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1. 发明申请

WO2004097020A3 RNA INTERFERENCE MEDIATED INHIBITION OF MAP KINASE GENE EXPRESSION 审中-公开
标题翻译： RNA干扰介导的MAP激酶基因表达的抑制
公开(公告)号：WO2004097020A3
公开(公告)日：2006-01-05
申请号：PCT/US2004012517
申请日：2004-04-23
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , BEIGELMAN LEONID , USMAN NASSIM , HAEBERLI PETER , CHOWRIRA BHARAT , POLISKY BARRY
发明人： MCSWIGGEN JAMES , BEIGELMAN LEONID , USMAN NASSIM , HAEBERLI PETER , CHOWRIRA BHARAT , POLISKY BARRY
IPC分类号： A61K38/00 , C12N15/11 , C12N15/113 , C12N15/87 , A61K31/713
CPC分类号： C12N15/111 , A61K38/00 , C12N15/1137 , C12N15/87 , C12N2310/14 , C12N2310/315 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2320/11 , C12N2320/32 , C12N2330/30 , C12Y207/11024 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating mitogen activated protein kinase (MAP kinase ) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of MAP kinase and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of mitogen activated protein kinase (MAP kinase ) genes such as c-JUN, JNK1, JNK2, p38, ERK1, or ERK2.
摘要翻译：本发明涉及使用短干扰核酸（siNA）分子调节有丝分裂原活化蛋白激酶（MAP激酶）基因表达的化合物，组合物和方法。本发明还涉及可用于通过使用小核酸分子调节涉及MAP激酶和/或活性通过RNA干扰（RNAi）的途径的其它基因的表达和活性的化合物，组合物和方法。特别地，本发明的特征在于小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA））分子和用于调节丝裂原活化蛋白激酶（MAP激酶）基因如c-JUN，JNK1，JNK2，p38，ERK1或ERK2的表达的方法。

2. 发明申请

WO2005044981A3 RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA介导的干扰介导的短基因干扰抑制基因表达（SINA）
公开(公告)号：WO2005044981A3
公开(公告)日：2005-09-22
申请号：PCT/US2004027403
申请日：2004-08-20
申请人： SIRNA THERAPEUTICS INC , HAEBERLI PETER , MCSWIGGEN JAMES
发明人： HAEBERLI PETER , MCSWIGGEN JAMES
IPC分类号： A61K38/00 , C12N5/02 , C12N5/22 , C12N15/11 , C12N15/113 , C12N15/87 , A61K31/713 , A61P31/00 , C07H3/02 , C07H21/04 , C12P19/34
CPC分类号： A61K49/0008 , C12N15/111 , C12N15/1135 , C12N15/87 , C12N2310/111 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2320/32 , C12N2330/30 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating gene expression using short interfering nucleic acid (siNA) molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of genes, such as expressed pseudogenes associated with the maintenance or development of diseases, disorders, traits, and conditions in a subject or organism.
摘要翻译：本发明涉及用于使用短干扰核酸（siNA）分子调节基因表达的化合物，组合物和方法。特别地，本发明特征在于小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA））分子和方法，用于调节基因的表达，例如与受试者或生物体中疾病，病症，特征和病症的维持或发展有关的表达假基因。

3. 发明申请

WO2004092383A2 RNA INTERFERENCE MEDIATED INHIBITION OF SEVERE ACUTE RESPIRATORY SYNDROME (SARS) VIRUS GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的严重急性呼吸道综合征（SARS）病毒基因表达的抑制使用短暂的核酸（siNA）
公开(公告)号：WO2004092383A2
公开(公告)日：2004-10-28
申请号：PCT/US2004011320
申请日：2004-04-13
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , BHARAT CHOWIRA , HAEBERLI PETER
发明人： MCSWIGGEN JAMES , BHARAT CHOWIRA , HAEBERLI PETER
IPC分类号： A61K38/00 , C12N15/113 , C12N15/87 , C12N15/11
CPC分类号： C12N15/87 , A61K38/00 , C12N15/1131 , C12N2310/14 , C12N2310/315 , C12N2310/316 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2310/3521
摘要： The present invention comprises compounds, compositions, and methods useful for modulating the expression of genes associated with respiratory and pulmonary disease, such as severe acute respiratory syndrome (SARS) virus genes, using short interfering nucleic acid (siNA) molecules. This invention also comprises compounds, compositions, and methods useful for modulating the expression and activity of SARS virus genes, or other genes involved in pathways of SARS virus gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of SARS virus RNA.
摘要翻译：本发明包括使用短干扰核酸（siNA）分子来调节与呼吸和肺部疾病相关基因表达的重要的急性呼吸综合征（SARS）病毒基因的化合物，组合物和方法。本发明还包括可用于通过使用小核酸分子调节SARS病毒基因或涉及SARS病毒基因表达和/或活性通过RNA干扰（RNAi）的途径的其它基因的表达和活性的化合物，组合物和方法。特别地，本发明的特征在于小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA））分子和用于调节SARS病毒RNA表达的方法。

