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公开(公告)号:US20110178157A1
公开(公告)日:2011-07-21
申请号:US12963600
申请日:2010-12-08
申请人: Xiaomei Jin , Lei Yu , Hirokazu Takahashi , Yasunobu Tanaka , Yoshiro Niitsu , Elena Feinstein , Sharon Avkin-Nachum , Hagar Kalinski , Igor Mett , Shai Erlich , Elizabeth C. Squiers
发明人: Xiaomei Jin , Lei Yu , Hirokazu Takahashi , Yasunobu Tanaka , Yoshiro Niitsu , Elena Feinstein , Sharon Avkin-Nachum , Hagar Kalinski , Igor Mett , Shai Erlich , Elizabeth C. Squiers
IPC分类号: A61K31/713 , C07H21/02 , A61P43/00 , C07H21/00
CPC分类号: C12N15/113 , A61K31/713 , C12N2310/11 , C12N2310/14 , C12N2310/31 , C12N2310/315 , C12N2310/317 , C12N2310/32 , C12N2310/321 , C12N2310/33 , C12N2310/531 , C12N2320/30 , C12N2310/3521
摘要: Provided herein are compositions, methods and kits for modulating expression of target genes, particularly heat shock protein 47 (hsp47). The compositions, methods and kits may include nucleic acid molecules (for example, short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA) or short hairpin RNA (shRNA)) that modulate a gene encoding hsp47, for example, the gene encoding human hsp47. The composition and methods disclosed herein may also be used in treating conditions and disorders associated with hsp47 such as liver fibrosis, pulmonary fibrosis, peritoneal fibrosis and kidney fibrosis.
摘要翻译: 本文提供用于调节靶基因,特别是热休克蛋白47(hsp47)的表达的组合物,方法和试剂盒。 组合物,方法和试剂盒可以包括核酸分子(例如,短干扰核酸(siNA),短干扰RNA(siRNA),双链RNA(dsRNA),微RNA(miRNA)或短发夹RNA(shRNA) )),其调节编码hsp47的基因,例如编码人hsp47的基因。 本文公开的组合物和方法也可用于治疗与hsp47相关的病症和病症,例如肝纤维化,肺纤维化,腹膜纤维化和肾纤维化。
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公开(公告)号:US08710209B2
公开(公告)日:2014-04-29
申请号:US12963600
申请日:2010-12-08
申请人: Xiaomei Jin , Lei Yu , Hirokazu Takahashi , Yasunobu Tanaka , Yoshiro Niitsu , Elena Feinstein , Sharon Avkin-Nachum , Hagar Kalinski , Igor Mett , Shai Erlich , Elizabeth C. Squiers , Ning Chen
发明人: Xiaomei Jin , Lei Yu , Hirokazu Takahashi , Yasunobu Tanaka , Yoshiro Niitsu , Elena Feinstein , Sharon Avkin-Nachum , Hagar Kalinski , Igor Mett , Shai Erlich , Elizabeth C. Squiers , Ning Chen
IPC分类号: C07H21/04
CPC分类号: C12N15/113 , A61K31/713 , C12N2310/11 , C12N2310/14 , C12N2310/31 , C12N2310/315 , C12N2310/317 , C12N2310/32 , C12N2310/321 , C12N2310/33 , C12N2310/531 , C12N2320/30 , C12N2310/3521
摘要: Provided herein are compositions, methods and kits for modulating expression of target genes, particularly heat shock protein 47 (hsp47). The compositions, methods and kits may include nucleic acid molecules (for example, short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA) or short hairpin RNA (shRNA)) that modulate a gene encoding hsp47, for example, the gene encoding human hsp47. The composition and methods disclosed herein may also be used in treating conditions and disorders associated with hsp47 such as liver fibrosis, pulmonary fibrosis, peritoneal fibrosis and kidney fibrosis.
