SI9200236B - Sestavek za uvedbo nukleinskih kislin v višje evkariotske celice - Google Patents

Sestavek za uvedbo nukleinskih kislin v višje evkariotske celice Download PDF

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SI9200236B
SI9200236B SI9200236A SI9200236A SI9200236B SI 9200236 B SI9200236 B SI 9200236B SI 9200236 A SI9200236 A SI 9200236A SI 9200236 A SI9200236 A SI 9200236A SI 9200236 B SI9200236 B SI 9200236B
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nucleic acid
conjugate
gly
composition according
composition
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SI9200236A
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SI9200236A (en
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David Curiel
Ernst Wagner
Matt Cotten
Kurt Zatloukal
Christian Plank
L. Max Birnstiel
Bernd Oberhauser
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Boehringer Ingelheim
The University Of North
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Claims (94)

1 Patentni zahtevki 1. Sestavek za transfekcijo višjih evkariotskih celic, vsebujoč kompleks, v katerem je nukleinska kislina kompleksirana s snovjo z afiniteto za nukleinsko kislino, ki je v danem primeru konjugirana z intemalizimim faktoijem za celice, označen s tem, da sestavek poleg tega vsebuje endosomolitsko sredstvo, pri čemer a) se endosomolitsko sredstvo nahaja kot sestavina kompleksa, tako daje preko njemu lastne vezivne domene nukleinske kisline ali preko snovi z afiniteto za nukleinsko kislino, na katero je vezano, kompleksirano z nukleinsko kislino, in pri čemer kompleks v danem primeru poleg tega vsebuje intemalizimi faktor, ki je preko snovi z afiniteto za nukleinsko kislino, s katero je konjugiran, prav tako kompleksiran z nukleinsko kislino ali pri čemer b) se kot endosomolitsko sredstvo virus ali virusna komponenta, ki ima sposobnost, da sam zase (sama zase) prodre v celico, ki jo je treba transficirati, nahaja izven kompleksa, v katerem je snov z afiniteto za nukleinsko kislino, ki je v danem primeru konjugirana z intemalizimim faktorjem, kompleksirana z nukleinsko kislino.
2. Sestavek po zahtevku 1, označen s tem, da je v sestavku po a) endosomolitsko sredstvo kovalentno vezano na snov z afiniteto za nukleinsko kislino.
3. Sestavek po zahtevku 1, označen s tem, da je v sestavku po a) endosomolitsko sredstvo nekovalentno vezano na snov z afiniteto za nukleinsko kislino.
4. Sestavek po zahtevku 3, označen s tem, da poteče vezava preko mostu biotina-streptavidina.
5. Sestavek po zahtevku 3, označen s tem, da vezavo izvedemo ionsko. 2
6. Sestavek po zahtevku 1, označen s tem, da je v sestavku po a) endosomolitsko sredstvo virus ali virusna komponenta.
7. Sestavek po zahtevku 1 ali 6, označen s tem, da je v sestavku po a) ali b) endosomolitsko sredstvo adenovirus.
8. Sestavek po zahtevku 7, označen s tem, daje adenovirus mutant.
9. Sestavek po zahtevku 8, označen s tem, da je adenovirus replikacijsko nekompetenten mutant.
10. Sestavek po zahtevku 9, označen s tem, da ima adenovirus eno ali več mutacij in/ali delecij v El A regiji.
11. Sestavek po zahtevku 7, označen s tem, daje adenovirus deaktiviran.
12. Sestavek po zahtevku 11, označen s tem, da je adenovirus deaktiviran s kratkovalovno UV.
13. Sestavek po zahtevku 11, označen s tem, da je adenovirus deaktiviran z UV/psoralenom.
14. Sestavek po zahtevku 11, označen s tem, da je adenovirus deaktiviran s formaldehidom.
15. Sestavek po zahtevku 6, označen s tem, da virusna komponenta obstoji iz enega ali več adenovirusnih proteinov. 3
16. Sestavek po zahtevku 1 ali 6, označen s tem, da je v sestavku po a) ali b) endosomolitsko sredstvo pikoma virus.
17. Sestavek po zahtevku 16, označen s tem, da je pikoma vims rinovirus, po želji deaktiviran.
18. Sestavek po zahtevku 1, označen s tem, daje endosomolitsko sredstvo v sestavku po a) vims, kije infekciozen za vrsto, ki ni humana.
19. Sestavek po zahtevku 18, označen s tem, daje vims adenovims.
20. Sestavek po zahtevku 19, označen s tem, daje adenovims ptičji adenovims.
21. Sestavek po zahtevku 20, označen s tem, da je adenovims letalni vims sirota piščančjega embria.
22. Sestavek po zahtevku 1, označen s tem, daje endosomolitsko sredstvo v sestavku po a) v danem primem modificiran endosomolitski vimsni peptid.
23. Sestavek po zahtevku 22, označen s tem, daje endosomolitski peptid influenčni hemaglutininski HA2-peptid.
24. Sestavek po zahtevku 23, označen s tem, da ima peptid sekvenco Gly-Leu-Phe-Glu-Ala-Ile-Ala-Gly-Phe-Ile-Glu-Asn-Gly-Trp-Glu-Gly-Met-Ile-Asp-Gly-Gly-Gly-Cys.
25. Sestavek po zahtevku 23, označen s tem, da ima peptid sekvenco Gly-Leu-Phe-Gly-Ala-Ile-Ala-Gly-Phe-Ile-Glu-Asn-Gly-Trp-Glu-Gly-Met-Ile-Asp-Gly-Gly-Gly-Cys. 4
26. Sestavek po zahtevku 1, označen s tem, daje endosomolitsko sredstvo v sestavku po a) nevirusni, v danem primeru modificiran, naraven ali sintetičen peptid, ki ima sposobnost porušenja celičnih membran, ki se da določiti s pomočjo testa liposomske prepustnosti in/ali testa eritrocitne liže.
27. Sestavek po zahtevku 22 ali 26, označen s tem, da ima peptid vezivno domeno nukleinske kisline.
28. Sestavek po zahtevku 27, označen s tem, da ima peptid oligolizinski podaljšek.
29. Sestavek po zahtevku 1, označen s tem, daje intemalizimi faktor transferin.
30. Sestavek po zahtevku 1, označen s tem, da je intemalizimi faktor ligand za hepatocite.
31. Sestavek po zahtevku 30, označen s tem, da je intemalizimi faktor ligand za asialoglikoproteinski receptor.
32. Sestavek po zahtevku 31, označen s tem, daje intemalizimi faktor tetragalaktoza-polilizin.
33. Kompleks kot sestavina sestavka po enem od zahtevkov 1 do 32, označen s tem, da vsebuje eno ali več nukleinskih kislin, s katerimi je endosomolitsko sredstvo preko njemu lastne vezavne domene nukleinske kisline ali preko snovi z afiniteto za nukleinsko kislino, na katero je vezano, kompleksirano in da v danem primem vsebuje intemalizimi faktor, ki je preko snovi z afiniteto za nukleinsko kislino, s katero je konjugiran, prav tako kompleksiran z nukleinsko kislino. 5
34. Kompleks po zahtevku 33, označen s tem, da vsebuje terapevtsko aktivno nukleinsko kislino.
35. Kompleks po zahtevku 34, označen s tem, da nukleinska kislina obstoji iz ene ali več DNA molekul, ki so aktivne pri genski terapiji.
36. Kompleks po zahtevku 35, označen s tem, da DNA molekula kodira za citokin.
37. Kompleks po zahtevku 34, označen s tem, da nukleinska kislina vsebuje nukleotidno sekvenco, iz katere transkribiramo RNA molekule, ki specifično inhibirajo celične funkcije.
38. Kompleks po zahtevku 33, označen s tem, da je snov z afiniteto za nukleinsko kislino organski polikation.
39. Kompleks po zahtevku 38, označen s tem, daje polikation polilizin.
40. Kompleks po zahtevku 33, označen s tem, da vsebuje intemalizimi faktor, ki je vezan na isto snov z afiniteto za nukleinsko kislino kot endosomolitsko sredstvo.
