公开(公告)号：WO2013056132A3

公开(公告)日：2013-06-13

申请号：PCT/US2012060072

申请日：2012-10-12

Applicant: STC UNM ,  SANDIA CORP

Inventor： ASHLEY CARLEE ERIN ,  BRINKER C JEFFREY ,  CARNES ERIC C ,  FEKRAZAD MOHAMMAD HOUMAN ,  FELTON LINDA A ,  NEGRETE OSCAR ,  PADILLA DAVID PATRICK ,  WILKINSON BRIAN S ,  WILKINSON DAN C ,  WILLMAN CHERYL L

IPC: A61K9/16 ,  A61K31/7088 ,  A61K31/7105 ,  A61K38/17 ,  A61K47/48 ,  A61K48/00 ,  A61K49/08

CPC classification number: A61K47/48823 ,  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 ,  A61K47/483 ,  A61K47/48561 ,  A61K47/48815 ,  A61K47/48861 ,  A61K47/6923 ,  A61K48/0008 ,  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，并且促进内源性逃逸的融合肽 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。

公开(公告)号：WO2010056351A3

公开(公告)日：2010-11-18

申请号：PCT/US2009006117

申请日：2009-11-16

Applicant: STC UNM ,  WILLMAN CHERYL L ,  HARVEY RICHARD ,  KANG HUINING ,  BEDRICK EDWARD ,  WANG XUEFEI ,  ATLAS SUSAN ,  CHEN I-MING

Inventor： WILLMAN CHERYL L ,  HARVEY RICHARD ,  KANG HUINING ,  BEDRICK EDWARD ,  WANG XUEFEI ,  ATLAS SUSAN ,  CHEN I-MING

IPC: C12Q1/68 ,  C12N15/12

CPC classification number: C12Q1/6886 ,  C12Q2600/106 ,  C12Q2600/118 ,  C12Q2600/136 ,  C12Q2600/158

Abstract: The present invention relates to the identification of genetic markers patients with leukemia, especially including acute lymphoblastic leukemia (ALL) at high risk for relapse, especially high risk B-precursor acute lymphoblastic leukemia (B-ALL) and associated methods and their relationship to therapeutic outcome. The present invention also relates to diagnostic, prognostic and related methods using these genetic markers, as well as kits which provide microchips and/or immunoreagents for performing analysis on leukemia patients.

Abstract translation: 本发明涉及具有高度复发风险的急性淋巴细胞白血病（ALL）特别是高风险B-前体急性淋巴细胞白血病（B-ALL）的白血病遗传标记患者的鉴定及相关方法及其与治疗剂的关系 结果。 本发明还涉及使用这些遗传标记的诊断，预后和相关方法，以及提供用于对白血病患者进行分析的微芯片和/或免疫试剂的试剂盒。

公开(公告)号：WO2004053074A3

公开(公告)日：2006-01-19

申请号：PCT/US0338738

申请日：2003-12-05

Applicant: SCIENCE AND TECHNOLOGY CORP UN ,  SANDIA CORP ,  WILLMAN CHERYL L ,  HELMAN PAUL ,  VEROFF ROBERT ,  MOSQUERA-CARO MONICA ,  DAVIDSON GEORGE S ,  MARTIN SHAWN B ,  ATLAS SUSAN R ,  ANDRIES ERIK ,  KANG HUINING ,  SHUSTER JONATHAN J ,  HAALAND DAVID M ,  POTTER JEFFREY W ,  WANG XUEFEI ,  HARVEY RICHARD

Inventor： WILLMAN CHERYL L ,  HELMAN PAUL ,  VEROFF ROBERT ,  MOSQUERA-CARO MONICA ,  DAVIDSON GEORGE S ,  MARTIN SHAWN B ,  ATLAS SUSAN R ,  ANDRIES ERIK ,  KANG HUINING ,  SHUSTER JONATHAN J ,  HAALAND DAVID M ,  POTTER JEFFREY W ,  WANG XUEFEI ,  HARVEY RICHARD

IPC: C12N20060101 ,  C12Q1/68 ,  C07H21/02 ,  A61K48/00 ,  C07H21/04 ,  C07K14/00 ,  G01N33/53

CPC classification number: C12Q1/6886 ,  C12Q2600/106 ,  C12Q2600/112 ,  C12Q2600/136

Abstract: Genes and gene expression profiles useful for predicting outcome, risk classification, cytogenetics and/or etiology in pediatric acute lymphoblastic leukemia (ALL). OPAL 1 is a novel gene associated with outcome and, along with other newly identified genes, represent a novel therapeutic targets.

Abstract translation: 基因和基因表达谱可用于预测儿科急性淋巴细胞白血病（ALL）的结局，风险分类，细胞遗传学和/或病因。 OPAL 1是与结果相关的新基因，并与其他新鉴定的基因一起代表了一种新的治疗靶点。