公开(公告)号：US07553830B2

公开(公告)日：2009-06-30

申请号：US10966174

申请日：2005-04-14

Applicant: Leonid Beigelman ,  Jasenka Matulic-Adamic ,  Alexander Karpeisky ,  Peter Haeberli ,  David Sweedler ,  Mark Reynolds ,  Nilabh Chaudhary ,  John Min

Inventor： Leonid Beigelman ,  Jasenka Matulic-Adamic ,  Alexander Karpeisky ,  Peter Haeberli ,  David Sweedler ,  Mark Reynolds ,  Nilabh Chaudhary ,  John Min

IPC: A61K31/26 ,  C07J9/00

CPC classification number: C07D213/64 ,  A61K47/54 ,  A61K47/542 ,  A61K47/543 ,  C07C237/06 ,  C07C237/22 ,  C07C279/04 ,  C07C279/12 ,  C07C279/14 ,  C07H19/06 ,  C07J1/00 ,  C07J41/0005 ,  C07J43/003 ,  C07J51/00 ,  C12N15/88

Abstract: This invention features permeability enhancer molecules, and methods, to increase membrane permeability of various molecules, such as nucleic acids, polynucleotides, oligonucleotides, enzymatic nucleic acid molecules, antisense nucleic acid molecules, 2-5A antisense chimeras, triplex forming oligonucleotides, decoy RNAS, dsRNAs, siRNAs, aptamers, or antisense nucleic acids containing nucleic acid cleaving chemical groups, peptides, polypeptides, proteins, carbohydrates, steroids, metals and small molecules, thereby facilitating cellular uptake of such molecules.

Abstract translation: 本发明的特征在于渗透性增强剂分子和方法，以提高各种分子如核酸，多核苷酸，寡核苷酸，酶核酸分子，反义核酸分子，2-5A反义嵌合体，形成三链体的寡核苷酸，诱饵RNAS， dsRNA，siRNA，适体或含有核酸切割化学基团，肽，多肽，蛋白质，碳水化合物，类固醇，金属和小分子的反义核酸，从而促进这些分子的细胞摄取。

公开(公告)号：US20100036115A1

公开(公告)日：2010-02-11

申请号：US12470382

申请日：2009-05-21

Applicant: Leonid Beigelman ,  Nilabh Chaudhary ,  Peter Haeberli ,  Alexander Karpeisky ,  Jasenka Matulic-Adamic ,  John Min ,  Mark Reynolds ,  David Sweedler

Inventor： Leonid Beigelman ,  Nilabh Chaudhary ,  Peter Haeberli ,  Alexander Karpeisky ,  Jasenka Matulic-Adamic ,  John Min ,  Mark Reynolds ,  David Sweedler

IPC: C07J43/00

CPC classification number: C07D213/64 ,  A61K47/54 ,  A61K47/542 ,  A61K47/543 ,  C07C237/06 ,  C07C237/22 ,  C07C279/04 ,  C07C279/12 ,  C07C279/14 ,  C07H19/06 ,  C07J1/00 ,  C07J41/0005 ,  C07J43/003 ,  C07J51/00 ,  C12N15/88

Abstract: This invention features permeability enhancer molecules, and methods, to increase membrane permeability of various molecules, such as nucleic acids, polynucleotides, oligonucleotides, enzymatic nucleic acid molecules, antisense nucleic acid molecules, 2-5A antisense chimeras, triplex forming oligonucleotides, decoy RNAs, dsRNAs, siRNAs, aptamers, or antisense nucleic acids containing nucleic acid cleaving chemical groups, peptides, polypeptides, proteins, carbohydrates, steroids, metals and small molecules, thereby facilitating cellular uptake of such molecules.

Abstract translation: 本发明的特征在于渗透性增强剂分子和方法，以增加各种分子如核酸，多核苷酸，寡核苷酸，酶核酸分子，反义核酸分子，2-5A反义嵌合体，形成三链体的寡核苷酸，诱饵RNA， dsRNA，siRNA，适体或含有核酸切割化学基团，肽，多肽，蛋白质，碳水化合物，类固醇，金属和小分子的反义核酸，从而促进这些分子的细胞摄取。

公开(公告)号：US5614503A

公开(公告)日：1997-03-25

申请号：US467114

申请日：1995-06-06

Applicant: Nilabh Chaudhary ,  Krishna Jayaraman ,  Veeraiah Bodepudi ,  Michael E. Hogan

Inventor： Nilabh Chaudhary ,  Krishna Jayaraman ,  Veeraiah Bodepudi ,  Michael E. Hogan

IPC: A61K9/127 ,  A61K48/00 ,  C07J41/00 ,  C07C261/00 ,  C07J9/00

CPC classification number: C07J41/0055 ,  A61K48/00 ,  A61K9/1272

Abstract: A nucleic acid transporter to deliver a nucleic acids into cells, comprising a cationic compound having a cationic head group for binding the nucleic acid and a lipid tail for association with the membrane. A cationic compound usually is a polyamine or a short basic peptide. The lipid tail is usually selected from the group consisting of plant steroid, animal steroid, isoprenoid compound, aliphatic lipid, pore forming protein, pore forming peptides and fusogenic peptides. The cationic head and the lipid tail are linked through a carbamate linkage. When polyamine is used, it is preferably either spermidine or spermine and the nucleic acid can be any of a variety, including triplex forming oligonucleotides, antisense oligonucleotide, aptamers, ribozymes, plasmids and DNA for gene therapy. Also described is a method for treating individuals using the transporter linked to a therapeutic nucleic acid.