41. Kompleks po zahtevku 40, označen s tem, da je snov z afiniteto za nukleinsko kislino polilizin.
42. Kompleks po zahtevku 41, označen s tem, da poleg tega vsebuje nekovalentno vezan polilizin.
43. Konjugat kot sestavina kompleksa po enem od zahtevkov 33 do 42, označen s tem, da konjugat obstoji iz endosomolitskega sredstva in snovi z afiniteto za nukleinsko kislino. 6
44. Konjugat po zahtevku 43, označen s tem, da je endosomolitsko sredstvo kovalentno vezano na snov z afiniteto za nukleinsko kislino.
45. Konjugat po zahtevku 43, označen s tem, da je endosomolitsko sredstvo nekovalentno vezano na snov z afiniteto za nukleinsko kislino.
46. Konjugat po zahtevku 45, označen s tem, da vezavo izvedemo preko mostu biotina-streptavidina.
47. Konjugat po zahtevku 45, označen s tem, da vezavo izvedemo ionsko.
48. Konjugat po zahtevku 43, označen s tem, daje endosomolitsko sredstvo virus ali virusna komponenta.
49. Konjugat po zahtevku 48, označen s tem, da je endosomolitsko sredstvo adenovirus.
50. Konjugat po zahtevku 49, označen s tem, daje adenovirus mutant.
51. Konjugat po zahtevku 50, označen s tem, da je adenovirus replikacijsko nekompetenten mutant.
52. Konjugat po zahtevku 51, označen s tem, da ima adenovirus eno ali več mutacij in/ali delecij v ElA-regiji.
53. Konjugat po zahtevku 49, označen s tem, daje adenovirus deaktiviran. 7
54. Konjugat po zahtevku 53, označen s tem, da je adenovirus deaktiviran s kratkovalovno UV.
55. Konjugat po zahtevku 53, označen s tem, da je adenovirus deaktiviran z UV/psoralenom.
56. Konjugat po zahtevku 53, označen s tem, da je adenovirus deaktiviran s formaldehidom.
57. Konjugat po zahtevku 48, označen s tem, da virusna komponenta obstoji iz enega ali več adenovirusnih proteinov.
58. Konjugat po zahtevku 48, označen s tem, daje virus pikoma vims.
59. Konjugat po zahtevku 58, označen s tem, daje vims rinovirus, po želji deaktiviran.
60. Konjugat po zahtevku 43, označen s tem, da endosomolitsko sredstvo nima samo zase sposobnosti, da prodre v celico, ki jo je treba transficirati.
61. Konjugat po zahtevku 60, označen s tem, daje endosomolitsko sredstvo vims ali vimsna komponenta, ki ni intemalizimi faktor za humano celico.
62. Konjugat po zahtevku 61, označen s tem, da je endosomolitsko sredstvo vims, ki je infekciozen za vrsto, ki ni človeška.
63. Konjugat po zahtevku 62, označen s tem, da je vims adenovims.
64. Konjugat po zahtevku 63, označen s tem, da je adenovims ptičji adenovims. 8
65. Konjugat po zahtevku 64, označen s tem, da je adenovirus letalni virus sirota piščančjega embrija.
66. Konjugat po zahtevku 60, označen s tem, da je endosomolitsko sredstvo v danem primeru modificiran endosomolitski virusni peptid z lastnostjo, definirano v zahtevku 26.
67. Konjugat po zahtevku 66, označen s tem, da je endosomolitski peptid influenčni hemaglutininski HA2 peptid.
68. Konjugat po zahtevku 67, označen s tem, da ima peptid sekvenco Gly-Leu-Phe-Glu-Ala-Ile-Ala-Gly-Phe-Ile-Glu-Asn-Gly-Trp-Glu-Gly-Met-Ile-Asp-Gly-Gly-Gly- Cys.
69. Konjugat po zahtevku 67, označen s tem, da ima peptid sekvenco Gly-Leu-Phe-Gly-Ala-Ile-Ala-Gly-Phe-Ile-Glu-Asn-Gly-Trp-Glu-Gly-Met-Ile-Asp-Gly-Gly-Gly-Cys.
70. Konjugat po zahtevku 60, označen s tem, da je endosomolitsko sredstvo peptid, definiran v zahtevku 26.
71. Konjugat po zahtevku 70, označen s tem, da ima peptid vezivno domeno nukleinske kisline.
72. Konjugat po zahtevku 71, označen s tem, da ima peptid oligolizinski podaljšek.
73. Endosomolitski peptid, primeren kot sestavina sestavka po zahtevku 27, označen s tem, da ima endosomolitsko domeno in umetno uvedeno vezivno domeno nukleinske kisline. 9
74. Peptid po zahtevku 73, označen s tem, da je vezivna domena nukleinske kisline oligolizinski podaljšek.
75. Postopek za pripravo konjugata po zahtevku 44, označen s tem, da virus ali (poli)peptidno endosomolitsko sredstvo in poliamin encimatsko pripajamo v prisotnosti transglutaminaze.
76. Postopek za pripravo konjugata po zahtevku 44, označen s tem, da virus ali (poli)peptidno endosomolitsko sredstvo in poliamin kemično pripajamo.
77. Postopek za pripravo konjugata po zahtevku 46, označen s tem, da virus ali virusno komponento modificiramo z biotinom in da modificiran virus ali modificirano virusno komponento vežemo na streptavidinsko pripajan poliamin.
78. Postopek za uvedbo nukleinske kisline v višje evkariotske celice in vitro ali ex vivo, označen s tem, da celice obdelamo s sestavkom po enem od zahtevkov 1 do 32.
79. Postopek po zahtevku 78, označen s tem, da so celice humane celice.
80. Postopek po zahtevku 79, označen s tem, da so celice tumorske celice.
81. Postopek po zahtevku 79, označen s tem, da so celice mioblasti.
82. Postopek po zahtevku 79, označen s tem, da so celice fibroblasti.
83. Postopek po zahtevku 79, označen s tem, da so celice hepatociti.
84. Postopek po zahtevku 79, označen s tem, da so celice endotelne celice. 10
85. Postopek po zahtevku 84, označen s tem, da so celice celice dihalnih poti.
86. Postopek po enem od zahtevkov 79 do 85, označen s tem, daje nukleinska kislina v sestavku aktivna v genski terapiji.
87. Postopek po zahtevkih 80 in 86, označen s tem, da tumorske celice obdelamo s sestavkom ex vivo in da DNA kodira za eno ali več imunomodulacijskih snovi, prednostno za citokine.
88. Postopek za pripravo heterolognega proteina v višji evkariotski celici in vitro, označen s tem, da celice obdelamo s sestavkom po zahtevku 1, pri čemer nukleinska kislina vsebuje DNA sekvenco, ki kodira za želeni protein, celice gojimo ob pogojih, ki so primerni za ekspresijo proteina, in protein izoliramo.
89. Farmacevtski sestavek, označen s tem, da kot učinkovito sestavino vsebuje sestavek po enem od zahtevkov 1 do 32, ki vsebuje terapevtsko učinkovito nukleinsko kislino in farmacevtsko sprejemljiv nosilec.
90. Transfekcijski komplet, označen s tem, da vsebuje nosilčno enoto, v kateri se nahajata dve ali več posod, pri čemer prva posoda vsebuje snov z afiniteto za nukleinsko kislino, ki je po želji konjugirana z intemalizimim faktorjem za višjo evkariotsko celico, in druga posoda vsebuje endosomolitsko sredstvo, ki je virus ali virusna komponenta, ki ima sposobnost, da samo zase (sama zase) prodre v celico, ki jo je treba transficirati.
