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PLASMID DELIVERY IN THE TREATMENT OF CANCER AND OTHER DISEASE STATES
专利权人:
STC.UNM
发明人:
BRINKER, C., Jeffrey,CARNES, Eric, Christopher,ASHLEY, Carlee, Erin,SANTARPIA, Joshua,GREENE, Adrienne, Celeste,NEGRETE, Oscar,BRANDA, Steven,MUNIZ, Ayse
申请号:
USUS2015/053244
公开号:
WO2016/054225A1
申请日:
2015.09.30
申请国别(地区):
WO
年份:
2016
代理人:
摘要:
The present invention relates to the delivery of polynucleotides and/or oligonucleotides using silica delivery platforms, e.g., silica carriers or protocells. In particular, in the present invention, polynucleotides in the form of plasmids expressing siRNA may be administered as cargo in the silica delivery platform to a patient or subject to inhibit and/or treat cancer in a patient. In one aspect, the silica delivery platform that have been charged with cargo comprising plasmid DNA (in particular, CRISPR ds plasmid DNA) which expresses siRNA, shRNA, mRNA and other RNA which may be used to administer these plasmids to patients in order to effect inhibition of cancer cells (especially including apoptosis of those cancer cells) and effective and/or prophylaxis of cancer, as well as numerous pathogens, including viruses, bacteria, fungi, and/or other disease states and/or conditions. In another aspect, the silica delivery platform comprises a biological package (e.g., plasmid nucleic acid, such as a for a CRISPR/Cas system) that interacts with a genomic sequence to either activate or inhibit gene expression. Such vehicles can be employed to control gene activation and repression in a host (e.g., a patient) and/or a pathogen.La présente invention concerne ladministration de polynucléotides et/ou doligonucléotides à laide de plates-formes de distribution de silice, par exemple des porteurs de silice ou des protocellules. En particulier, dans la présente invention, des polynucléotides sous la forme de plasmides exprimant ARNic peuvent être administrés sous la forme de cargaison dans la plate-forme de distribution de silice à un patient ou à un sujet pour inhiber et/ou traiter un cancer chez un patient. Selon un premier aspect, la plate-forme de distribution de silice qui a été chargée dune cargaison comportant un ADN de plasmide (en particulier un ADN de plasmide ds CRISPR) qui exprime ARNic, ARNsh, ARNm et dautres ARN, peut être utilisée pour administrer ces plasmides à
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