48 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.

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.

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.

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.

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 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.

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.