4. 发明申请

WO2005045034A3 RNA INTERFERENCE MEDIATED TREATMENT OF PARKINSON DISEASE USING SHORT INTERERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的治疗帕金森病的短交会核酸（siNA）
公开(公告)号：WO2005045034A3
公开(公告)日：2005-08-11
申请号：PCT/US2004017630
申请日：2004-06-03
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , HAEBERLI PETER , BEIGELMAN LEONID
发明人： MCSWIGGEN JAMES , HAEBERLI PETER , BEIGELMAN LEONID
IPC分类号： C12N15/113 , C12N15/11 , A01N43/04 , A61K31/713 , C07H21/02 , C07H21/04 , C12P19/34
CPC分类号： C12N15/113 , A61K49/0008 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/3521
摘要： The present invention concerns methods and reagents useful in modulating Parkinson genes, for example, PARK1 (SNCA), PARK2, PARK7, and/or PARK5 gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against SNCA gene expression and/or activity. The small nucleic acid molecules are useful in the diagnosis and treatment of Parkinson Disease (PD), and any other disease or condition that responds to modulation of PARK1 (SNCA), PARK2, PARK7, and/or PARK5 expression or activity.
摘要翻译：本发明涉及用于在多种应用中调节帕金森基因例如PARK1（SNCA），PARK2，PARK7和/或PARK5基因表达的方法和试剂，包括用于治疗，诊断，靶标验证和基因组发现应用。具体而言，本发明涉及小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA）能够介导针对SNCA基因表达和/或活性的RNA干扰（RNAi）的分子。小核酸分子可用于帕金森病（PD）的诊断和治疗以及对PARK1（SNCA），PARK2，PARK7和/或PARK5表达或活性的调节作出反应的任何其他疾病或病症。

5. 发明申请

WO2005045035A2 RNA INTERFERENCE MEDIATED INHIBITION OF NOGO AND NOGO RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的抑制NOGO和NOGO受体基因表达使用短暂的干扰核酸（siNA）
公开(公告)号：WO2005045035A2
公开(公告)日：2005-05-19
申请号：PCT/US2004026930
申请日：2004-08-20
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , CHOWRIRA BHARAT M , HAEBERLI PETER
发明人： MCSWIGGEN JAMES , CHOWRIRA BHARAT M , HAEBERLI PETER
IPC分类号： A61K38/00 , C12N5/02 , C12N5/22 , C12N15/11 , C12N15/113 , C12N15/87
CPC分类号： A61K49/0008 , C12N15/111 , C12N15/1135 , C12N15/87 , C12N2310/111 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2320/32 , C12N2330/30 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating NOGO and/or NOGO receptor gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of NOGO and/or NOGO receptor gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of NOGO and/or NOGO receptor genes, such as NOGO-A, NOGO-B, NOGO-C, NOGO-66 receptor, NI-35, NI-220, NI-250, myelin-associated glycoprotein, tenascin-R, and NG-2
摘要翻译：本发明涉及使用短干扰核酸（siNA）分子调节NOGO和/或NOGO受体基因表达的化合物，组合物和方法。本发明还涉及可用于通过使用小核酸分子调节参与NOGO和/或NOGO受体基因表达和/或通过RNA干扰（RNAi）的活性的其它基因的表达和活性的化合物，组合物和方法。特别地，本发明的特征在于小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA））分子和用于调节NOGO和/或NOGO受体基因如NOGO-A，NOGO-B，NOGO-C，NOGO-66受体，NI-35，NI-220，NI-250，髓磷脂相关糖蛋白，腱生蛋白-R和NG-2

6. 发明专利

AU2003215345A1 RNA INTERFERENCE MEDIATED INHIBITION OF HIV GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 未知
公开(公告)号：AU2003215345A1
公开(公告)日：2003-09-09
申请号：AU2003215345
申请日：2003-02-20
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES
发明人： BEIGELMAN LEONID , MACEJAK DENNIS , MCSWIGGEN JAMES
IPC分类号： A61K31/7088 , A61K31/712 , A61K31/713 , A61K38/00 , A61K45/06 , A61P31/18 , C07H21/00 , C12N15/11 , C12N15/113
摘要： The present invention concerns methods and reagents useful in modulating HIV gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small interfering RNA (siRNA) molecules capable of mediating RNA interference (RNAi) against HIV polypeptide and polynucleotide targets.

