8 results about "Beta-thalassaemia" patented technology

The invention relates to the use of ferroportin inhibitor compounds of the general formula (I) for treating transfusion-dependent β-thalassemia.

The invention relates to the technical field of medicines, in particular to application of huperzine A in treatment of beta-thalassemia. It is found for the first time that the anti-Alzheimer disease drug huperzine A can be directly combined with gamma-globin to inhibit degradation of gamma-globin through a ubiquitin-proteasome pathway, so that the stability of gamma-globin and the fetal hemoglobin level are improved; the abnormal erythropoiesis and hemolysis phenotype of the beta-thalassemia are improved under the condition of not changing the mRNA expression. Animal experiments show that huperzine A obviously improves hematocrit and the number of red blood cells, reduces the size distribution width of the red blood cells and the proportion of reticulocytes, relieves oxidative stress, hemolysis, splenomegaly and iron overload, and has no obvious liver and kidney toxicity. The invention creatively provides a new strategy for improving HbF by regulating and controlling protein homeostasis instead of transcriptional up-regulation, and provides a new drug action mechanism and application direction for treatment of beta-thalassemia and other hemoglobin diseases.

Disclosed herein are potency assays for a gene therapy treatment for β-thalassemia. Also disclosed herein are methods for measuring relative potency of a drug product.

This invention provides vectors (e.g., lentiviral vectors) and expression cassettes for incorporation into vectors for expressing an alpha-globin (α-globin) gene. In certain embodiments method of treatment using such vectors are provided.

Antibodies to the enzyme matriptase-2 (MTP-2) are presented. Inhibiting MTP-2 reduces uptake of dietary iron and reduces the release of iron from cellular stores in the body. Inhibitors of MTP-2 (such as antibodies to the serine protease domain) can be used to treat iron overload, which is a feature of diseases such as beta-thalassaemia and which otherwise leads to toxic accumulation of iron. Combination of an MTP-2 inhibitor with an activin receptor ligand trap, or with erythropoietin, provides additional therapeutic effects.

The invention relates to a composite peptide nutritional composition as well as a preparation method and application thereof, and belongs to the technical field of functional foods. The composite peptide nutritional composition comprises a hematopoietic function regulating system (such as cattle spleen peptide, blood protein polypeptide and salmon hydrolyzed protein peptide), an iron metabolism balance regulating system (concentrated hawthorn clear juice) and a nutrition absorption promoting system (endothelium corneum gigeriae galli peptide and pericarpium citri reticulatae powder) which have a synergistic effect. The traditional Chinese medicine composition does not contain an exogenous iron agent, and aims to improve malnutrition caused by chronic hemolytic diseases such as thalassemia. The uniformity and the stability of the product are guaranteed through a specific proportioning and a staged mixing preparation process. Animal experiments show that the composition can significantly improve hemoglobin and red blood cell count of beta-thalassemia model mice, effectively control serum iron and liver iron accumulation levels of the mice, show unique advantages of efficient hematopoiesis and low iron accumulation, and has broad application prospects. And a new nutrition support scheme is provided for improving anemia and abnormal iron metabolism conditions caused by chronic hemolysis.

Provided herein, inter alia, are conjugates comprising a targeting agent (e.g., a hematopoietic cell (e.g., erythroid precursor cell) targeting agent) comprising a protein (e.g., an antibody) that specifically binds to the transferrin receptor (TFR) (e.g., human TFR (hTFR) (e.g., hTFR1)); (b) at least one oligonucleotide operably linked to (b) at least one oligonucleotide that modulates the expression and / or activity of a target gene, nucleic acid (e.g., mRNA) and / or protein expressed by a target cell; as well as methods of making the conjugates and pharmaceutical compositions comprising the conjugates. Further provided herein are methods of utilizing the conjugates, including, for example, methods of treating hemoglobinopathy (e.g., sickle cell disease (SCD) or thalassemia (e.g., alpha-thalassemia, beta-thalassemia, delta-thalassemia, or gamma-thalassemia)).