vector suitable for gene therapy of thalassemia and sickle anemia. The lentiviral vector comprises a beta globin expression cassette; the expression cassette comprises a micro-locus control area, a gene sequence of a beta globin, a promoter sequence of a flank of the upstream of the gene sequence of the beta globin, and a flanking sequence of the downstream ofthe gene sequence of the beta globin; the micro-locus control area is a micro-control element which is screened out from the locus control area of beta globin 16kb and does not contain an HS1 area. Compared with the prior art, the screened beta globin expression cassette has both efficiency and specificity, and does not cause reduction of lentivirus titer; a screened insulator sequence which comesfrom foamy virus and is only 36 bp does not contain a hidden RNA splicing signal, and has the functions of maintaining gene expression and preventing gene activation in a region where the insulator sequence is located; the lentiviral vector can be used for gene therapy of thalassemia and sickle anemia.

The present invention relates to vectors comprising an alpha -globin locus control region ( alpha LCR) and a gene encoding an erythroid protein. In particular embodiments, a retroviral vector comprising an alpha LCR and a globin gene may be used to treat globin-based genetic disorders, including sickle cell anemia and beta -thalassemia.

The present invention provides methods for discovering agents that are effective in reversing epigenetic silencing by inhibiting the interaction of methyl-binding (MBD) proteins with methylated genomic DNA. Also provided are methods for reactivating silenced genes having CpG island hypermethylation along with methods for treatment and prevention of diseases, such as cancer and sickle cell anemia, by administering an agent that modulates methyl-binding domain (MBD) protein-mediated transcriptional repression, thereby increasing gene transcription to prevent or treat disease. Additionally, compounds identified by the present invention useful for treatment and prevention of diseases, such as cancer and sickle cell anemia, are provided.

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 invention provides gRNA for knocking out BCL11A genes or BCL11A gene enhancers, a gRNA composition, an expression vector, CRISPR-Cas9 RNP, a CRISPR-Cas9 RNP composition, a CRISPR-Cas9 system, an electrorotation method, a reagent kit and applications thereof. A CRISPR-Cas9 technique is adopted to perform targeted cutting on hemopoietic stem cells, so that the BCL11A gene expression amount is reduced, and the hemochrome expression quantity of fetuses is high. The technique is hopeful to become a new means for treating thalassaemia and sickle cell anemia. The CRISPR-Cas9 technique is adoptedto realize mutation of BCL11A genes. The design is simple, the use is convenient, the cost is low, and the efficiency is high.

Transplant of donor perfectly matched HLA hematopoietic stem cell to cure Sickle cell anemia and other anemia such as leukemia. Sterilized In vivo transplantation of clinically adequate quantities of antibiotic protected HLA vector / or insertion corrected chimera stem cells, and switching protein. Stem cells can be transfected for Hbg SS, and other proteins such as minor HLA type that may cause Graft versus host disease (GvHD) or Host versus Graft disease (HvGD Universal donor blood, Rh-negative of any HLA type can be corrected to perfectly match that of any recipient. Batch universal stem calls are grown and selectively transformed to a chimera stem cell. The chimera stem cells are incubated in a bio-reactor in growth medium also containing human growth and maturation promotion polypeptide factors. The harvest is then prepared for clinical use and transplantation into the matching recipient. Recipient's stem cells are transformed by transfection or insertion of the beta hemoglobin gene.

The invention discloses a virus vector for expressing recombinant human beta-globin and application of the virus vector. The invention provides a DNA fragment A which is formed through linking of thefollowing components from a 5' terminal to a 3' terminal: three DNA enzyme I hypersensitive sites, an HBB promoter, a translation enhancer, an HBB gene expression frame and an HBB transcription enhancer. An HBB expression vector provided by the invention has the capability of efficiently expressing exogenous HBB proteins and has extremely high application values in treatment of beta-thalassemia and sickle cell anemia.

