摘要:
The present invention relates, first, to methods for the modulation of acid sphingomyelinase (ASM)-related processes, including apoptosis. Such apoptosis can include, but is not limited to, environmental stress-induced apoptosis such as, for example, ionizing radiation and/or chemotherapeutic agent-induced apoptosis. Apoptosis can be characterized by a cellular morphology comprising cellular condensation, nuclear condensation or zeiosis. The present invention further relates to methods for the identification of compounds which modulate (i.e., either increase or decrease) sensitivity to ASM-related processes, including apoptosis.
摘要:
The present invention relates, first, to methods and compositions for the modulation of acid sphingomyelinase (ASM)-related processes, including apoptosis. Such apoptosis can include, but is not limited to, environmental stress-induced apoptosis such as, for example, ionizing radiation and/or chemotherapeutic agent-induced apoptosis. Apoptosis can be characterized by a cellular morphology comprising cellular condensation, nuclear condensation or zeiosis. The present invention further relates to methods for the identification of compounds which modulate (i.e., either increase or decrease) sensitivity to ASM-related processes, including apoptosis.
摘要:
The present invention relates to methods of treating a subject with a lysosomal disorder, by administering an agent for enzyme replacement therapy and an agent for anti-TNF-α therapy; by administering a pentosan polysulfate therapy; or by administering a substrate reduction therapy and an anti-TNF-α therapy. The invention further relates to a method of reducing inflammatory cytokines in a subject with a lysosomal disorder that is being treated by enzyme replacement therapy, by administering an agent for anti-TNF-α therapy.
摘要:
The present invention relates to the acid sphingomyelinase gene and to methods of diagnosing Niemann-Pick disease. It is based, at least in part, on the cloning and expression of the full-length cDNA encoding acid sphingomyelinase and on the discovery of mutations in the acid sphingomyelinase gene of Ashkenazi Jewish Niemann-Pick disease patients.
摘要:
The present invention relates to methods of producing chondrocytes, improving the phenotype of a chondrocyte population, promoting chondrogenesis, maintaining a cell population in a differentiated state or increasing the number of cells of a population in a differentiated state, producing a population of differentiated cells, and treating certain diseases or disorders. Each of these methods involves treating a cell population with, and/or culturing a cell population in, a ceramidase.
摘要:
The present invention relates to methods of treating a subject with a lysosomal disorder, by administering an agent for enzyme replacement therapy and an agent for anti-TNF-α therapy; by administering a pentosan polysulfate therapy; or by administering a substrate reduction therapy and an anti-TNF-α therapy. The invention further relates to a method of reducing inflammatory cytokines in a subject with a lysosomal disorder that is being treated by enzyme replacement therapy, by administering an agent for anti-TNF-α therapy.
摘要:
The present invention relates to the acid sphingomyelinase gene and to methods of diagnosing Niemann-Pick disease. It is based, at least in part, on the cloning and expression of the full-length cDNA encoding acid sphingomyelinase, the cloning and characterization of the genomic structure of the acid sphingomyelinase gene, and on the discovery of frequent mutations in the acid sphingomyelinase gene of Ashkenazi Jewish Niemann-Pick disease patients.
摘要:
The present invention provides a method for treating individuals affected with the acid sphingomyelinase-deficient forms of Niemann-Pick disease (i.e., Type A or Type B Niemann-Pick) by administering small molecules as specific molecular “chaperones” for the deficient acid sphingomyelinase (ASM) enzyme associated with the disease. The molecules are ceramide, sphingomyelin, or phosphonucleotide analogues.
摘要:
The present invention relates to the acid sphingomyelinase gene and to methods of diagnosing Niemann-Pick disease. It is based, at least in part, on the cloning and expression of the full-length cDNA encoding acid sphingomyelinase and on the discovery of mutations in the acid sphingomyelinase gene of Ashkenazi Jewish Niemann-Pick disease patients.
摘要:
The present invention relates to methods of promoting the survival of cells by treating the cells with acid ceramidase. A kit for promoting ex vivo cell survival is also disclosed, as is a method of predicting in vitro fertilization outcome of a female subject.