91. Transfekcijski komplet, označen s tem, da vsebuje nosilčno enoto, v kateri se nahajata dve ali več posod, pri čemer prva posoda vsebuje snov z afiniteto za nukleinsko kislino, ki je konjugirana z intemalizimim faktorjem za višjo evkariotsko 11 celico, in druga posoda vsebuje snov z afiniteto za nukleinsko kislino, kije vezana na endosomolitsko sredstvo.
92. Transfekcijski komplet po zahtevku 91, označen s tem, da v drugi posodi vsebuje transglutaminazno pripajan konjugat adenovirusa-polilizina.
93. Transfekcijski komplet, označen s tem, da vsebuje nosilčno enoto, v kateri se nahajata dve ali več posod, pri čemer prva posoda vsebuje biotinsko modificirano endosomolitsko sredstvo in druga posoda vsebuje streptavidinsko modificirano snov z afiniteto za nukleinsko kislino.
94. Transfekcijski komplet po zahtevku 93, označen s tem, da prva posoda vsebuje biotiniliran adenovirus in druga posoda vsebuje streptavidin-polilizin.
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Families Citing this family (415)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5354844A (en) * 1989-03-16 1994-10-11 Boehringer Ingelheim International Gmbh Protein-polycation conjugates
NZ244306A (en) * 1991-09-30 1995-07-26 Boehringer Ingelheim Int Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation
US5981273A (en) * 1991-09-30 1999-11-09 Boehringer Ingelheim Int'l. Gmbh Composition comprising an endosomolytic agent for introducing nucleic acid complexes into higher eucaryotic cells
EP0632722A4 (en) * 1992-03-20 1997-07-30 Baylor College Medicine DNA TRANSPORTATION SYSTEM AND INSTRUCTIONS FOR USE.
WO1993019768A1 (en) * 1992-04-03 1993-10-14 The Regents Of The University Of California Self-assembling polynucleotide delivery system
WO1994006923A1 (en) * 1992-09-24 1994-03-31 The University Of Connecticut Modification of a virus to redirect infectivity and enhance targeted delivery of polynucleotides to cells
EP0689604A1 (de) * 1993-03-19 1996-01-03 BOEHRINGER INGELHEIM INTERNATIONAL GmbH Verfahren zur herstellung von krebsvakzinen
DE4311651A1 (de) * 1993-04-08 1994-10-13 Boehringer Ingelheim Int Virus für den Transport von Fremd-DNA in höhere eukaryotische Zellen
US5578475A (en) * 1993-07-12 1996-11-26 Life Technologies, Inc. Composition and methods for transfecting eukaryotic cells
DE4335025A1 (de) * 1993-10-14 1995-04-20 Boehringer Ingelheim Int Endosomolytisch wirksame Partikel
EP0648493A1 (en) * 1993-10-19 1995-04-19 Tadatsugu Prof. Dr. Taniguchi A method to reverse the phenotype of transformed cells by the transcription factor IRF-1
US5928944A (en) * 1994-02-04 1999-07-27 The United States Of America As Represented By The Department Of Health And Human Services Method of adenoviral-medicated cell transfection
MX9603866A (es) * 1994-03-18 1997-03-29 Boehringer Ingelheim Int Procedimiento para el tratamiento de celulas eucarioticas.
AU2194895A (en) * 1994-03-25 1995-10-17 Uab Research Foundation, The Composition and methods for creating syngeneic recombinant virus-producing cells
US5670347A (en) 1994-05-11 1997-09-23 Amba Biosciences Llc Peptide-mediated gene transfer
EP0767840A2 (de) 1994-05-30 1997-04-16 BOEHRINGER INGELHEIM INTERNATIONAL GmbH Verfahren zum einbringen von fremdmaterial in höhere eukaryotische zellen
DE4418965A1 (de) * 1994-05-31 1995-12-07 Boehringer Ingelheim Int Verfahren zum Einbringen von Nukleinsäure in höhere eukaryotische Zellen
DE4426429A1 (de) * 1994-07-26 1996-02-01 Boehringer Ingelheim Int Verfahren zum Einführen von DNA in höhere eukaryotische Zellen
US6468981B1 (en) 1994-07-29 2002-10-22 Emory University Compositions and methods for targeting pharmaceutically active materials to cells containing androgen receptors
US5525606A (en) * 1994-08-01 1996-06-11 The United States Of America As Represented By The Department Of Health And Human Services Substituted 06-benzylguanines and 6(4)-benzyloxypyrimidines
US5846782A (en) * 1995-11-28 1998-12-08 Genvec, Inc. Targeting adenovirus with use of constrained peptide motifs
US5962311A (en) * 1994-09-08 1999-10-05 Genvec, Inc. Short-shafted adenoviral fiber and its use
US5559099A (en) * 1994-09-08 1996-09-24 Genvec, Inc. Penton base protein and methods of using same
US5965541A (en) * 1995-11-28 1999-10-12 Genvec, Inc. Vectors and methods for gene transfer to cells
US6465253B1 (en) 1994-09-08 2002-10-15 Genvec, Inc. Vectors and methods for gene transfer to cells
US5837533A (en) * 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
WO1996010089A1 (fr) * 1994-09-29 1996-04-04 Ajinomoto Co., Inc. Modification d'un peptide et d'une proteine
US6221959B1 (en) 1994-11-18 2001-04-24 Supratek Pharma, Inc. Polynucleotide compositions
US6359054B1 (en) 1994-11-18 2002-03-19 Supratek Pharma Inc. Polynucleotide compositions for intramuscular administration
US6008202A (en) * 1995-01-23 1999-12-28 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5795587A (en) * 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5770442A (en) 1995-02-21 1998-06-23 Cornell Research Foundation, Inc. Chimeric adenoviral fiber protein and methods of using same
US6127525A (en) * 1995-02-21 2000-10-03 Cornell Research Foundation, Inc. Chimeric adenoviral coat protein and methods of using same
AU5103996A (en) * 1995-03-09 1996-10-02 Bavarian Nordic Research Institute A/S Vectors carrying therapeutic genes encoding antimicrobial peptides for gene therapy
WO1996029423A1 (en) * 1995-03-20 1996-09-26 Baylor College Of Medicine Compositions and methods for inducing infection by retroviral vectors outside of their host range
DE19510344C1 (de) * 1995-03-22 1996-11-07 Boehringer Ingelheim Int Verwendung einer Tumorvakzine
US6420549B1 (en) 1995-06-06 2002-07-16 Isis Pharmaceuticals, Inc. Oligonucleotide analogs having modified dimers
US20030069173A1 (en) * 1998-03-16 2003-04-10 Life Technologies, Inc. Peptide-enhanced transfections
CA2222550A1 (en) * 1995-06-07 1996-12-19 Baylor College Of Medicine Nucleic acid transporters for delivery of nucleic acids into a cell
US6051429A (en) * 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
EP0874910A4 (en) * 1995-06-07 1999-04-21 Life Technologies Inc ENHANCED CATIONIC LIPID TRANSFECTION BY PEPTIDES
SI0833934T2 (sl) * 1995-06-15 2013-04-30 Crucell Holland B.V. Pakirni sistemi za humani rekombinantni adenovirus za uporabo v genski terapiji
US6783980B2 (en) * 1995-06-15 2004-08-31 Crucell Holland B.V. Packaging systems for human recombinant adenovirus to be used in gene therapy