摘要翻译: 本文提供用于调节靶基因,特别是热休克蛋白47(hsp47)的表达的组合物,方法和试剂盒。 组合物,方法和试剂盒可以包括核酸分子(例如,短干扰核酸(siNA),短干扰RNA(siRNA),双链RNA(dsRNA),微RNA(miRNA)或短发夹RNA(shRNA) )),其调节编码hsp47的基因,例如编码人hsp47的基因。 本文公开的组合物和方法也可用于治疗与hsp47相关的病症和病症,例如肝纤维化,肺纤维化,腹膜纤维化和肾纤维化。
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公开(公告)号:US20130030034A9
公开(公告)日:2013-01-31
申请号:US13246621
申请日:2011-09-27
申请人: Yoshiro Niitsu , Hirokazu Takahashi , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nachum , Hagar Kalinski , Igor Mett
发明人: Yoshiro Niitsu , Hirokazu Takahashi , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nachum , Hagar Kalinski , Igor Mett
IPC分类号: A61K31/7088 , A61P43/00 , C07H21/02
CPC分类号: C12N15/113 , A61K9/0019 , A61K31/713 , C07K14/8146 , C12N2310/14 , C12N2310/3519 , C12N2320/30 , C12N2320/32
摘要: Provided herein are compositions, methods and kits for modulating expression of target genes, particularly of tissue inhibitor of metalloproteinase 1 and of tissue inhibitor of metalloproteinase 2 (TIMP1 and TIMP2, respectively). The compositions, methods and kits may include nucleic acid molecules (for example, short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA) or short hairpin RNA (shRNA)) that modulate a gene encoding TIMP1 and TIMP2, for example, the gene encoding human TIMP1 and TIMP2. The composition and methods disclosed herein may also be used in treating conditions and disorders associated with TIMP1 and TIMP2 including fibrotic diseases and disorders including liver fibrosis, pulmonary fibrosis, peritoneal fibrosis and kidney fibrosis.
摘要翻译: 本文提供用于调节靶基因,特别是金属蛋白酶1的组织抑制剂和金属蛋白酶2的组织抑制剂(分别为TIMP1和TIMP2)的表达的组合物,方法和试剂盒。 组合物,方法和试剂盒可以包括核酸分子(例如,短干扰核酸(siNA),短干扰RNA(siRNA),双链RNA(dsRNA),微RNA(miRNA)或短发夹RNA(shRNA) )),其调节编码TIMP1和TIMP2的基因,例如编码人TIMP1和TIMP2的基因。 本文公开的组合物和方法也可用于治疗与TIMP1和TIMP2相关的病症和病症,包括纤维化疾病和病症,包括肝纤维化,肺纤维化,腹膜纤维化和肾纤维化。
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公开(公告)号:US20120142754A1
公开(公告)日:2012-06-07
申请号:US13246621
申请日:2011-09-27
申请人: Yoshiro Niitsu , Hirokazu Takahashi , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nachum , Hagar Kalinski , Igor Mett
发明人: Yoshiro Niitsu , Hirokazu Takahashi , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nachum , Hagar Kalinski , Igor Mett
IPC分类号: A61K31/7088 , A61P43/00 , C07H21/02
CPC分类号: C12N15/113 , A61K9/0019 , A61K31/713 , C07K14/8146 , C12N2310/14 , C12N2310/3519 , C12N2320/30 , C12N2320/32
摘要: Provided herein are compositions, methods and kits for modulating expression of target genes, particularly of tissue inhibitor of metalloproteinase 1 and of tissue inhibitor of metalloproteinase 2 (TIMP1 and TIMP2, respectively). The compositions, methods and kits may include nucleic acid molecules (for example, short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA) or short hairpin RNA (shRNA)) that modulate a gene encoding TIMP1 and TIMP2, for example, the gene encoding human TIMP1 and TIMP2. The composition and methods disclosed herein may also be used in treating conditions and disorders associated with TIMP1 and TIMP2 including fibrotic diseases and disorders including liver fibrosis, pulmonary fibrosis, peritoneal fibrosis and kidney fibrosis.
