摘要:
The present description relates to an inhibitory RNA molecule, comprising an oligonucleotide that selectively knocks down expression a Nanog pseudogene expressed in many human cancers, a replicating viral vector capable of encoding such inhibitory RNA molecule, pharmaceutical compositions comprising said vector, and methods of treating cancer by administration of said pharmaceutical composition.
摘要:
The present invention provides isolated nucleic acid molecules encoding a Herpesviridae thymidine kinase enzyme comprising one or more mutations, at least one of the mutations encoding an amino acid substitution located toward the N-terminus from a DRH nucleoside binding site which increases a biological activity of the thymidine kinase, as compared to unmutated thymidine kinase. Such mutations include amino acid substitutions within a Q substrate binding domain which increases a biological activity of the thymidine kinase, as compared to unmutated thymidine kinase. Within a further aspect, fusion proteins are provided which have both guanylate kinase and thymidine kinase biological properties. Also provided are vectors suitable for expressing such DNA molecules, as well as methods for utilizing such vectors.
摘要:
A method of inducing transposition in a transgenic embryo, sperm and egg is described, comprising the steps of (a) generating a first adult transgenic organism comprising within its genome one or more copies of a transposon; (b) generating a second adult transgenic organism comprising within its genome one or more copies of a gene encoding a transposase cognate for the transposon and/or a sequence capable of regulating expression of the gene encoding the transposase; (c) crossing the first adult transgenic organism with the second transgenic adult organism to provide a progeny which comprises, in the genome of one or more of its cells, both (i) one or more copies of the transposon and (ii) a gene encoding a transposase cognate for the transposon, wherein the gene encoding the transposase is under the control of one or more inducible regulatory sequences which permit expression of the transposase, and (d) expressing the gene encoding the transposase in the embryo, sperm or egg to cause mobilization of the transposon within a portion of the tissues or cells of the progeny. Using the method, mobilization of a transposon can advantageously be induced at predetermined stages of development of an embryo, sperm or egg and the mutated gene of a single cell may be replicated in subsequent cell divisions, resulting in groups of cells which are essentially homogenous for the transposed gene.
摘要:
The present invention provides a method of preventing or reducing cellular proliferation. The method involves administering to cells a composition which increases the level of YY1 protein, in particular YY1 mRNA, in the cells.
摘要:
The invention relates to engineered Herpes simplex virus (HSV) particles that are targeted to one or more specific binding pair members, such as receptors. Also, recombinant vectors for producing such HSV particles are provided. By reducing the affinity of HSV for its natural receptor(s) and increasing the affinity for a selected receptor, the HSV particles of the invention are useful for targeting cells that express the selected receptor, which itself may be a product of genetic engineering. The ability to selectively target cells render the HSV particles. particularly useful in selectively diagnosing, treating, and imaging cells bearing the selected binding pair member, such as a receptor. The invention also provides for polynucleotide-based therapy to cells bearing the selected binding pair member such as a receptor.
摘要:
The invention concerns glycosylated proteins having human factor VIII activity. In a preferred embodiment, the protein is glycosylated with oligosaccharides that include an alpha-(2,6)-linked sialic acid and a bisecting GlcNAc linked to a core beta-mannose.
摘要:
Disclosed herein are methods and compositions for treatment of conditions requiring inhibition of angiogenesis. Such conditions include those characterized by neovascularization, such as retinopathies, macular degeneration and various malignancies.
摘要:
The invention includes therapeutic compositions and methods useful in the treatment of neurodegenerative diseases, such as those characterized by accumulation of extracellular plaques. Such neurodegenerative diseases include Alzheimer's disease. The compositions of the invention include HSVAβ/TtxFC, which can be used to deliver effective therapeutic benefits to a patient without inducing inflammation.
摘要:
Methods and compositions are provided for determining whether target, e.g., an endogenous or novel (non-endogenous), nucleic acid sequence of a neuronal cell is involved in sensory function. In practicing the subject methods, a neuronal cell selective vector including a modulating domain for a neuronal cell nucleic acid sequence, optionally having a domain encoding a directly detectable product, is administered to an animal. Sensory function in harvested neuronal cells is then evaluated to determine whether the target nucleic acid sequence is involved in sensory function. Also provided are compositions, kits, and systems for practicing the subject methods.
摘要:
This invention relates to a method of treating or preventing neuro-degenerative disorders and neuro-developmental disorders by altering the ratio of the amount of full-length TrkB polypeptide to the amount of truncated TrkB polypeptides in a neuron or by altering the ratio of the amount of full-length TrkC polypeptide to the amount of truncated TrkC polypeptides in a neuron.