公开(公告)号：US20160090603A1

公开(公告)日：2016-03-31

申请号：US14871748

申请日：2015-09-30

Applicant: Sandia Corporation

Inventor： Eric Christopher Carnes ,  Anne Ruffing ,  Carlee Erin Ashley

IPC: C12N15/82

CPC classification number: A61K9/1274 ,  A61K9/501 ,  A61K9/5115 ,  A61K9/5123 ,  A61K31/7088 ,  A61K45/06 ,  A61K48/005 ,  A61K49/005 ,  C12N9/22 ,  C12N15/8206 ,  C12N2310/11 ,  C12N2310/20

Abstract: The present invention relates to a delivery platform that can be used to genetically modify a target in a plant or an alga. In one instance, polypeptides and/or polynucleotides can be delivered using silica delivery platforms, e.g., silica carriers or protocells. Such platforms can be employed to control gene activation and repression in the plant or alga.

Abstract translation: 本发明涉及可用于在植物或藻类中遗传修饰靶标的递送平台。 在一个实例中，多肽和/或多核苷酸可以使用二氧化硅递送平台例如二氧化硅载体或原细胞递送。 这样的平台可用于控制植物或藻类中的基因激活和抑制。

公开(公告)号：US20150272885A1

公开(公告)日：2015-10-01

申请号：US14350674

申请日：2012-10-12

Applicant: STC.UNM ,  SANDIA CORPORATION

Inventor： Carlee Erin Ashley ,  C. Jeffrey Brinker ,  Eric C. Carnes ,  Mohammad Houman Fekrazad ,  Linda A. Felton ,  Oscar Negrete ,  David Patrick Padilla ,  Brian S. Wilkinson ,  Dan C. Wilkinson ,  Cheryl L. Willman

IPC: A61K9/127 ,  A61K45/06 ,  A61K31/704 ,  A61K31/513 ,  A61K31/713 ,  A61K38/17

CPC classification number: A61K48/0008 ,  A61K9/0014 ,  A61K9/107 ,  A61K9/1271 ,  A61K9/5078 ,  A61K31/192 ,  A61K31/465 ,  A61K31/506 ,  A61K31/513 ,  A61K31/704 ,  A61K31/7088 ,  A61K31/7105 ,  A61K31/713 ,  A61K33/24 ,  A61K38/00 ,  A61K38/17 ,  A61K38/45 ,  A61K38/47 ,  A61K45/06 ,  A61K47/6923 ,  A61K49/0082 ,  A61K49/0423 ,  B82Y5/00 ,  C07K7/06 ,  C07K2319/00 ,  C12N15/113 ,  C12N15/1131 ,  C12N15/88 ,  C12N2310/14 ,  C12N2320/32 ,  C12N2810/40 ,  C12Y204/02036 ,  C12Y302/02022 ,  A61K2300/00

Abstract: The present invention is directed to protocells for specific targeting of hepatocellular and other cancer cells which comprise a nanoporous silica core with a supported lipid bilayer; at least one agent which facilitates cancer cell death (such as a traditional small molecule, a macromolecular cargo (e.g. siRNA or a protein toxin such as ricin toxin A-chain or diphtheria toxin A-chain) and/or a histone-packaged plasmid DNA disposed within the nanoporous silica core (preferably supercoiled in order to more efficiently package the DNA into protocells) which is optionally modified with a nuclear localization sequence to assist in localizing protocells within the nucleus of the cancer cell and the ability to express peptides involved in therapy (apoptosis/cell death) of the cancer cell or as a reporter, a targeting peptide which targets cancer cells in tissue to be treated such that binding of the protocell to the targeted cells is specific and enhanced and a fusogenic peptide that promotes endosomal escape of protocells and encapsulated DNA. Protocells according to the present invention may be used to treat cancer, especially including hepatocellular (liver) cancer using novel binding peptides (c-MET peptides) which selectively bind to hepatocellular tissue or to function in diagnosis of cancer, including cancer treatment and drug discovery.

Abstract translation: 本发明涉及用于特异性靶向肝细胞和其它癌细胞的原细胞，其包含具有支持的脂质双层的纳米多孔二氧化硅核心; 至少一种促进癌细胞死亡的药物（例如传统的小分子，大分子货物（例如siRNA或蛋白质毒素如蓖麻毒素A链或白喉毒素A链）和/或组蛋白包装的质粒DNA 设置在纳米多孔硅芯内（优选超螺旋以更有效地将DNA包装到原细胞中），其任选地用核定位序列修饰以帮助定位癌细胞核内的原细胞，以及表达参与治疗的肽的能力 （凋亡/细胞死亡）或作为报告物，靶向肽靶向待治疗的组织中的癌细胞，使得原细胞与靶细胞的结合是特异性和增强的，并且促进内体逃逸的融合肽 原细胞和包封的DNA。根据本发明的原细胞可用于治疗癌症，特别是包括肝细胞（肝）癌症 选择性结合肝细胞组织或在癌症诊断中起作用的新型结合肽（c-MET肽），包括癌症治疗和药物发现。