序号 专利名 申请号 申请日 公开(公告)号 公开(公告)日 发明人
21 siRNA targeting ribonucleotide reductase M2 polypeptide (RRM2 or RNR-R2) US11818555 2007-06-13 US20080227967A1 2008-09-18 Anastasia Khvorova; Angela Reynolds; Devin Leake; William Marshall; Steven Read; Stephen Scaringe
Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to RRM2.
22 POLYPEPTIDES FOR THE DIAGNOSIS AND THE TREATMENT OF C3 NEF ASSOCIATED C3 GLOMERULOPATHY EP16736396.9 2016-06-17 EP3310804A1 2018-04-25 FREMEAUX-BACCHI, Véronique; ROUMENINA, Lubka; MARINOZZI, Maria-Chiara
The present invention relates to polypeptides for the diagnosis and treatment of C3 NeF associated C3 Glomerulopathy. In particular, the present invention is defined by the claims. In particular, the present invention relates to a polypeptide that is capable of inhibiting the binding of C3 NeF to C3 convertase and which comprises a first segment which consists of n consecutive amino acids selected in a first amino acid sequence set forth in SEQ ID NO:1 fused to a second segment which consists of n′ consecutive amino acids selected in a second amino acid sequence set forth in SEQ ID NO:2, wherein n and n′ represent integer number, n and n′≥3 and n+n′≥10.
23 COMPOSITIONS AND METHODS FOR MODULATING COMPLEMENT FACTOR B EXPRESSION EP15786214 2015-05-01 EP3137596A4 2017-09-20 PRAKASH THAZHA P; SETH PUNIT P; SWAYZE ERIC E; GROSSMAN TAMAR R; MCCALEB MICHAEL L; WATT ANDREW T; FREIER SUSAN M
The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.
24 AGE-RELATED MACULAR DEGENERATION TREATMENT EP14737958 2014-01-08 EP2943571A4 2016-11-30 SUHY DAVID; MAO TIN; KAO SHIH-CHU
This invention is directed to an RNA interference (RNAi) agent and the use of that RNAi agent to treat Age-related Macular Degeneration, as well as pharmaceutical compositions containing the RNAi agents of the invention. The RNAi agent is a DNA-directed RNA interference (ddRNAi) agent (being an RNA molecule), together with an expression cassette or construct to express that agent in a cell (including in vivo), for inhibiting, preventing or reducing expression of an AMD associated gene. Preferably that AMD associated gene is one that is associated with wet AMD.
25 Age-related macular degeneration treatment US14759401 2014-01-08 US10000753B2 2018-06-19 David Suhy; Tin Mao; Shih-Chu Kao
This invention is directed to an RNA interference (RNAi) agent and the use of that RNAi agent to treat Age-related Macular Degeneration, as well as pharmaceutical compositions containing the RNAi agents of the invention. The RNAi agent is a DNA-directed RNA interference (ddRNAi) agent (being an RNA molecule), together with an expression cassette or construct to express that agent in a cell (including in vivo), for inhibiting, preventing or reducing expression of an AMD associated gene. Preferably that AMD associated gene is one that is associated with wet AMD.
26 POLYPEPTIDES FOR THE DIAGNOSIS AND THE TREATMENT OF C3 NEF ASSOCIATED C3 GLOMERULOPATHY US15737358 2016-06-17 US20180162911A1 2018-06-14 Véronique FREMEAUX-BACCHI; Lubka ROUMENINA; Maria-Chiara MARINOZZI
The present invention relates to polypeptides for the diagnosis and treatment of C3 NeF associated C3 Glomerulopathy. In particular, the present invention is defined by the claims. In particular, the present invention relates to a polypeptide that is capable of inhibiting the binding of C3 NeF to C3 convertase and which comprises a first segment which consists of n consecutive amino acids selected in a first amino acid sequence set forth in SEQ ID NO:1 fused to a second segment which consists of n′ consecutive amino acids selected in a second amino acid sequence set forth in SEQ ID NO:2, wherein n and n′ represent integer number, n and n′≥3 and n+n′≥10.
27 AGE-RELATED MACULAR DEGENERATION TREATMENT US14759401 2014-01-08 US20160145611A1 2016-05-26 David SUHY; Tin MAO; Shih-Chu KAO
This invention is directed to an RNA interference (RNAi) agent and the use of that RNAi agent to treat Age-related Macular Degeneration, as well as pharmaceutical compositions containing the RNAi agents of the invention. The RNAi agent is a DNA-directed RNA interference (ddRNAi) agent (being an RNA molecule), together with an expression cassette or construct to express that agent in a cell (including in vivo), for inhibiting, preventing or reducing expression of an AMD associated gene. Preferably that AMD associated gene is one that is associated with wet AMD.
