Patent classifications
C12N2510/02
VESICLE AND USE THEREOF
Provided are a vesicle and the use thereof. The vesicle is an induced vesicle, and the sources thereof include stem cells or somatic cells, and the possessed markers include Syntaxin 4. Compared with an exosome in mesenchymal stem cells, the vesicle can specifically express Syntaxin 4 and can be used to distinguish characteristic markers of MSC-derived vesicles and exosomes. The vesicle can play a procoagulant effect in vitro, can improve the bleeding tendency of mice with hemophilia after in vivo injection, and can be used for the treatment of improving the bleeding tendency of hemophilia. In addition, the vesicle can be expelled through the skin and hair.
PRODUCTION CELL AND PACKAGING CELL FOR RETROVIRAL VECTOR AND PREPARATION METHOD THEREFOR
The present disclosure relates to a method for constructing a producer cell and the producer cell obtained by the method, wherein the producer cell is for producing a retroviral vector carrying a nucleic acid fragment of interest.
MONOCLONAL CELL LINES EXPRESSING AN EXOGENOUS SUBSTANCE AND USES THEREOF
Described herein are genetically modified cells derived from a human cell and which contain at least one exogenous transcription unit inserted into at least one of four open chromatin regions (OCRs) located on Chromosomes 2, 5, 8 and 12, as well as compositions, pharmaceutical preparations, and implantable devices comprising the genetically modified cells, and methods of using the same for preventing or treating a disease, disorder, or condition.
Therapeutic Engineered Microbial Cell System and Methods for Treating Hyperuricemia and Gout
The present disclosure relates to engineered microbial cells that have been engineered to include a uricase, a uric acid transporter, or both a uricase and a uric acid transporter. The engineered microbial cells of the present disclosure are useful in degrading uric acid inside the engineered microbial cell. The engineered microbial cells of the present disclosure are useful in methods of treating hyperuricemia. The engineered microbial cells of the present disclosure are also useful in methods of treating gout, and in particular chronic refractory gout.
Highly potent acid alpha-glucosidase with enhanced carbohydrates
Recombinant human alpha glucosidase (rhGAA) composition derived from CHO cells that contains a more optimized glycan composition consisting of a higher amount of rhGAA containing N-glycans carrying mannose-6-phosphate (M6P) or bis-M6P than conventional rhGAAs, along with low amount of non-phosphorylated high mannose glycans, and low amount of terminal galactose on complex oligosaccharides. Compositions containing the rhGAA, and methods of use are described.
METHODS FOR INCREASING MANNOSE CONTENT OF RECOMBINANT PROTEINS
The present invention relates to methods of modulating the mannose content of recombinant proteins.
ANTI-CXCR4 ANTIBODIES AND ANTIBODY-DRUG CONJUGATES
The present invention provides antibodies and related molecules that bind to chemokine receptor 4 (CXCR4). The invention further provides antibody-drug conjugates comprising such antibodies, antibody encoding nucleic acids, and methods of obtaining such antibodies. The invention further relates to therapeutic methods for use of these antibodies and anti-CXCR4 antibody-drug conjugates for the treatment of a disorder associated with CXCR4 function or expression (e.g., cancer), such as colon, RCC, esophageal, gastric, head and neck, lung, ovarian, pancreatic cancer or hematological cancers.
B CELLS FOR IN VIVO DELIVERY OF THERAPEUTIC AGENTS
The present disclosure relates to genetically modified B cells, including memory cells, differentiated to plasmablasts or plasma cells useful for long tem in vivo expression of a transgene, such as a specific antibody or other protein therapeutic. Also disclosed are methods of producing the cells and methods of treatment.
METHODS AND CONSTRUCTS FOR TRANSIENT PRODUCTION OF LENTIVIRAL VECTOR
The present disclosure relates to methods for producing lentiviral vectors using mammalian cells. Specifically, the methods utilize three plasmids, rather than four, to provide the required packaging elements and transfer vector to a cell, allowing for the production of a large number of lentiviral vectors in mammalian cells, including suspension-based cells. These methods allow for the production of lentiviral vectors that can be tailored to include a specific gene of interest.
Methods of treating neurodegenerative disorders
A method of treating a mammal with one of a central nervous system injury and a neurodegenerative disorder comprising isolating, culturing, and generating neural progenitor cells from a mammalian placenta, and transplanting the placenta derived neural progenitor cells into a brain of the mammal.