摘要翻译: 本文提供用于调节靶基因,特别是金属蛋白酶1的组织抑制剂和金属蛋白酶2的组织抑制剂(分别为TIMP1和TIMP2)的表达的组合物,方法和试剂盒。 组合物,方法和试剂盒可以包括核酸分子(例如,短干扰核酸(siNA),短干扰RNA(siRNA),双链RNA(dsRNA),微RNA(miRNA)或短发夹RNA(shRNA) )),其调节编码TIMP1和TIMP2的基因,例如编码人TIMP1和TIMP2的基因。 本文公开的组合物和方法也可用于治疗与TIMP1和TIMP2相关的病症和病症,包括纤维化疾病和病症,包括肝纤维化,肺纤维化,腹膜纤维化和肾纤维化。
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公开(公告)号:US08741867B2
公开(公告)日:2014-06-03
申请号:US13492594
申请日:2012-06-08
申请人: Yoshiro Niitsu , Victor Knopov , Joseph E. Payne , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Violetta Akopian , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nahum , Hagar Kalinski , Igor Mett , Kenjiro Minomi , Wenbin Ying , Yun Liu
发明人: Yoshiro Niitsu , Victor Knopov , Joseph E. Payne , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Violetta Akopian , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nahum , Hagar Kalinski , Igor Mett , Kenjiro Minomi , Wenbin Ying , Yun Liu
IPC分类号: C12N15/11
CPC分类号: A61K9/1271 , A61K9/127 , A61K31/713 , C07H21/02 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2320/32 , C12N2320/52
摘要: What is described are pharmaceutical compositions comprising a double-stranded nucleic acid molecule comprising a sense strand and an antisense strand wherein the sense and antisense strands are selected from the oligonucleotides described as SERPINH1_2 (SEQ ID NOS: 60 and 127), SERPINH1_45a (SEQ ID NOS: 98 and 165), and SERPINH1_51 (SEQ ID NOS: 101 and 168), and drug carrier comprising a mixture of a retinoid and a lipid vesicle, and methods of using these pharmaceutical compositions to treat a disease associated with hsp47 expression, including fibrosis.
摘要翻译: 描述的是包含包含有义链和反义链的双链核酸分子的药物组合物,其中有义链和反义链选自描述为SERPINH1_2的寡核苷酸(SEQ ID NO:60和127),SERPINH1_45a(SEQ ID NO: NOS:98和165)和SERPINH1_51(SEQ ID NO:101和168)和包含类视黄醇和脂质囊泡的混合物的药物载体,以及使用这些药物组合物治疗与hsp47表达相关的疾病的方法,包括 纤维化。
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6.
公开(公告)号:US20130071478A1
公开(公告)日:2013-03-21
申请号:US13492639
申请日:2012-06-08
申请人: Yoshiro Niitsu , Victor Knopov , Joseph E. Payne , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Violetta Akopian , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nahum , Hagar Kalinski , Igor Mett , Kenjiro Minomi , Wenbin Ying , Yun Liu
发明人: Yoshiro Niitsu , Victor Knopov , Joseph E. Payne , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Violetta Akopian , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nahum , Hagar Kalinski , Igor Mett , Kenjiro Minomi , Wenbin Ying , Yun Liu
IPC分类号: C07H21/02
CPC分类号: A61K9/1271 , A61K9/127 , A61K31/713 , C07H21/02 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2320/32 , C12N2320/52
摘要: What is described are pharmaceutical compositions comprising a double-stranded nucleic acid molecule comprising a sense strand and an antisense strand wherein the sense and antisense strands are selected from the oligonucleotides described as SERPINH1—2 (SEQ ID NOS: 60 and 127), SERPINH1—45a (SEQ ID NOS: 98 and 165), and SERPINH1—51 (SEQ ID NOS: 101 and 168), and drug carrier comprising a mixture of a retinoid and a lipid vesicle, and methods of using these pharmaceutical compositions to treat a disease associated with hsp47 espresssion, including fibrosis.