US5908777A (en) * 1995-06-23 1999-06-01 University Of Pittsburgh Lipidic vector for nucleic acid delivery
GB9515356D0 (en) * 1995-07-26 1995-09-20 Medical Res Council Improvements in or relating to delivery of nucleic acid
US5770720A (en) * 1995-08-30 1998-06-23 Barnes-Jewish Hospital Ubiquitin conjugating enzymes having transcriptional repressor activity
IL115199A (en) 1995-09-07 2005-05-17 Opperbas Holding Bv Composition comprising a polynucleic acid molecule in a liposome and method using said composition
CA2279669A1 (en) * 1995-12-15 1997-06-16 Enzo Therapeutics, Inc. Property effecting and/or property exhibiting constructs for the expression of non-native nucleic acid processing components for therapeutic and diagnostic uses
US6077834A (en) * 1996-02-09 2000-06-20 Cheng; Pi-Wan Receptor ligand-facilitated delivery of biologically active molecules
DE19605548A1 (de) * 1996-02-15 1997-09-04 Boehringer Ingelheim Int Zusammensetzung für die Transfektion höherer eukaryotischer Zellen
JP2000506865A (ja) * 1996-03-14 2000-06-06 ジ イミューン リスポンス コーポレイション インターフェロンをコードする遺伝子の標的を定めた送達
DE19615803A1 (de) 1996-04-20 1997-10-23 Boehringer Ingelheim Int CELO-Virus
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US7812149B2 (en) 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
DE19632532A1 (de) * 1996-08-13 1998-02-19 Boehringer Ingelheim Int Verfahren zur Herstellung von Säugetieren mit definierten genetischen Eigenschaften
WO1998006437A2 (en) * 1996-08-13 1998-02-19 Chiron Corporation Compositions and methods for polynucleotide delivery
US5948681A (en) * 1996-08-14 1999-09-07 Children's Hospital Of Philadelphia Non-viral vehicles for use in gene transfer
CN1181422A (zh) * 1996-10-31 1998-05-13 上海市肿瘤研究所 与生长因子受体结合的多肽所构建的基因转移载体
US6544523B1 (en) 1996-11-13 2003-04-08 Chiron Corporation Mutant forms of Fas ligand and uses thereof
US5965441A (en) * 1996-11-13 1999-10-12 The General Hospital Coporation HSV/AAV hybrid amplicon vectors
US6797276B1 (en) 1996-11-14 2004-09-28 The United States Of America As Represented By The Secretary Of The Army Use of penetration enhancers and barrier disruption agents to enhance the transcutaneous immune response
US20060002949A1 (en) 1996-11-14 2006-01-05 Army Govt. Of The Usa, As Rep. By Secretary Of The Office Of The Command Judge Advocate, Hq Usamrmc. Transcutaneous immunization without heterologous adjuvant
US5980898A (en) 1996-11-14 1999-11-09 The United States Of America As Represented By The U.S. Army Medical Research & Material Command Adjuvant for transcutaneous immunization
WO1998027209A1 (en) * 1996-12-18 1998-06-25 Emory University Polycationic oligomers
WO1998041240A1 (en) * 1997-03-14 1998-09-24 The Children's Hospital Of Philadelphia Methods and compositions for use in gene therapy for treatment of hemophilia
EP0981548A4 (en) * 1997-04-30 2005-11-23 Enzon Inc SINGLE CHAIN PROTEINS FIXING ANTIGENS CAPABLE OF GLYCOSYLATION, PRODUCTION AND USES THEREOF
US20040009166A1 (en) * 1997-04-30 2004-01-15 Filpula David R. Single chain antigen-binding polypeptides for polymer conjugation
US20050096288A1 (en) * 1997-06-13 2005-05-05 Aragene, Inc. Lipoproteins as nucleic acid vectors
US6635623B1 (en) 1997-06-13 2003-10-21 Baylor College Of Medicine Lipoproteins as nucleic acid vectors
DE19726186A1 (de) * 1997-06-20 1998-12-24 Boehringer Ingelheim Int Komplexe für den Transport von Nukleinsäure in höhere eukaryotische Zellen
US6172211B1 (en) 1997-07-11 2001-01-09 Boehringer Ingelheim International Gmbh Nucleic acid encoding tag7 polypeptide
AU8569698A (en) * 1997-07-11 1999-02-08 Brandeis University Method of inducing apoptosis by reducing the level of thiamin
US7923250B2 (en) 1997-07-30 2011-04-12 Warsaw Orthopedic, Inc. Methods of expressing LIM mineralization protein in non-osseous cells
CA2297489A1 (en) 1997-07-30 1999-02-11 Emory University Novel bone mineralization proteins, dna, vectors, expression systems
WO1999007723A1 (en) * 1997-08-07 1999-02-18 University Of Maryland, Baltimore Nucleic acid uptake and release vehicle
US6706693B1 (en) 1997-08-13 2004-03-16 The Uab Research Foundation Vaccination by topical application of genetic vectors
US6716823B1 (en) 1997-08-13 2004-04-06 The Uab Research Foundation Noninvasive genetic immunization, expression products therefrom, and uses thereof
US6197332B1 (en) * 1997-08-13 2001-03-06 Chiron Corporation Lipid-conjugated polyamide compounds and related compositions and methods thereof
US20030045492A1 (en) * 1997-08-13 2003-03-06 Tang De-Chu C. Vaccination by topical application of recombinant vectors
US6348450B1 (en) 1997-08-13 2002-02-19 The Uab Research Foundation Noninvasive genetic immunization, expression products therefrom and uses thereof
US20030125278A1 (en) * 1997-08-13 2003-07-03 Tang De-Chu C. Immunization of animals by topical applications of a salmonella-based vector
US5998386A (en) 1997-09-19 1999-12-07 Feldman; Arthur M. Pharmaceutical compositions and method of using same for the treatment of failing myocardial tissue
WO1999019500A1 (en) * 1997-10-10 1999-04-22 The Government Of The United States Of America, Represented By The Secretary, Department Of Healt H And Human Services Complex of biotinylated viral vector and ligand for targeted gene delivery
US6914131B1 (en) 1998-10-09 2005-07-05 Chiron S.R.L. Neisserial antigens
ATE476508T1 (de) 1997-11-06 2010-08-15 Novartis Vaccines & Diagnostic Neisseriale antigene
JP2001523468A (ja) 1997-11-14 2001-11-27 セダーシナイ メディカル センター トランスジェニック種の産生のための雄性生殖細胞のトランスフェクションおよび移入
US7294755B1 (en) 1997-11-14 2007-11-13 Cedars-Sinai Medical Center Genetic modification of male germ cells for generation of transgenic species and genetic therapies
US6734338B1 (en) 1997-11-14 2004-05-11 Cedars-Sinai Medical Center Transfection, storage and transfer of male germ cells for generation of transgenic species and genetic therapies
EP0945138A1 (en) * 1997-12-04 1999-09-29 Université Louis Pasteur de Strasbourg Transfection particles
US6835393B2 (en) * 1998-01-05 2004-12-28 University Of Washington Enhanced transport using membrane disruptive agents
EP2210945B1 (en) 1998-01-14 2013-06-26 Novartis Vaccines and Diagnostics S.r.l. Neisseria meningitidis antigens
AU4070499A (en) 1998-04-30 1999-11-16 Cornell Research Foundation Inc. Adenoviral vectors with tandem fiber proteins
NZ508366A (en) 1998-05-01 2004-03-26 Chiron Corp Neisseria meningitidis antigens and compositions
