摘要:
Substituted pyrazolopyrimidines and dihydropyrazolopyrimidines and related compounds, their methods of manufacture, compositions containing these compounds, and methods of use of these compounds in treating lysosomal storage disorders such as Gaucher disease are described herein. The compounds are of general Formula (I) in which variables R1-R7 and X are described in the application.
摘要:
Methods of obtaining a cell population enriched for tumor-reactive T cells, the method comprising: (a) obtaining a bulk population of peripheral blood mononuclear cells (PBMCs) from a sample of peripheral blood; (b) specifically selecting CD8+T cells that also express PD-1 and/or TIM-3 from the bulk population; and (c) separating the cells selected in (b) from unselected cells to obtain a cell population enriched for tumor-reactive T cells are disclosed. Related methods of administering a cell population enriched for tumor-reactive T cells to a mammal, methods of obtaining a pharmaceutical composition comprising a cell population enriched for tumor-reactive T cells, and isolated or purified cell populations are also disclosed.
摘要:
Disclosed are methods of enhancing the chemotherapeutic treatment of tumor cells, reducing resistance of a cancer cell to a chemotherapeutic agent, a method of inhibiting ABCG2 or MRP1 in a mammal afflicted with cancer, and a method of increasing the bioavailability of an ABCG2 substrate drug in a mammal. The methods comprise administering peliomycin and other compounds described herein.
摘要:
The invention provides human cells, particularly human T cells, comprising a murine T Cell Receptor (TCR) having antigen specificity for the cancer antigen gp100. Isolated or purified TCRs having antigenic specificity for amino acids 154-162 of gp100 (SEQ ID NO: 1), as well as related polypeptides, proteins, nucleic acids, recombinant expression vectors, host cells, populations of cells, antibodies, or antigen binding fragments thereof, conjugates, and pharmaceutical compositions, are further provided. The invention further provides a method of detecting the presence of cancer in a host and a method of treating or preventing cancer in a host comprising the use of the inventive materials described herein.
摘要翻译:本发明提供了包含对癌抗原gp100具有抗原特异性的鼠T细胞受体(TCR)的人细胞,特别是人T细胞。 具有对gp100(SEQ ID NO:1)的氨基酸154-162具有抗原特异性的分离或纯化的TCR,以及相关多肽,蛋白质,核酸,重组表达载体,宿主细胞,细胞群,抗体或抗原结合 其片段,缀合物和药物组合物。 本发明还提供了检测宿主中癌症存在的方法以及在宿主中治疗或预防癌症的方法,所述方法包括使用本文所述的本发明材料。
摘要:
The present invention provides compositions comprising nucleic acids that target CSN5 gene expression and methods of using such compositions to silence CSN5 gene expression. More particularly, the present invention provides unmodified and chemically modified interfering RNA molecules which silence CSN5 gene expression and methods of use thereof, e.g., for treating cell proliferative disorders such as cancer. The present invention also provides nucleic acid-lipid particles that target CSN5 gene expression comprising an interfering RNA molecule, a cationic lipid, a non-cationic lipid, and optionally a conjugated lipid that inhibits aggregation of particles.
摘要:
The allelic variants or mutations in three genes: GNPTAB, GNPTG and NAGPA, that correlate with stuttering in humans, as well as the encoded mutated polypeptides and related vectors, host cells, antibodies, antibody-producing cell lines and methods of diagnosing, prognosticating and treating stuttering are provided.
摘要:
The invention provides methods of detecting abnormal cells in a sample using the radial position of one or more genes within the nucleus of a cell, as well as a kit for detecting abnormal cells using such methods. The invention also provides methods of identifying gene markers for abnormal cells using the radial position of one or more genes within the nucleus of a cell.
摘要:
The invention provides human cells, particularly human T cells, comprising a murine T Cell Receptor (TCR) having antigen specificity for the cancer antigen gp100. Isolated or purified TCRs having antigenic specificity for amino acids 154-162 of gp100 (SEQ ID NO: 1), as well as related polypeptides, proteins, nucleic acids, recombinant expression vectors, host cells, populations of cells, antibodies, or antigen binding fragments thereof, conjugates, and pharmaceutical compositions, are further provided. The invention further provides a method of detecting the presence of cancer in a host and a method of treating or preventing cancer in a host comprising the use of the inventive materials described herein.
摘要翻译:本发明提供了包含对癌抗原gp100具有抗原特异性的鼠T细胞受体(TCR)的人细胞,特别是人T细胞。 具有对gp100(SEQ ID NO:1)的氨基酸154-162具有抗原特异性的分离或纯化的TCR,以及相关多肽,蛋白质,核酸,重组表达载体,宿主细胞,细胞群,抗体或抗原结合 其片段,缀合物和药物组合物。 本发明还提供了检测宿主中癌症存在的方法以及在宿主中治疗或预防癌症的方法,所述方法包括使用本文所述的本发明材料。
摘要:
The invention provides a chimeric T cell receptor (TCR) comprising a variable region of a human TCR and a constant region comprising at least an extracellular domain of a constant region of a non-human TCR, as well as functional variants thereof. The invention also provides polypeptides and proteins related to the inventive TCRs, as well as nucleic acids encoding the TCRs, polypeptides, or proteins, recombinant expression vectors, and host cells. Further provided are pharmaceutical compositions related to the inventive TCRs and methods of preventing or treating a disease, e.g., an infectious disease, cancer, in a host, methods of detecting a diseased cell in a host, and methods of improving the biological activity of a TCR.
摘要:
Compounds of Formula I, Formula II, and Formula III and the pharmaceutically acceptable salts thereof are disclosed. The variables R1-10 are disclosed herein. The compounds are useful for treating central nervous system disorders, especially those involving substance use disorder and/or withdrawal syndrome. Pharmaceutical compositions containing compounds of Formula I or Formula II or Formula III and methods of treatment comprising administering compounds of Formula I or Formula II or Formula III are also disclosed.