摘要翻译: 所描述的是包含包含有义链和反义链的双链核酸分子的药物组合物,其中有义链和反义链选自描述为SERPINH1-2(SEQ ID NO:60和127)的寡核苷酸,SERPINH1- 45a(SEQ ID NO:98和165)和SERPINH1-51(SEQ ID NO:101和168)和包含类视黄醇和脂质囊泡的混合物的药物载体,以及使用这些药物组合物治疗疾病的方法 与hsp47浓缩相关,包括纤维化。
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公开(公告)号:US20130071467A1
公开(公告)日:2013-03-21
申请号:US13492594
申请日:2012-06-08
申请人: Yoshiro Niitsu , Victor Knopov , Joseph E. Payne , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Violetta Akopian , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nahum , Hagar Kalinski , Igor Mett , Kenjiro Minomi , Wenbin Ying , Yun Liu
发明人: Yoshiro Niitsu , Victor Knopov , Joseph E. Payne , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Violetta Akopian , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nahum , Hagar Kalinski , Igor Mett , Kenjiro Minomi , Wenbin Ying , Yun Liu
IPC分类号: A61K9/127 , A61K31/713
CPC分类号: A61K9/1271 , A61K9/127 , A61K31/713 , C07H21/02 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2320/32 , C12N2320/52
摘要: What is described are pharmaceutical compositions comprising a double-stranded nucleic acid molecule comprising a sense strand and an antisense strand wherein the sense and antisense strands are selected from the oligonucleotides described as SERPINH1_2 (SEQ ID NOS: 60 and 127), SERPINH1_45a (SEQ ID NOS: 98 and 165), and SERPINH1_51 (SEQ ID NOS: 101 and 168), and drug carrier comprising a mixture of a retinoid and a lipid vesicle, and methods of using these pharmaceutical compositions to treat a disease associated with hsp47 expression, including fibrosis.
摘要翻译: 描述的是包含包含有义链和反义链的双链核酸分子的药物组合物,其中有义链和反义链选自描述为SERPINH1_2的寡核苷酸(SEQ ID NO:60和127),SERPINH1_45a(SEQ ID NO: NOS:98和165)和SERPINH1_51(SEQ ID NO:101和168)和包含类视黄醇和脂质囊泡的混合物的药物载体,以及使用这些药物组合物治疗与hsp47表达相关的疾病的方法,包括 纤维化。
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8.
公开(公告)号:US08664376B2
公开(公告)日:2014-03-04
申请号:US13492639
申请日:2012-06-08
申请人: Yoshiro Niitsu , Victor Knopov , Joseph E. Payne , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Violetta Akopian , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nahum , Hagar Kalinski , Igor Mett , Kenjiro Minomi , Wenbin Ying , Yun Liu
发明人: Yoshiro Niitsu , Victor Knopov , Joseph E. Payne , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Violetta Akopian , Yasunobu Tanaka , Elena Feinstein , Sharon Avkin-Nahum , Hagar Kalinski , Igor Mett , Kenjiro Minomi , Wenbin Ying , Yun Liu
IPC分类号: C07H21/04
CPC分类号: A61K9/1271 , A61K9/127 , A61K31/713 , C07H21/02 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2320/32 , C12N2320/52
摘要: What is described are pharmaceutical compositions comprising a double-stranded nucleic acid molecule comprising a sense strand and an antisense strand wherein the sense and antisense strands are selected from the oligonucleotides described as SERPINH1—2 (SEQ ID NOS: 60 and 127), SERPINH1—45a (SEQ ID NOS: 98 and 165), and SERPINH1—51 (SEQ ID NOS: 101 and 168), and drug carrier comprising a mixture of a retinoid and a lipid vesicle, and methods of using these pharmaceutical compositions to treat a disease associated with hsp47 espresssion, including fibrosis.
