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Bioreversible Phosphotriester Rnai-Inducing Molecules

详细技术说明: RNAi responses have great potential to treat human disease, especially cancer and viral infections. However, siRNAs are macromolecules with no bioavailability to enter cells and require a delivery agent. Here we developed RNAi-molecules delivered into cells by the TAT Peptide Transduction Domain (PTD) peptide. Although we have only performed cell culture experiments, the technology holds promise for eventual development of RNAi therapeutics.

*Principal Investigation: Name: Steven Dowdy
Department:

Name: Khirud Gogoi
Department:

Name: Bryan Meade
Department:

附加资料: Inventor: DOWDY, Steven F. | MEADE, Bryan R. | GOGOI, Khirud
Priority Number: WO2014031575A1
IPC Current: C07H002100 | C12N001511
Assignee Applicant: The Regents of the University of California
Title: POLYNUCLEOTIDES HAVING BIOREVERSIBLE GROUPS | POLYNUCLÉOTIDES POSSÉDANT DES GROUPES BIORÉVERSIBLES
Usefulness: POLYNUCLEOTIDES HAVING BIOREVERSIBLE GROUPS | POLYNUCLÉOTIDES POSSÉDANT DES GROUPES BIORÉVERSIBLES
Summary: The polynucleotide construct is useful: for reducing the expression of a protein in a cell via by reducing gene expression (claimed); and as therapeutics for treating disorders such as cancer (leukemia or myelodysplastic syndrome) in humans, and as diagnostics and tools for research. Test details are described but no results given.
Novelty: New polynucleotide construct comprising first component (e.g. peptide and therapeutic agent) linked to it through bioreversible group attached to internucleotide bridging group, used e.g. to reduce protein expression and treat cancer

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