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公开(公告)号：US20160333354A1
公开(公告)日：2016-11-17
申请号：US15228214
申请日：2016-08-04
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.
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公开(公告)号：US20160102313A1
公开(公告)日：2016-04-14
申请号：US14973388
申请日：2015-12-17
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.
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公开(公告)号：US20140350068A1
公开(公告)日：2014-11-27
申请号：US14139584
申请日：2013-12-23
申请人： Elena Feinstein , Igor Mett , Hagar Kalinski
发明人： Elena Feinstein , Igor Mett , Hagar Kalinski
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C07H21/02 , C12N2310/14 , C12N2310/319 , C12N2310/343 , C12N2310/321 , C12N2310/3521
摘要： The invention provides chemically modified siRNA oligonucleotides that target RTP801L, compositions comprising same and to the use of such molecules to treat, inter alia, respiratory diseases including acute and chronic pulmonary disorders, eye diseases including glaucoma and ION, microvascular disorders, angiogenesis- and apoptosis-related conditions, and hearing impairments.
摘要翻译： 本发明提供靶向RTP801L的化学修饰的siRNA寡核苷酸，包含其的组合物，以及使用此类分子治疗尤其是呼吸系统疾病，包括急性和慢性肺病，包括青光眼和ION在内的眼睛疾病，微血管病症，血管生成和凋亡 相关条件和听力障碍。
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公开(公告)号：US08614309B2
公开(公告)日：2013-12-24
申请号：US12733998
申请日：2008-09-04
申请人： Elena Feinstein , Huseyin Aygun , Rami Skaliter , Hagar Kalinski , Igor Mett , James McSwiggen , Leonid Beigelman
发明人： Elena Feinstein , Huseyin Aygun , Rami Skaliter , Hagar Kalinski , Igor Mett , James McSwiggen , Leonid Beigelman
IPC分类号： C07H21/04
CPC分类号： C07H21/02 , C12N15/111 , C12N2310/14 , C12N2310/319 , C12N2310/321 , C12N2310/3231 , C12N2310/332 , C12N2310/346 , C12N2320/51 , C12N2310/3521
摘要： The invention relates to siRNA compounds possessing novel sequences and structural motifs which down-regulate the expression of specific human genes. The invention also relates to pharmaceutical compositions comprising such compounds and a pharmaceutically acceptable carrier. The present invention also provides a method of treating and/or preventing the incidence or severity of various diseases or conditions associated with the genes and/or symptoms associated with such diseases or conditions comprising administering to a subject in need of treatment for such disease or condition and/or symptom the compound or the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the subject.
摘要翻译： 本发明涉及具有下调特定人基因表达的新型序列和结构基序的siRNA化合物。 本发明还涉及包含这些化合物和药学上可接受的载体的药物组合物。 本发明还提供了治疗和/或预防与这些疾病或病症相关的基因和/或症状相关的各种疾病或病症的发生率或严重程度的方法，包括向需要治疗的受试者施用此类疾病或病症 和/或治疗有效剂量的化合物或药物组合物的症状，从而治疗受试者。
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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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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：US20110288155A1
公开(公告)日：2011-11-24
申请号：US13140790
申请日：2009-12-17
申请人： Elena Feinstein , Igor Mett , Hagar Kalinski
发明人： Elena Feinstein , Igor Mett , Hagar Kalinski
IPC分类号： A61K31/713 , A61P25/28 , A61P11/00 , C07H21/04 , A61P9/00 , A61P27/16 , A61P9/10 , C07H21/02 , A61P27/02
CPC分类号： C12N15/1137 , C12N2310/14 , C12N2310/321 , C12N2310/3231 , C12N2310/332 , C12N2310/344 , C12N2310/3521
摘要： The present application relates to double stranded oligonucleotide inhibitors of target genes, pharmaceutical compositions comprising same and the use of such molecules to treat, inter alia, neurodegenerative disorders including Alzheimer's disease and Amyotrophic Lateral Sclerosis, eye diseases including glaucoma and ION, acute renal failure, hearing loss, acute respiratory distress syndrome and in preventing or treating ischemia-reperfusion injury in organ transplant patients.
摘要翻译： 本申请涉及靶基因的双链寡核苷酸抑制剂，包含其的药物组合物和这种分子的用途，特别是治疗包括阿尔茨海默病和肌萎缩性侧索硬化的神经变性疾病，眼疾，包括青光眼和ION，急性肾衰竭， 听力损失，急性呼吸窘迫综合征和预防或治疗器官移植患者的缺血再灌注损伤。
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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：US09045755B2
公开(公告)日：2015-06-02
申请号：US13700942
申请日：2011-06-23
IPC分类号： C07H21/04 , 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.
摘要翻译： 本发明涉及化合物，包含其的药物组合物，其使用方法和用于下调RhoA基因的试剂盒。 化合物，组合物，方法和试剂盒可用于治疗患有RhoA表达具有不良后果和赋予神经保护作用的疾病或病症和/或与疾病或病症相关的症状的受试者。
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10.发明申请DOUBLE-STRANDED OLIGONUCLEOTIDE COMPOUNDS FOR TREATING HEARING AND BALANCE DISORDERS 审中-公开用于治疗听觉和平衡障碍的双链寡核苷酸化合物公开(公告)号：US20150018404A1
公开(公告)日：2015-01-15
申请号：US14235780
申请日：2012-08-03
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2310/321 , C12N2310/332 , C12N2310/333 , C12N2310/3341 , C12N2310/335 , C12N2310/336 , C12N2310/337 , C12N2310/3521 , C12N2310/50
摘要： The present application relates to double stranded nucleic acid compounds, compositions comprising same and methods of use thereof for the treatment of hearing loss in a subject in need thereof. The compounds are preferably chemically synthesized and modified dsRNA molecules which inhibit expression of a gene expressed selected from the group consisting of HES1, HES5, HEY1, HEY2, ID1, ID2, ID3, CDKN1B, and NOTCH1.
摘要翻译： 本申请涉及双链核酸化合物，包含其的组合物以及用于治疗有此需要的受试者的听力损失的方法。 所述化合物优选化学合成并抑制表达选自HES1，HES5，HEY1，HEY2，ID1，ID2，ID3，CDKN1B和NOTCH1的基因表达的dsRNA分子。
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