US7244714B1 (en) * 1998-06-12 2007-07-17 Aradigm Corporation Methods of delivering aerosolized polynucleotides to the respiratory tract
US6509323B1 (en) 1998-07-01 2003-01-21 California Institute Of Technology Linear cyclodextrin copolymers
US7091192B1 (en) 1998-07-01 2006-08-15 California Institute Of Technology Linear cyclodextrin copolymers
US6245427B1 (en) 1998-07-06 2001-06-12 DüZGüNES NEJAT Non-ligand polypeptide and liposome complexes as intracellular delivery vehicles
US20040043489A1 (en) * 1998-07-08 2004-03-04 Menzo Havenga Gene delivery vectors provided with a tissue tropism for dendritic cells and methods of use
US20030017138A1 (en) * 1998-07-08 2003-01-23 Menzo Havenga Chimeric adenoviruses
US7396919B1 (en) * 1998-07-17 2008-07-08 Mirus Bio Corporation Charge reversal of polyion complexes
AU8818398A (en) * 1998-08-21 2000-03-14 Felix Frey Conjugates of dna interacting groups with steroid hormones for use as nucleic acid transfection agent
AU767975B2 (en) 1998-09-11 2003-11-27 Genvec, Inc. Alternatively targeted adenovirus
US6077709A (en) 1998-09-29 2000-06-20 Isis Pharmaceuticals Inc. Antisense modulation of Survivin expression
EP1953229A3 (en) 1998-10-15 2008-12-24 Novartis Vaccines and Diagnostics, Inc. Metastatic breast and colon cancer regulated genes
US6333396B1 (en) 1998-10-20 2001-12-25 Enzon, Inc. Method for targeted delivery of nucleic acids
EP2298728A1 (en) 1998-11-12 2011-03-23 Life Technologies Corporation Transfection reagents
US20050063950A1 (en) * 1998-11-19 2005-03-24 Georgetown University Systemic viral/ligand gene delivery system and gene therapy
JP5448283B2 (ja) 1998-11-19 2014-03-19 ジョージタウン・ユニバーシティ 全身性ウイルス／リガンド遺伝子送達システムおよび遺伝子治療
US6929946B1 (en) 1998-11-20 2005-08-16 Crucell Holland B.V. Gene delivery vectors provided with a tissue tropism for smooth muscle cells, and/or endothelial cells
ATE519840T1 (de) 1998-12-16 2011-08-15 Novartis Vaccines & Diagnostic Menschliche cyclin-abhängige kinase (hpnqalre)
US7098192B2 (en) 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
GB9908195D0 (en) 1999-04-09 1999-06-02 Microbiological Res Authority Treatment of intracellular infection
US6514762B1 (en) 1999-04-23 2003-02-04 New Mexico State University Technology Transfer Corporation Delivery of nucleotides by electrochemical release
DE19918446A1 (de) * 1999-04-23 2000-11-23 Univ Eberhard Karls Verwendung von Coxsackievirus B3 zur Verbesserung der Transfektion von Zellen
US7368261B1 (en) 1999-04-30 2008-05-06 Novartis Vaccines And Diagnostics Srl Conserved Neisserial antigens
US20040009936A1 (en) * 1999-05-03 2004-01-15 Tang De-Chu C. Vaccine and drug delivery by topical application of vectors and vector extracts
US6913922B1 (en) * 1999-05-18 2005-07-05 Crucell Holland B.V. Serotype of adenovirus and uses thereof
US20050232900A1 (en) * 1999-05-18 2005-10-20 Crucell Holland B.V. Serotype of adenovirus and uses thereof
US6492169B1 (en) 1999-05-18 2002-12-10 Crucell Holland, B.V. Complementing cell lines
GB9911683D0 (en) 1999-05-19 1999-07-21 Chiron Spa Antigenic peptides
US6696272B1 (en) 1999-06-02 2004-02-24 Hsc Research & Development Limited Partnership Products and methods for gaucher disease therapy
US8541548B2 (en) * 1999-06-07 2013-09-24 Arrowhead Madison Inc. Compounds and methods for reversible modification of biologically active molecules
DK1102785T3 (da) * 1999-06-07 2013-05-13 Arrowhead Res Corp Sammensætninger til lægemiddeltilførsel ved anvendelse af pH-følsomme molekyler
US8137695B2 (en) * 2006-08-18 2012-03-20 Arrowhead Madison Inc. Polyconjugates for in vivo delivery of polynucleotides
US20080281041A1 (en) 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers
GB9916529D0 (en) 1999-07-14 1999-09-15 Chiron Spa Antigenic peptides
DE60017924T2 (de) * 1999-09-17 2006-03-30 Tgt Laboratories, S.A. De C.V. Rekombinante, adenovirale vektoren und ihre verwendung zur behandlung von leberzirrhose
MXPA02004283A (es) 1999-10-29 2002-10-17 Chiron Spa Peptidos antigenicos neisseriales.
JP2003519199A (ja) * 1999-12-30 2003-06-17 ノバルティス アクチエンゲゼルシャフト 遺伝子治療のための新規なコロイド合成ベクター
WO2001051092A2 (en) 2000-01-07 2001-07-19 University Of Washington Enhanced transport of agents using membrane disruptive agents
DK1248647T3 (da) 2000-01-17 2010-09-27 Novartis Vaccines & Diagnostic Ydre membranvesikel (OMV) vaccine omfattende N. meningitidis serogruppe B ydre membranproteiner
US6261840B1 (en) 2000-01-18 2001-07-17 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
US20020055479A1 (en) 2000-01-18 2002-05-09 Cowsert Lex M. Antisense modulation of PTP1B expression
CN1800385B (zh) 2000-02-28 2010-06-02 启龙有限公司 奈瑟球菌蛋白质的异源表达
US6680172B1 (en) 2000-05-16 2004-01-20 Regents Of The University Of Michigan Treatments and markers for cancers of the central nervous system
EP1157999A1 (en) * 2000-05-24 2001-11-28 Introgene B.V. Methods and means for enhancing skin transplantation using gene delivery vehicles having tropism for primary fibroblasts, as well as other uses thereof
EP1950297A2 (en) 2000-05-31 2008-07-30 Novartis Vaccines and Diagnostics, Inc. Compositions and methods for treating neoplastic disease using chemotherapy and radiation sensitizers
JP2003534806A (ja) * 2000-05-31 2003-11-25 ジェンベク、インコーポレイティッド アデノウイルスベクターのターゲティングの方法および組成物
DE10130849A1 (de) 2000-06-28 2002-06-06 Max Delbrueck Centrum Verfahren zur Verbesserung der Transfektionseffizienz
WO2002010219A1 (en) * 2000-07-26 2002-02-07 Cho, Sung-Woo Oligolysine transducing domain, oligolysine-cargo molecule complex and uses thereof
US7198924B2 (en) 2000-12-11 2007-04-03 Invitrogen Corporation Methods and compositions for synthesis of nucleic acid molecules using multiple recognition sites
EP1322774A2 (en) * 2000-09-20 2003-07-02 Crucell Holland B.V. Gene delivery vectors provided with a tissue tropism for dendritic cells
EP1191104A1 (en) * 2000-09-26 2002-03-27 Introgene B.V. Gene delivery vehicles and use thereof in the preparation of a medicament and/or vaccine
DE60138403D1 (de) * 2000-09-26 2009-05-28 Crucell Holland Bv Adenovirale vektoren für die übertragung von genen in zellen der skelettmuskulatur oder myoblasten
DE10049010A1 (de) * 2000-10-04 2002-04-18 Boehringer Ingelheim Int Transferrin-Polykation/DNS-Komplexe für die systemische Therapie von Tumorerkrankungen mit zytotoxischen Proteinen
MXPA03003690A (es) 2000-10-27 2004-05-05 Chiron Spa Acidos nucleicos y proteinas de los grupos a y b de estreptococos.