摘要翻译: 所描述的是包含包含有义链和反义链的双链核酸分子的药物组合物,其中有义链和反义链选自描述为SERPINH1-2(SEQ ID NO:60和127)的寡核苷酸,SERPINH1- 45a(SEQ ID NO:98和165)和SERPINH1-51(SEQ ID NO:101和168)和包含类视黄醇和脂质囊泡的混合物的药物载体,以及使用这些药物组合物治疗疾病的方法 与hsp47浓缩相关,包括纤维化。
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公开(公告)号:US20130011336A1
公开(公告)日:2013-01-10
申请号:US13063193
申请日:2009-09-11
申请人: Yoshiro Niitsu , Lei Yu , Gang Zhao , Sang Van , Xinghe Wang , Jian Liu , Sanjib Kumar Das , Yasunobu Tanaka , Keiko Kajiwara , Hirokazu Takahashi , Miyono Miyazaki
发明人: Yoshiro Niitsu , Lei Yu , Gang Zhao , Sang Van , Xinghe Wang , Jian Liu , Sanjib Kumar Das , Yasunobu Tanaka , Keiko Kajiwara , Hirokazu Takahashi , Miyono Miyazaki
IPC分类号: A61K49/00 , A61K49/04 , A61K49/10 , G01N21/62 , A61K51/06 , A61K49/22 , A61P1/16 , C08G69/48 , C07C403/08 , A61K51/04
CPC分类号: A61K49/085 , A61K49/0002 , A61K49/0041 , A61K49/0052 , A61K49/0054 , A61K49/128 , A61K51/065 , C08G69/10 , C08G69/48
摘要: Agents and methods for imaging a cell and/or a portion of tissue characterized by fibrosis, as well as to agents and methods for determining and/or diagnosing fibrotic diseases are disclosed herein. Also disclosed herein are polymer conjugates that can include a detectable label, a retinoid and a polymer. The polymer conjugates can be used to image a portion of tissue, deliver a detectable label to a portion of tissue or a cell and/or diagnosis a condition or disease.
摘要翻译: 本文公开了用于成像细胞和/或特征在于纤维化的组织的一部分以及用于确定和/或诊断纤维化疾病的药剂和方法的药剂和方法。 本文还公开了可以包括可检测标记,类视色素和聚合物的聚合物缀合物。 聚合物缀合物可用于对组织的一部分进行成像,将可检测标记递送至组织或细胞的一部分和/或诊断病症或疾病。
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10.
公开(公告)号:US20130017249A1
公开(公告)日:2013-01-17
申请号:US13492424
申请日:2012-06-08
申请人: Yoshiro Niitsu , Joseph E. Payne , John A. Gaudette , Zheng Hou , Victor Knopov , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Yasunobu Tanaka , Violetta Akopian
发明人: Yoshiro Niitsu , Joseph E. Payne , John A. Gaudette , Zheng Hou , Victor Knopov , Richard P. Witte , Mohammad Ahmadian , Loren A. Perelman , Yasunobu Tanaka , Violetta Akopian
IPC分类号: A61K9/127 , A61K31/16 , C07C235/78 , A61K31/166 , A61K31/23 , A61P1/16 , A61P1/18 , A61P11/04 , C07F9/10 , A61K31/685 , A61K31/713 , C07C229/32 , C07C235/48 , C07C235/08 , C07C235/30
CPC分类号: C07C237/22 , A61K9/127 , A61K9/1271 , A61K31/07 , A61K31/713 , A61K47/551 , C07C233/21 , C07C235/08 , C07C235/48 , C07C2601/16 , C07F9/10 , C12N15/113 , C12N2310/14 , C12N2320/32 , Y10S977/773 , Y10S977/906 , Y10S977/907
摘要: Here described are compounds consisting of the structure (targeting molecule)m-linker-(targeting molecule)n, wherein the targeting molecule is a retinoid or a fat soluble vitamin having a specific receptor on the target cell; wherein m and n are independently 0, 1, 2 or 3; and wherein the linker comprises a polyethylene glycol (PEG) or PEG-like molecule, as well as compositions and pharmaceutical formulations including these compounds which are useful for the targeting and delivery of therapeutic agents; and methods of using these compositions and pharmaceutical formulations.
摘要翻译: 这里描述的是由结构(靶向分子)m-连接体(靶向分子)n组成的化合物,其中靶向分子是靶细胞上具有特异性受体的类视黄醇或脂溶性维生素; 其中m和n独立地为0,1,2或3; 并且其中所述接头包含聚乙二醇(PEG)或PEG样分子,以及包含这些化合物的组合物和药物制剂,其可用于治疗剂的靶向和递送; 以及使用这些组合物和药物制剂的方法。
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