28 siRNA targeting cyclin-dependent kinase inhibitor 1B (p27, Kip1) (CDKN1B) US13135443 2011-07-05 US08232385B2 2012-07-31 Anastasia Khvorova; Angela Reynolds; Devin Leake; William Marshall; Steven Read; Stephen Scaringe
Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for CDKN1B.
29 siRNA targeting TNFa US12928950 2010-12-23 US20110105363A1 2011-05-05 Anastasia Khvorova; Angela Reynolds; Devin Leake; William Marshall; Steven Read; Stephen Scaringe
Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to TNFα.
30 siRNA targeting nucleoporin 62kDa (Nup62) US12657721 2010-01-26 US20100145039A1 2010-06-10 Anastasia Khvorova; Angela Reynolds; Devin Leake; William Marshall; Steven Read; Stephen Scaringe
Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for Nup62
31 siRNA Targeting TNFalpha US12209698 2008-09-12 US20090306356A1 2009-12-10 Anastasia Khvorova; Angela Reynolds; Devin Leake; William Marshall; Steven Read; Stephen Scaringe
Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to TNFα.
32 siRNA targeting ribonucleotide reductase M2 polypeptide (RRM2 or RNR-R2) US11818555 2007-06-13 US07592442B2 2009-09-22 Anastasia Khvorova; Angela Reynolds; Devin Leake; William Marshall; Steven Read; Stephen Scaringe
Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to RRM 2.
33 siRNA targeting inner centromere protein antigens (INCENP) US12157151 2008-06-06 US07582747B2 2009-09-01 Anastasia Khvorova; Angela Reynolds; Devin Leake; William Marshall; Steven Read; Stephen Scaringe
Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for INCENP.
34 siRNA targeting cyclin-dependent kinase inhibitor 1B (p27, Kip1) (CDKN1B) US11978900 2007-10-30 US20080177051A1 2008-07-24 Anastasia Khvorova; Angela Reynolds; Devin Leake; William Marshall; Steven Read; Stephen Scaringe
Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for CDKN1B.
35 보체 인자 B 발현을 조절하기 위한 조성물 및 방법 KR1020167032652 2015-05-01 KR1020160147892A 2016-12-23 프라카쉬,타즈하,피.; 세쓰,퍼닛,피.; 스웨이즈,에릭,이.; 그로스먼,타마르,알.; 맥칼렙,마이클,엘.; 와트,앤드루,티.; 프레이어,수잔,엠.
본구현예는보체인자 B (CFB) 특이적억제제를대상체에게투여함으로써보체대안경로의이상조절과연관된질환을치료, 예방, 또는개선하기위한방법, 화합물, 및조성물을제공한다.
36 보체 성분 iRNA 조성물 및 이의 이용 방법 KR1020167018461 2014-12-12 KR1020160097307A 2016-08-17 보로도브스키안나; 베텐코트브라이언
본발명은보체인자 B(CFB) 유전자, 보체성분 C3 유전자및 보체성분 C9 유전자를표적으로하는 iRNA, 예를들어, 이중-가닥리보핵산(dsRNA), 조성물, 및 CFB, C9 및/또는 C3의발현을억제하고, 보체성분-관련질병, 예를들어, 발작성야간혈색소뇨증(paroxysmal nocturnal hemoglobinuria) 및비정형용혈성요독증후군(atypical hemolytic uremic syndrome)을갖는피검자를치료하기위한이러한 iRNA, 예를들어, dsRNA, 조성물의이용방법에관한것이다.
37 COMPOSITIONS AND METHODS FOR MODULATING COMPLEMENT FACTOR B EXPRESSION US15307526 2015-05-01 US20170159055A1 2017-06-08 Thazha P. Prakash; Punit P. Seth; Eric E. Swayze; Tamar R. Grossman; Michael L. McCaleb; Andrew T. Watt; Susan M. Freier
The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.
38 Heat-inactivated complement factor B compositions and methods US14631263 2015-02-25 US09534212B2 2017-01-03 Nancy A. Turner
The present disclosure is directed to compositions comprising heat-inactivated complement factor B and methods of using the same to treat thrombotic or complement-mediated inflammatory disorders.
39 siRNA Targeting Cyclin-dependent Kinase Inhibitor 1B (p27, Kip1) (CDKN1B) US13867168 2013-04-22 US20130225667A1 2013-08-29 Anastasia Khvorova; Angela Reynolds; Devin Leake; William Marshall; Steven Read; Stephen Scaringe
Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for CDKN1B.
40 SiRNA targeting cyclin-dependent kinase inhibitor 1B (p27, Kip1) (CDKN1B) US13531657 2012-06-25 US08445667B2 2013-05-21 Anastasia Khvorova; Angela Reynolds; Devin Leake; William Marshall; Steven Read; Stephen Scaringe
Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for CDKN1B.
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