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。

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。

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。

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。

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。

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。

公开(公告)号：US20160333354A1

公开(公告)日：2016-11-17

申请号：US15228214

申请日：2016-08-04

申请人： Sharon Avkin-Nachum ,  Elena Feinstein ,  Hagar Kalinski ,  Igor Mett

发明人： Sharon Avkin-Nachum ,  Elena Feinstein ,  Hagar Kalinski ,  Igor Mett

IPC分类号： C12N15/113 ,  C12Q1/68

CPC分类号： C12N15/1135 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/321 ,  C12Q1/6888

摘要： The present invention relates to compounds, pharmaceutical compositions comprising same, methods of use thereof and kits for the down-regulation of RhoA gene. The compounds, compositions, methods and kits are useful in the treatment of subjects suffering from diseases or conditions and or symptoms associated with diseases or conditions in which RhoA expression has adverse consequences and for conferring neuroprotection.

公开(公告)号：US20160102313A1

公开(公告)日：2016-04-14

申请号：US14973388

申请日：2015-12-17

申请人： Sharon Avkin-Nachum ,  Elena Feinstein ,  Hagar Kalinski ,  Igor Mett

发明人： Sharon Avkin-Nachum ,  Elena Feinstein ,  Hagar Kalinski ,  Igor Mett

IPC分类号： C12N15/113

CPC分类号： C12N15/1135 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/321 ,  C12Q1/6888

摘要： The present invention relates to compounds, pharmaceutical compositions comprising same, methods of use thereof and kits for the down-regulation of RhoA gene. The compounds, compositions, methods and kits are useful in the treatment of subjects suffering from diseases or conditions and or symptoms associated with diseases or conditions in which RhoA expression has adverse consequences and for conferring neuroprotection.

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。

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。