NZ525440A (en) * 2000-10-27 2005-03-24 Invitrogen Corp Method for introducing antisense oligonucleotides into eucaryotic cells using cationic lipids
US6892140B1 (en) * 2000-11-27 2005-05-10 Enteron, Inc. Immunogenic cancer peptides and uses thereof
MXPA00011713A (es) * 2000-11-28 2002-05-31 Tgt Lab S A De C V Vectores recombinantes virales y no virales conteniendo el gen humano del activador de plasminogeno derivado de urocinasa y su utilidad en el tratamiento de diversos tipos de fibrosis hepatica, renal, pulmonar, pancreatica, cardiaca y cicatrices hipe
US7635571B2 (en) * 2000-12-07 2009-12-22 Siemens Healthcare Diagnostics Products Gmbh Amplified signal in binding assays
US6635466B2 (en) * 2001-01-09 2003-10-21 University Of Iowa Research Foundation Adenovirus serotype 30 (Ad30)
AU2002240201A1 (en) * 2001-02-02 2002-08-19 Yale University Peptides for facilitating composite receptor expression and translocation of macromolecules
PT1372708E (pt) 2001-02-13 2008-09-29 Us Gov Sec Army Vacina para imunização transcutânea
US20020150566A1 (en) * 2001-03-23 2002-10-17 Kun-Liang Guan Method of inhibiting cancerous cell proliferation using Ras mutants of GDP-bound conformation
GB0107661D0 (en) 2001-03-27 2001-05-16 Chiron Spa Staphylococcus aureus
GB0107658D0 (en) 2001-03-27 2001-05-16 Chiron Spa Streptococcus pneumoniae
US20020193295A1 (en) * 2001-05-04 2002-12-19 Emanuel Calenoff Immunogenic peptides and uses thereof
JP2004535388A (ja) * 2001-04-30 2004-11-25 ターゲティッド ジェネティクス コーポレイション 脂質含有薬物送達複合体およびそれらの生成方法
WO2002089586A1 (en) * 2001-05-10 2002-11-14 St. Jude Children's Research Hospital Lung epithelial cell line for propagating viruses
US20060159657A1 (en) * 2001-06-14 2006-07-20 Macromed, Incorporated Formulations of lymphokines and method of use thereof for local or both local and systemic control of proliferative cell disorders
US20030003074A1 (en) * 2001-06-14 2003-01-02 Macromed, Inc. Formulations of lymphokines and method of use thereof for local or both local and systemic control of proliferative cell disorders
CA2633171C (en) 2001-06-20 2012-11-20 Genentech, Inc. Antibodies against tumor-associated antigenic target (tat) polypeptides
US7803915B2 (en) 2001-06-20 2010-09-28 Genentech, Inc. Antibody compositions for the diagnosis and treatment of tumor
CA2790034A1 (en) 2001-06-21 2003-01-03 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
US7425545B2 (en) 2001-07-25 2008-09-16 Isis Pharmaceuticals, Inc. Modulation of C-reactive protein expression
US6964950B2 (en) 2001-07-25 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of C-reactive protein expression
US20030096772A1 (en) 2001-07-30 2003-05-22 Crooke Rosanne M. Antisense modulation of acyl CoA cholesterol acyltransferase-2 expression
US7407943B2 (en) 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
US7227014B2 (en) 2001-08-07 2007-06-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein (a) expression
GB0120022D0 (en) * 2001-08-16 2001-10-10 Photobiotics Ltd Conjugate
NZ573831A (en) 2001-09-18 2010-07-30 Genentech Inc Compositions and methods for the diagnosis and treatment of tumor, particularly breast tumor - TAT193
AU2002365077A1 (en) * 2001-09-19 2003-06-30 Massachusetts Institute Of Technology Methods and products related to non-viral transfection
US6750019B2 (en) 2001-10-09 2004-06-15 Isis Pharmaceuticals, Inc. Antisense modulation of insulin-like growth factor binding protein 5 expression
NZ585001A (en) 2001-10-09 2011-08-26 Isis Pharmaceuticals Inc Antisense modulation of insulin-like growth factor binding protein 5 expression
US7745418B2 (en) * 2001-10-12 2010-06-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting viral replication
JP2005508396A (ja) * 2001-11-02 2005-03-31 イントラディグム、コーポレイション 核酸送達ビヒクルのための治療方法
EP1575976A4 (en) 2001-11-02 2006-08-23 Insert Therapeutics Inc METHODS AND COMPOSITIONS FOR THE THERAPEUTIC USE OF RNA INTERFERENCE
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
NZ562929A (en) 2001-12-12 2009-07-31 Novartis Vaccines & Diagnostic Immunisation against chlamydia trachomatis
KR20080106369A (ko) 2002-01-02 2008-12-04 제넨테크, 인크. 종양의 진단 및 치료 방법 및 이를 위한 조성물
US8138383B2 (en) * 2002-03-11 2012-03-20 Arrowhead Madison Inc. Membrane active heteropolymers
US8008355B2 (en) * 2002-03-11 2011-08-30 Roche Madison Inc. Endosomolytic poly(vinyl ether) polymers
WO2003078576A2 (en) * 2002-03-12 2003-09-25 Nitto Denko Corporation Vector for transfection of eukaryotic cells
AU2003267949A1 (en) * 2002-03-15 2003-12-31 U.S. Government As Represented By The Department Of Veterans Affairs Methods and compositions using cellular asialodeterminants and glycoconjugates for targeting cells to tissues and organs
US20030180712A1 (en) 2002-03-20 2003-09-25 Biostratum Ab Inhibition of the beta3 subunit of L-type Ca2+ channels
KR20040101502A (ko) 2002-04-16 2004-12-02 제넨테크, 인크. 종양의 진단 및 치료 방법 및 이를 위한 조성물
CA2477954C (en) * 2002-04-25 2012-07-10 Crucell Holland B.V. Means and methods for the production of adenovirus vectors
US20040142450A1 (en) * 2002-05-10 2004-07-22 Seo Sang Heui Lung epithelial cell line for propagating viruses
US7199107B2 (en) 2002-05-23 2007-04-03 Isis Pharmaceuticals, Inc. Antisense modulation of kinesin-like 1 expression
US20050233993A1 (en) * 2002-06-05 2005-10-20 Kadonaga James T Methods for promoting homologous recombination
DK1515752T3 (da) * 2002-06-19 2009-03-23 Univ Health Network ACE2-aktivering til behandling af hjerte-, lunge- og nyresygdomme og hypertension
US20060003316A1 (en) * 2002-07-15 2006-01-05 John Simard Immunogenic compositions derived from poxviruses and methods of using same
KR101476067B1 (ko) 2002-09-06 2014-12-23 인설트 테라페틱스, 인코퍼레이티드 공유결합된 치료제 전달을 위한 사이클로덱스트린-기초 중합체
WO2004022716A2 (en) * 2002-09-09 2004-03-18 University Of Tennessee Research Foundation Recombinatant mutants of rhabdovirus and methods of use thereof
CA2498777C (en) 2002-09-13 2015-01-13 Replicor, Inc. Non-sequence complementary antiviral oligonucleotides
US7229976B2 (en) 2002-09-26 2007-06-12 Isis Pharmaceuticals, Inc. Modulation of forkhead box O1A expression
BR0314471A (pt) * 2002-10-10 2005-08-02 Us Dept Veterans Affairs Detecção, localização e estadiamento de tumores usando linfócitos ativados classificados dirigidos a um epitopo especìfico do tumor
AU2003290598A1 (en) 2002-11-05 2004-06-03 Isis Pharmaceuticals, Inc. Modified oligonucleotides for use in rna interference
EP1560839A4 (en) 2002-11-05 2008-04-23 Isis Pharmaceuticals Inc CHIMERIC OLIGOMER COMPOUNDS AND THEIR USE IN GENE MODULATION
US20080026077A1 (en) * 2002-11-12 2008-01-31 John Hilfinger Methods and compositions of gene delivery agents for systemic and local therapy
US20050026859A1 (en) * 2002-11-12 2005-02-03 John Hilfinger Methods and compositions of gene delivery agents for systemic and local therapy
