摘要:
The present invention provides methods for the chemical synthesis of nucleosides and derivatives thereof, including 2'-amino, 2'-N-phthaloyl, 2'-O-methyl, 2'-O-silyl, 2'-OH nucleosides, C-nucleosides, nucleoside phosphoramidites, C-nucleoside phosphoramidites, and non-nucleoside derivatives.
摘要:
Enzymatic RNA molecules which cleave ICAM-1 mRNA, IL-5 mRNA, rel A mRNA, TNF-α mRNA, RSV mRNA or RSV genomic RNA, or CML associated mRNA, and use of these molecules for the treatment of pathological conditions related to those mRNA-levels; ribonucleosides or nucleotides modified in 2', 3' or 5', methods for their synthesis, purification and deprotection; vectors containing multiple enzymatic nucleic acids, optionally in chimeric form with tRNAs; method for introducing enzymatic nucleic acids into cells by forming a complex with a second nucleic acid, where the complex is capable of taking an R-loop base-paired structure; method for altering a mutant nucleic acid in vivo by hybridization with an oligonucleotide capable of activating dsRNA deaminase, comprising an enzymatic activity or a chemical mutagen. Further are disclosed trans-cleaving or -ligating hairpin ribozymes lacking a substrate RNA moiety, as well as hammerhead ribozymes having an interconnecting loop between base pairs in stem II.
摘要:
The present invention provides methods for the chemical synthesis of nucleosides and derivatives thereof, including 2'-amino, 2'-N-phthaloyl, 2'-O-methyl, 2'-O-silyl, 2'-OH nucleosides, C-nucleosides, nucleoside phosphoramidites, C-nucleoside phosphoramidites, and non-nucleoside derivatives.
摘要:
This invention features permeability enhancer molecules, and methods, to increase membrane permeability of negatively charged polymers thereby facilitating cellular uptake of such polymers.
摘要:
Nucleic acid molecules (antisenses or ribozymes) useful as inhibitors of gene expression, especially of HER2, BACE, TERT, PTP-1B, MetAP-2, HBV, phospholamban, presenilin-2 and PKC-alpha. The nucleic acid molecules can be modified in various ways on the sugar and/or base moieties and/or on the phosphate backbone. They are used in pharmaceutical formulations for the treatment of diseases involving increased expression of the target genes. Also disclosed is a method for the synthesis of a modified pyrimidine nucleotide triphosphate and its incorporation into an oligonucleotide.
摘要:
Novel nucleotide triphosphates, methods of synthesis and process of incorporating these nucleotide triphosphates into oligonucleotides, and isolation of novel nucleic acid catalysts (e.g., ribozymes) are disclosed.
摘要:
Nucleic acid catalysts which modulate the expression of Raf gene; method of delivery, screening, identification, synthesis, deprotection, purification, of nucleic acid catalysts and processes for identification of nucleic acid molecules is described.