7. 发明申请

WO2005045036A3 RNA INTERFERENCE MEDIATED INHIBITION OF HAIRLESS (HR) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA干扰介导的使用短暂干扰核酸（SINA）的无毛（HR）基因表达的抑制
公开(公告)号：WO2005045036A3
公开(公告)日：2006-03-02
申请号：PCT/US2004027042
申请日：2004-08-18
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES
发明人： MCSWIGGEN JAMES
IPC分类号： A01N43/04 , A61K31/713 , A61K48/00 , C07H21/02 , C07H21/04 , C12N15/113 , C12P19/34
CPC分类号： C12N15/113 , C12N2310/14
摘要： This invention relates to compounds, compositions, and methods useful for modulating Hairless (HR) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of Hairless gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of Hairless.
摘要翻译：本发明涉及使用短干扰核酸（siNA）分子调节无毛（HR）基因表达的化合物，组合物和方法。本发明还涉及可用于通过使用小核酸分子调节涉及无毛基因表达和/或通过RNA干扰（RNAi）的活性的途径的其它基因的表达和活性的化合物，组合物和方法。特别地，本发明的特征在于小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA））分子和方法用于调节无毛的表达。

8. 发明申请

WO2005045038A8 RNA INTERFERENCE MEDIATED INHIBITION OF GPRA AND AAA1 GENE EXPRESSION USING SHORT NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的GPRA和AAA1基因表达的抑制使用短核酸（siNA）
公开(公告)号：WO2005045038A8
公开(公告)日：2005-07-14
申请号：PCT/US2004027231
申请日：2004-08-20
申请人： SIRNA THERAPEUTICS INC , RICHARDS IVAN , POLISKY BARRY , MCSWIGGEN JAMES
发明人： RICHARDS IVAN , POLISKY BARRY , MCSWIGGEN JAMES
IPC分类号： A01N43/04 , A61K31/713 , A61K38/00 , C07H21/02 , C07H21/04 , C12N15/113 , C12N15/87 , C12P19/34 , C12N15/11
CPC分类号： C12N15/87 , A61K38/00 , A61K49/0008 , C12N15/113 , C12N15/1138 , C12N2310/111 , C12N2310/12 , C12N2310/121 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating G protein-coupled receptor for asthma susceptibility (GPRA) and asthma-associated alternatively spliced gene 1 (AAA1) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of GPRA and/or AAA1 gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of GPRA and/or AAA1 genes.
摘要翻译：本发明涉及使用短干扰核酸（siNA）分子调节G蛋白偶联受体哮喘易感性（GPRA）和哮喘相关性剪接基因1（AAA1）基因表达的化合物，组合物和方法。本发明还涉及可用于通过使用小核酸分子调节涉及GPRA和/或AAA1基因表达和/或活性通过RNA干扰（RNAi）的途径的其它基因的表达和活性的方法。特别地，本发明具有小的核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA））分子和用于调节GPRA和/或AAA1基因表达的方法。