CA2505801A1 (en) 2002-11-13 2004-05-27 Rosanne Crooke Antisense modulation of apolipoprotein b expression
ES2420914T3 (es) 2002-11-13 2013-08-27 Genzyme Corporation Modulación antisentido de la expresión de la apolipoproteína B
US7144999B2 (en) 2002-11-23 2006-12-05 Isis Pharmaceuticals, Inc. Modulation of hypoxia-inducible factor 1 alpha expression
JP4753863B2 (ja) 2003-02-11 2011-08-24 アンチセンス セラピューティクス リミテッド インスリン様増殖因子ｉ受容体発現の調節
US7803781B2 (en) 2003-02-28 2010-09-28 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
US20040185559A1 (en) 2003-03-21 2004-09-23 Isis Pharmaceuticals Inc. Modulation of diacylglycerol acyltransferase 1 expression
US7217566B2 (en) * 2003-03-24 2007-05-15 Invitrogen Corporation Attached cell lines
GB0308198D0 (en) 2003-04-09 2003-05-14 Chiron Srl ADP-ribosylating bacterial toxin
US7598227B2 (en) 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
WO2004093788A2 (en) * 2003-04-17 2004-11-04 The Trustees Of Columbia University In The City Ofnew York Desmoglein 4 is a novel gene involved in hair growth
US7399853B2 (en) 2003-04-28 2008-07-15 Isis Pharmaceuticals Modulation of glucagon receptor expression
US20040220084A1 (en) * 2003-05-02 2004-11-04 Jasbir Sandhu Methods for nucleic acid delivery
US20040220085A1 (en) * 2003-05-02 2004-11-04 Jasbir Sandhu Compositions for nucleic acid delivery
CN1984921B (zh) 2003-06-03 2010-06-16 Isis药物公司 存活蛋白表达的调节
EP1639109B1 (en) * 2003-06-09 2011-04-13 Corixa Corporation Dna vectors
US7883720B2 (en) * 2003-07-09 2011-02-08 Wisconsin Alumni Research Foundation Charge-dynamic polymers and delivery of anionic compounds
CA2533701A1 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
US7425544B2 (en) 2003-09-18 2008-09-16 Eli Lilly And Company Modulation of eIF4E expression
SI1678194T1 (sl) 2003-10-10 2014-02-28 Alchemia Oncology Pty Limited Modulacija sinteze hialuronana in degradacija pri zdravljenju bolezni
EP2267154A1 (en) * 2003-10-23 2010-12-29 Illumigen Biosciences, Inc. Oligoadenylate synthetase
EP2418281B1 (en) 2003-10-24 2016-06-01 Gencia Corporation Methods and compositions for delivering polynucleotides
US8507277B2 (en) 2003-10-24 2013-08-13 Gencia Corporation Nonviral vectors for delivering polynucleotides
US8062891B2 (en) 2003-10-24 2011-11-22 Gencia Corporation Nonviral vectors for delivering polynucleotides to plants
US20090208478A1 (en) * 2003-10-24 2009-08-20 Gencia Corporation Transducible polypeptides for modifying metabolism
US8133733B2 (en) 2003-10-24 2012-03-13 Gencia Corporation Nonviral vectors for delivering polynucleotides to target tissues
US20090123468A1 (en) 2003-10-24 2009-05-14 Gencia Corporation Transducible polypeptides for modifying metabolism
US20050191653A1 (en) 2003-11-03 2005-09-01 Freier Susan M. Modulation of SGLT2 expression
NZ596981A (en) 2003-11-17 2013-10-25 Genentech Inc Compositions and methods for the treatment of tumor of hematopoietic origin
ATE469984T1 (de) 2003-12-01 2010-06-15 Life Technologies Corp Rekombinationsstellen enthaltende nukleinsäuremoleküle und verfahren zur verwendung davon
SG113562A1 (en) * 2004-01-12 2005-08-29 Agency Science Tech & Res Polyalkyleneimine-graft-biodegradable polymers for delivery of bioactive agents
EP1711606A2 (en) 2004-01-20 2006-10-18 Isis Pharmaceuticals, Inc. Modulation of glucocorticoid receptor expression
US7468431B2 (en) 2004-01-22 2008-12-23 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP2 expression
US20050227940A1 (en) * 2004-01-23 2005-10-13 City Of Hope Amplifying interfering RNA (RNAi) expression and effects
US8569474B2 (en) 2004-03-09 2013-10-29 Isis Pharmaceuticals, Inc. Double stranded constructs comprising one or more short strands hybridized to a longer strand
US20050203047A1 (en) * 2004-03-10 2005-09-15 Ulrich Thomann Delivery vectors for short interfering RNA, micro-RNA and antisense RNA
EP2700720A3 (en) 2004-03-15 2015-01-28 Isis Pharmaceuticals, Inc. Compositions and methods for optimizing cleavage of RNA by RNASE H
DK1730280T3 (en) * 2004-03-26 2019-02-04 Curis Inc RNA interference modulators for hedgehog signaling and applications thereof
US8268324B2 (en) 2004-03-29 2012-09-18 Galpharma Co., Ltd. Modified galectin 9 proteins and use thereof
US20050244869A1 (en) 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
US7884086B2 (en) 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
US8697139B2 (en) 2004-09-21 2014-04-15 Frank M. Phillips Method of intervertebral disc treatment using articular chondrocyte cells
US9315862B2 (en) 2004-10-05 2016-04-19 California Institute Of Technology Aptamer regulated nucleic acids and uses thereof
AU2005294265A1 (en) 2004-10-06 2006-04-20 University Of Rochester Treatment of pulmonary hypertension using an agent that inhibits a tissue factor pathway
US7833513B2 (en) 2004-12-03 2010-11-16 Rhode Island Hospital Treatment of Alzheimer's Disease
EP1856526A4 (en) 2005-01-20 2008-11-12 Univ Rochester THIOREDOXIN-INTERACTING PROTEIN (TXNIP) AS REGULATOR OF VASCULAR FUNCTION
ES2852549T3 (es) 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
JP2008531581A (ja) * 2005-02-23 2008-08-14 ユーエービー リサーチ ファウンデーション アルキル−グリコシドで増強されたワクチン接種
MX2007010922A (es) 2005-03-10 2007-10-12 Genentech Inc Metodos y composiciones para modular la integridad vascular.
DK1866414T3 (da) 2005-03-31 2012-04-23 Calando Pharmaceuticals Inc Inhibitorer af ribonukleotidreduktase-underenhed 2 og anvendelser deraf.
US8734851B2 (en) * 2005-04-29 2014-05-27 Wisconsin Alumni Research Foundation Localized delivery of nucleic acid by polyelectrolyte assemblies
UA95446C2 (ru) 2005-05-04 2011-08-10 Іллюміджен Байосайєнсіз, Інк. Мутаци в генах oas1
DE102005023993A1 (de) * 2005-05-20 2006-11-23 TransMIT Gesellschaft für Technologietransfer mbH Nicht virales Vektorsystem zum Transport von Nukleinsäure in die Lunge
WO2007062380A2 (en) 2005-11-21 2007-05-31 Isis Pharmaceuticals, Inc. Modulation of eif4e-bp2 expression
US20090068158A1 (en) * 2005-12-09 2009-03-12 Medin Jeffrey A Thymidylate kinase mutants and uses thereof
US20090317802A1 (en) * 2005-12-09 2009-12-24 Bhatia Sangeeta N Compositions and Methods to Monitor RNA Delivery to Cells
US20090074733A1 (en) * 2005-12-09 2009-03-19 Medin Jeffrey A Thymidylate kinase mutants and uses thereof
EP2428227B1 (en) 2006-01-26 2016-06-22 Ionis Pharmaceuticals, Inc. Compositions and their uses directed to Huntingtin
CA2638915A1 (en) * 2006-02-10 2007-08-23 The Regents Of The University Of California Transducible delivery of sirna by dsrna binding domain fusions to ptd/cpps
EP2495327B1 (en) 2006-03-03 2016-09-28 ProMIS Neurosciences Inc. Methods and compositions to treat and detect misfolded-SOD1 mediated diseases
WO2007109584A1 (en) * 2006-03-16 2007-09-27 University Of Washington Temperature-and ph-responsive polymer compositions
