17 results about "Sickle cells" patented technology

The present invention provides a liquid composition comprising effective amount of Hydroxyurea for oral administration. The invention further discloses liquid formulation in form of ready to use oral suspension which remains stable at room temperature at the time of dispensing, storage and even after reopening of the container comprising the suspension. Also provided is a method of preparing the formulation and treating sickle cell anemia in pediatric patients.

The present invention discloses the preparation of antibodies against sickle cell hemoglobin variants and the application of a typing detection kit, belonging to the field of biomedicine. By comparing the sequences of different hemoglobins, the present invention analyzes the key mutation points of sickle cell hemoglobin. A prokaryotic expression system is used to recombinantly express the hemoglobin α chain (HBA) and hemoglobin β chain (including HBB, HBS, and HBC). The target proteins of different typings are then conjugated with KLH to prepare immunogens. Animals are then immunized and screened for antibodies targeting the different hemoglobin variants. Rabbit antibodies are obtained for HBB and HBS proteins, while mouse antibodies are obtained for HBA and HBC. DNA sequencing is used to analyze the antibody sequence information. The present kit has high sensitivity and specificity, and can rapidly distinguish hemoglobin typing, thus overcoming the shortcomings of existing detection technologies and assisting in clinical infection control and treatment.

Disclosed herein are potency assays for a gene therapy treatment for sickle cell disease. Also disclosed herein are methods for measuring relative potency of a drug product used for the treatment of sickle cell disease.

The invention discloses preparation of an anti-sickle cell anemia hemoglobin variant antibody and application of a typing detection kit, and belongs to the field of biological medicine. Through sequence comparison of different hemoglobin, key mutation point structures of hemoglobin of sickle anemia are analyzed, a prokaryotic expression system is adopted for recombinant expression of hemoglobin alpha chains (HBA) and hemoglobin beta chains (containing HBB, HBS and HBC), and target proteins of different types are coupled with KLH to prepare immunogens. Respectively carrying out animal immunization and screening corresponding antibodies aiming at different mutants of hemoglobin. Wherein the HBB protein and the HBS protein obtain rabbit source antibodies, and the HBA protein and the HBC protein obtain mouse source antibodies; according to the present invention, the kit has advantages of high sensitivity, good specificity, rapid detection capable of distinguishing hemoglobin typing, compensation of the defects of the existing detection technology, and clinical infection control and treatment assistance.

The present invention provides a compound of formula (I) Formula (I) and the pharmaceutically acceptable salts, esters, and prodrugs thereof, 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.

The present invention provides a compound of Formula (I) (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.

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

The present disclosure provides methods and compositions for treating or preventing a sickle cell disease (SCD). The SCD may include sickle cell anemia (SCA). The method may include administering a composition such as a cyclodextrin to a subject in need of SCD treatment. Some embodiments include contacting a RBC with a cyclodextrin.

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.

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

The invention relates to the method of use of a pharmaceutical composition containing hydroxyurea, for the treatment of severe chronic anemia in a patient suffering from sickle cell anemia not already undergoing treatment with hydroxyurea and not suffering a vaso-occlusive crisis.

Disclosed is a method of using Netrin-1 (NTN1) driven rejuvenation of an aged hematopoietic system based on its shown dependency of reestablishing integrity of aged bone marrow niche and reactivating DNA damage response (DDR). NTN1 is shown as a master regulator of reactivating DNA damage response (DDR) pathways. Every organ / tissue accumulates DNA damage that is an underlying cause of diseases. Reactivating DDR has an extensive effect on treating diseases. NTN1 serves as a therapeutic modality that effectively reverses age-related hematopoietic deficiencies while simultaneously targeting growth and survival of acute myelogenous leukemia (AML). The ability to target and gene correct hematopoietic stem cells (HSC) ex vivo for the treatment of hemoglobinopathies, like thalassemia and sickle cell anemia, is limited due to inability of transducing the correct cell population. NTN1 is shown to maintain and expand bona fide HSCs, opening up the possibility to transduce a true stem cell overcoming previous limitations.

The utility model discloses a variety of hemoglobin in vitro simultaneous detection test strip, including a substrate and set on the substrate and along the chromatography direction contact setting one washing pad, one mark pad, one lysis pad, one reaction pad and one absorption pad. The utility model discloses can detect HbA, HbS and HbC simultaneously and so on variety of hemoglobin, is applicable to the rapid screening and clinical diagnosis of abnormal hemoglobinopathy such as sickle cell anemia, and is simple to operate, and the cost is low.

The present invention relates to a method to determine the concentration of sickle-cell haemoglobin in blood. Said method is based on a smear technique involving drawing a smear at a particular speed, wherein the length of the smear is a function of the concentration of the HbS. It also provides a smear card having graduations to measure the smear length and the corresponding viscosity (in cP) and a device comprising such smear card; reducing agent and substrate.