Abstract translation: 将核酸递送到细胞中的核酸转运蛋白，其包含具有用于结合核酸的阳离子头基的阳离子化合物和与所述膜缔合的脂质尾。 阳离子化合物通常是多胺或短碱性肽。 脂质尾巴通常选自植物类固醇，动物类固醇，类异戊二烯化合物，脂肪族脂质，成孔蛋白，成孔肽和融合肽。 阳离子头和脂质尾巴通过氨基甲酸酯键连接。 当使用多胺时，其优选为亚精胺或精胺，核酸可为任何多种，包括形成三链体的寡核苷酸，反义寡核苷酸，适体，核酶，质粒和用于基因治疗的DNA。 还描述了使用与治疗性核酸连接的转运蛋白来治疗个体的方法。

公开(公告)号：US20050234232A1

公开(公告)日：2005-10-20

申请号：US10966174

申请日：2005-04-14

Applicant: Leonid Beigelman ,  Jasenka Matulic-Adamic ,  Alexander Karpeisky ,  Peter Haeberli ,  David Sweedler ,  Mark Reynolds ,  Nilabh Chaudhary ,  John Min

Inventor： Leonid Beigelman ,  Jasenka Matulic-Adamic ,  Alexander Karpeisky ,  Peter Haeberli ,  David Sweedler ,  Mark Reynolds ,  Nilabh Chaudhary ,  John Min

IPC: A61K47/48 ,  C07C237/06 ,  C07C237/22 ,  C07C279/04 ,  C07C279/12 ,  C07C279/14 ,  C07D213/64 ,  C07H19/06 ,  C07J1/00 ,  C07J41/00 ,  C07J43/00 ,  C07J51/00 ,  C12N15/88

CPC classification number: C07D213/64 ,  A61K47/54 ,  A61K47/542 ,  A61K47/543 ,  C07C237/06 ,  C07C237/22 ,  C07C279/04 ,  C07C279/12 ,  C07C279/14 ,  C07H19/06 ,  C07J1/00 ,  C07J41/0005 ,  C07J43/003 ,  C07J51/00 ,  C12N15/88

Abstract: This invention features permeability enhancer molecules, and methods, to increase membrane permeability of various molecules, such as nucleic acids, polynucleotides, oligonucleotides, enzymatic nucleic acid molecules, antisense nucleic acid molecules, 2-5A antisense chimeras, triplex forming oligonucleotides, decoy RNAS, dsRNAs, siRNAs, aptamers, or antisense nucleic acids containing nucleic acid cleaving chemical groups, peptides, polypeptides, proteins, carbohydrates, steroids, metals and small molecules, thereby facilitating cellular uptake of such molecules.

Abstract translation: 本发明的特征在于渗透性增强剂分子和方法，以提高各种分子如核酸，多核苷酸，寡核苷酸，酶核酸分子，反义核酸分子，2-5A反义嵌合体，形成三链体的寡核苷酸，诱饵RNAS， dsRNA，siRNA，适体或含有核酸切割化学基团，肽，多肽，蛋白质，碳水化合物，类固醇，金属和小分子的反义核酸，从而促进这些分子的细胞摄取。

公开(公告)号：US06395713B1

公开(公告)日：2002-05-28

申请号：US09120520

申请日：1998-07-21

Applicant: Leonid Beigelman ,  Jasenka Matulic-Adamic ,  Alex Karpeisky ,  Peter Haeberli ,  David Sweedler ,  Mark Reynolds ,  Nilabh Chaudhary ,  John Min

Inventor： Leonid Beigelman ,  Jasenka Matulic-Adamic ,  Alex Karpeisky ,  Peter Haeberli ,  David Sweedler ,  Mark Reynolds ,  Nilabh Chaudhary ,  John Min

IPC: A01N4304

CPC classification number: C07D213/64 ,  A61K47/54 ,  A61K47/542 ,  A61K47/543 ,  C07C237/06 ,  C07C237/22 ,  C07C279/04 ,  C07C279/12 ,  C07C279/14 ,  C07H19/06 ,  C07J41/0005 ,  C07J43/003 ,  C07J51/00 ,  C12N15/88

Abstract: This invention features permeability enhancer molecules, and methods, to increase membrane permeability of negatively charged polymers thereby facilitating cellular uptake of such polymers.

Abstract translation: 本发明的特征在于渗透性增强剂分子和方法，以提高带负电荷的聚合物的膜渗透性，从而促进这些聚合物的细胞摄取。