The invention belongs to the technical field of biological medicine, discloses an application of ADDA and / or DMTA in preparing medicine for improving expression level of gamma-globin, and provides a candidate drug for curing anemia caused by beta-thalassemia, sickle-cell anemia, hematopoietic system tumor and hematopoietic dysfunction.

The invention relates to an algorithm for judging living system disease gene reaction rate, in particular to an allele competing reaction QM / MM method in living system. The method comprises the steps that 1, corresponding molecular crystal structure files are downloaded from databases of proteins, nucleic acid and the like, and multiple reasonable molecular structures are selected from the molecular crystal structure files; 2, the energy, electric charge and energy gradient of the MM part are calculated through a Tinker program; 3, the QM part is optimized through a Gaussian98 program; 4, configuration optimization is conducted on the MM part through a Newton program in tinker; 5, proper reaction coordinates are selected; 6, a reaction path is obtained, and calculation is conducted. According to the calculation method, proper therapeutic schemes can be found for genetic diseases such as sickle cell anemia, that is to say, the conditions of enabling a disease gene not to be capable of generating a reaction or enabling the reaction rate to be lower than that of a normal gene are sought. The method is very meaningful for medical science.

The disclosure provides in vitro and in vivo methods for identifying Heparins and Heparinoids that modulate the activity of selectins . The disclosure also provides Heparins and Heparinoids that modulate the activity of selectins . The identification and isolation of these heparin formulations has the potential to mediate a wide variety of pathologies mediated by P- and / or L-selectin, including hematogenous metastasis, diseases associated with inflammation (e.g., asthma, arthritis, allergic dermatitis), ischemia-reperfusion injury, or other pathologies such as sickle cell anemia. Selectin inhibition can be achieved at plasma concentrations lower than those that cause excessive anticoagulation or unwanted bleeding in a human subject.

The application discloses a recombinant lentivirus vector for treating beta-globulin afunction, a preparation method and application. A gene element of the recombinant lentivirus vector disclosed by the application is composed of a left side lentivirus long terminal repeat, a lentivirus counter-response element, a center poly-purine sequence, a gene locus regulating and controlling sequence of a beta-globulin gene, a beta-globulin gene, of which 87-th threonine is mutated into glutamine, and a right side lentivirus long terminal repeat which are connected in order. According to the recombinant lentivirus vector disclosed by the application, through optimizing and improving the gene element, the gene transduction efficiency is increased, and the beta-globulin gene can be efficiently expressed during erythroid differentiation of hemopoietic stem cells, so that the consumption of viruses can be effectively lowered, and the treatment cost is reduced while the safety is improved. The recombinant lentivirus vector disclosed by the application provides a novel treatment tool and scheme for beta-thalassemia and sickle cell anemia caused by the beta-globulin afunction.

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.

The invention relates to tea which comprises the main components of sow thistle and Chinese data with the mixing proportion of 5:1. A making method of the tea comprises the following steps of: cleaning selected sow thistle tender leaves, drying in air, disinfecting at high temperature, carrying out deactivation under high speed, mixing the processed sow thistle leaves with tea leaves, making shapes, frying, polishing, drying and improving fragrance. The invention has the pharmacologic functions of clearing heat, detoxicating, breaking bruise, activating blood, eliminating swelling, and stopping dysentery. The tea is a good health-care drinking product for people to prevent and treat sickle-cell anemia, maintain the normal physiological activity of a human body, promote growth and development and relieve summer heat.

The invention provides gene detecting primers of sickle cell anemia and a composition of the gene detecting primers and belongs to the technical field of anemia detection. The gene detecting primers and the composition thereof are characterized in that on the basis of LAMP reaction, the Allele-Specific LAMP technology is used for respectively designing specific primers aiming at the wild type and mutant type of the gene sequences of coding hemoglobin beta chains, and the genotypes related to the sickle cell anemia can be detected fast and accurately. The gene detecting primers and the composition thereof are low in cost, short in detecting time, high in accuracy and easy to popularize.