9. 发明申请

WO03070914A2 RNA INTERFERENCE MEDIATED INHIBITION OF FOS GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID siNA 审中-公开
标题翻译： RNA干扰介导的抑制FOS基因表达使用短暂的干扰核酸siNA
公开(公告)号：WO03070914A2
公开(公告)日：2003-08-28
申请号：PCT/US0305162
申请日：2003-02-20
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , BEIGELMAN LEONID
发明人： MCSWIGGEN JAMES , BEIGELMAN LEONID
IPC分类号： C12N15/09 , A61K31/7088 , A61K31/7105 , A61K31/711 , A61K31/7115 , A61K31/712 , A61K31/7125 , A61K31/713 , A61K38/00 , A61K47/48 , A61K48/00 , A61P1/00 , A61P1/04 , A61P1/16 , A61P3/00 , A61P3/10 , A61P11/06 , A61P13/08 , A61P13/10 , A61P13/12 , A61P17/00 , A61P17/02 , A61P19/00 , A61P19/02 , A61P21/00 , A61P25/00 , A61P25/02 , A61P25/28 , A61P27/02 , A61P27/16 , A61P29/00 , A61P31/00 , A61P31/04 , A61P31/10 , A61P31/12 , A61P31/14 , A61P31/16 , A61P31/18 , A61P31/20 , A61P31/22 , A61P35/00 , A61P35/02 , A61P37/00 , A61P37/04 , A61P37/06 , A61P37/08 , A61P43/00 , C07H21/02 , C07H21/04 , C12N15/11 , C12N15/113 , C12N15/82 , C12N
CPC分类号： C12N15/8218 , A61K38/00 , A61K47/54 , C07H21/02 , C12N15/111 , C12N15/1131 , C12N15/1138 , C12N2310/111 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/344 , C12N2310/346 , C12N2310/53 , C12N2320/51 , C12N2330/30 , C12N2310/3521
摘要： The present invention concerns methods and reagents useful in modulating c-fos gene expression in a variety of applications, including use of therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid siNA, short interfering RNA siRNA, double-stranded RNA dsRNA, micro-RNA miRNA, and short hairpin RNA shRNA molecules capable of mediating RNA interference RNAi against c-fos gene expression and/ activity. The small nucleic acid molecules are useful in the diagnosis and treatment of cancer, proliferative diseases, and any other disease or condition that responds to modulation of c-fos expression or activity.
摘要翻译：本发明涉及可用于调节各种应用中的c-fos基因表达的方法和试剂，包括使用治疗，诊断，靶标验证和基因组发现应用。具体地说，本发明涉及能够介导RNA干扰RNAi的小核酸分子，例如短干扰核酸siNA，短干扰RNA siRNA，双链RNA dsRNA，微RNA miRNA和短发夹RNA shRNA分子， fos基因表达和/活性。小核酸分子可用于诊断和治疗癌症，增殖性疾病以及对c-fos表达或活性调节作出反应的任何其它疾病或病症。

10. 发明申请

WO2006128141A3 RNA INTERFERENCE MEDIATED INHIBITION OF STROMAL CELL-DERIVED FACTOR-1 (SDF-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA干扰介导的使用短暂干扰核酸（SINA）的细胞衍生因子-1（SDF-1）基因表达的抑制作用
公开(公告)号：WO2006128141A3
公开(公告)日：2007-07-12
申请号：PCT/US2006020846
申请日：2006-05-26
申请人： SIRNA THERAPEUTICS INC , GUERCIOLINI ROBERTO , MCSWIGGEN JAMES , JADHAV VASANT , VARGEESE CHANDRA
发明人： GUERCIOLINI ROBERTO , MCSWIGGEN JAMES , JADHAV VASANT , VARGEESE CHANDRA
IPC分类号： C07K14/52 , C12N15/113
CPC分类号： C12N15/1136 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/3521
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of stromal cell-derived factor- 1 (SDF-I) gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in stromal cell-derived factor- 1 (SDF-I) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against stromal cell- derived factor- 1 (SDF-I) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate SDF-I gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce angiogenesis related diseases traits and conditions, including but not limited to ocular disease, cancer and proliferative diseases, traits, and conditions, and/or other disease states, conditions, or traits associated with SDF-I gene expression or activity in a subject or organism.
摘要翻译：本发明涉及用于研究，诊断和治疗对基质细胞衍生因子-1（SDF-1）基因表达和/或活性的调节作用的性状，疾病和病症的化合物，组合物和方法。本发明还涉及涉及对参与基质细胞衍生的因子-1（SDF-1）基因表达途径的基因的表达和/或活性的调节作用的性状，疾病和病症有关的化合物，组合物和方法或介导这些性状，疾病和状况的维持或发展的其他细胞过程。具体地说，本发明涉及包括小核酸分子的双链核酸分子，如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和能够介导针对基质细胞衍生因子-1（SDF-1）基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子，包括这种小核酸的这种小核酸分子和脂质纳米颗粒（LNP）制剂的混合物酸分子。本发明还涉及可以抑制内源性微RNA（miRNA）（例如miRNA抑制剂）或内源性短干扰RNA（例如miRNA抑制剂）的内源性RNA分子的功能的小核酸分子，例如siNA，siRNA等， siRNA）（例如siRNA抑制剂）或可以抑制RISC功能（例如RISC抑制剂）通过干扰与这些内源性RNA相关的内源性RNA或蛋白质的调节功能来调节SDF-1基因表达（例如，，RISC），包括这种小核酸分子的混合物和这种小核酸分子的脂质纳米颗粒（LNP）制剂。这样的小核酸分子并且可用于例如提供预防，抑制或减少血管生成相关疾病性状和病症的组合物，包括但不限于眼部疾病，癌症和增殖性疾病，性状和病症，和/或与受试者或生物体内的SDF-1基因表达或活性相关的其它疾病状态，病症或性状。

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