EP2614839A3 (en) 2006-04-05 2015-01-28 Genentech, Inc. Method for using BOC/CDO to modulate hedgehog signaling
WO2007125173A2 (en) 2006-05-03 2007-11-08 Baltic Technology Development, Ltd. Antisense agents combining strongly bound base - modified oligonucleotide and artificial nuclease
EP2054431B1 (en) 2006-06-09 2011-08-31 Novartis AG Conformers of bacterial adhesins
EP2046964B1 (en) * 2006-07-12 2018-07-04 The Regents of The University of California Transducible delivery of nucleic acids by reversible phosphotriester charge neutralization protecting groups
WO2008011473A2 (en) 2006-07-19 2008-01-24 Isis Pharmaceuticals, Inc. Compositions and their uses directed to hbxip
US8017109B2 (en) * 2006-08-18 2011-09-13 Roche Madison Inc. Endosomolytic poly(acrylate) polymers
JP5864077B2 (ja) 2006-09-08 2016-02-17 ロード アイランド ホスピタル アルコール誘発性肝疾患の治療、予防および回復
JP2010502719A (ja) 2006-09-08 2010-01-28 ロード アイランド ホスピタル アルコール誘発性脳疾患の治療、予防および回復
WO2008058291A2 (en) 2006-11-09 2008-05-15 California Institute Of Technology Modular aptamer-regulated ribozymes
EP2099496A2 (en) * 2006-12-08 2009-09-16 Massachusetts Institute of Technology Delivery of nanoparticles and/or agents to cells
EP2118118B1 (en) * 2007-01-19 2017-09-27 Exiqon A/S Mediated cellular delivery of lna oligonucleotides
US8834918B2 (en) * 2007-01-22 2014-09-16 Wisconsin Alumni Research Foundation Modified multilayered film
JP2010516625A (ja) 2007-01-24 2010-05-20 インサート セラピューティクス， インコーポレイテッド 制御された薬物送達のためのテザー基を有するポリマー−薬物コンジュゲート
EP2641971A1 (en) 2007-01-29 2013-09-25 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
WO2008103962A2 (en) 2007-02-22 2008-08-28 Genentech, Inc. Methods for detecting inflammatory bowel disease
US7981688B2 (en) 2007-03-08 2011-07-19 University Of Washington Stimuli-responsive magnetic nanoparticles and related methods
US8673859B2 (en) 2007-03-20 2014-03-18 New York University GM-CSF cosmeceutical compositions and methods of use thereof
CA2584494A1 (en) * 2007-03-27 2008-09-27 Jeffrey A. Medin Vector encoding therapeutic polypeptide and safety elements to clear transduced cells
US8765462B2 (en) 2007-05-04 2014-07-01 University Health Network IL-12 immunotherapy for cancer
US20090082217A1 (en) * 2007-07-16 2009-03-26 California Institute Of Technology Selection of nucleic acid-based sensor domains within nucleic acid switch platform
US20120165387A1 (en) 2007-08-28 2012-06-28 Smolke Christina D General composition framework for ligand-controlled RNA regulatory systems
US8367815B2 (en) * 2007-08-28 2013-02-05 California Institute Of Technology Modular polynucleotides for ligand-controlled regulatory systems
US8865667B2 (en) * 2007-09-12 2014-10-21 California Institute Of Technology Higher-order cellular information processing devices
JP2010539978A (ja) 2007-10-02 2010-12-24 アムジェン インコーポレイテッド マイクロ−ｒｎａおよびその前駆体にハイブリダイズしうる核酸を用いるエリスロポエチンの増加
WO2009049100A2 (en) * 2007-10-09 2009-04-16 Wisconsin Alumni Research Foundation Ultrathin multilayered films for controlled release of anionic reagents
US9029524B2 (en) * 2007-12-10 2015-05-12 California Institute Of Technology Signal activated RNA interference
EP2077119A1 (de) * 2007-12-21 2009-07-08 Apeiron Biologics Forschungs- und Entwicklungsgesellschaft M.B.H. Behandlung von Fibrosen und Lebererkrankungen
US8344116B2 (en) * 2008-03-17 2013-01-01 Case Western Reserve University Polymers and complexes for delivery of nucleic acids to intracellular targets
WO2009114942A1 (en) 2008-03-20 2009-09-24 University Health Network Thymidylate kinase fusions and uses thereof
CN102037123A (zh) 2008-04-04 2011-04-27 卡兰多制药股份有限公司 Epas1抑制剂的组合物和用途
GB2459436A (en) * 2008-04-08 2009-10-28 Henderson Morley Plc Vaccine adjuvant
US8324333B2 (en) * 2008-06-05 2012-12-04 Wisconsin Alumni Research Foundation Anionic charge-dynamic polymers for release of cationic agents
WO2010008582A2 (en) 2008-07-18 2010-01-21 Rxi Pharmaceuticals Corporation Phagocytic cell drug delivery system
CN103429270B (zh) 2008-08-25 2016-11-23 埃克斯雷德制药有限公司 阻止结缔组织生长因子的反义核苷酸及其用途
AU2009293658A1 (en) 2008-09-22 2010-03-25 James Cardia Reduced size self-delivering RNAi compounds
MX2011005910A (es) 2008-12-04 2011-06-17 Opko Curna Llc Tratamiento de enfermedades relacionadas con eritropoyetina (epo) mediante inhibicion del transcrito antisentido natural a eritropoyetina.
US20110294870A1 (en) 2008-12-04 2011-12-01 Opko Curna, Llc Treatment of tumor suppressor gene related diseases by inhibition of natural antisense transcript to the gene
CN108042560A (zh) 2008-12-04 2018-05-18 库尔纳公司 通过抑制针对沉默调节蛋白1的天然反义转录物来治疗沉默调节蛋白1相关的疾病
WO2010078536A1 (en) 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
KR101682735B1 (ko) 2009-02-12 2016-12-06 큐알엔에이, 인크. 뇌 유래된 신경영양성 인자 (ｂｄｎｆ)에 대한 자연 안티센스 전사체의 저해에 의한 뇌 유래된 신경영양성 인자 (ｂｄｎｆ) 관련된 질환의 치료
EP2396408B1 (en) 2009-02-12 2017-09-20 CuRNA, Inc. Treatment of glial cell derived neurotrophic factor (gdnf) related diseases by inhibition of natural antisense transcript to gdnf
US8329882B2 (en) 2009-02-18 2012-12-11 California Institute Of Technology Genetic control of mammalian cells with synthetic RNA regulatory systems
CA2754749C (en) 2009-03-04 2019-04-30 Opko Curna, Llc Treatment of sirtuin 1 (sirt1) related diseases by inhibition of natural antisense transcript to sirt1
WO2010107733A2 (en) 2009-03-16 2010-09-23 Curna, Inc. Treatment of nuclear factor (erythroid-derived 2)-like 2 (nrf2) related diseases by inhibition of natural antisense transcript to nrf2
CN102549159B (zh) 2009-03-17 2016-08-10 库尔纳公司 通过抑制针对δ样1同源物(DLK1)的天然反义转录物来治疗DLK1相关的疾病
WO2010107991A2 (en) 2009-03-18 2010-09-23 Government Of The United States Of America As Represented By The Secretary, Department Of Health And Human Services Use of ixolaris, a tissue factor inhibitor, for the treatment and prevention of cancer
US9145555B2 (en) 2009-04-02 2015-09-29 California Institute Of Technology Integrated—ligand-responsive microRNAs
EP3524275A1 (en) 2009-04-22 2019-08-14 Massachusetts Institute Of Technology Innate immune supression enables repeated delivery of long rna molecules
US8318690B2 (en) 2009-05-01 2012-11-27 Curna, Inc. Treatment of hemoglobin (HBF/HBG) related diseases by inhibition of natural antisense transcript to HBF/HBG
KR101835889B1 (ko) 2009-05-06 2018-03-08 큐알엔에이, 인크. 지질 수송 및 대사 유전자에 대한 천연 안티센스 전사체의 억제에 의해 지질 수송 및 대사 유전자 관련된 질환의 치료
CA2761142C (en) 2009-05-06 2021-06-08 Opko Curna, Llc Treatment of tristetraproline (ttp) related diseases by inhibition of natural antisense transcript to ttp
DK2432881T3 (en) 2009-05-18 2018-02-26 Curna Inc TREATMENT OF REPROGRAMMING FACTOR-RELATED DISEASES BY INHIBITING NATURAL ANTISENSE TRANSCRIPTS TO A REPROGRAMMING FACTOR
WO2010135695A2 (en) 2009-05-22 2010-11-25 Curna, Inc. TREATMENT OF TRANSCRIPTION FACTOR E3 (TFE3) and INSULIN RECEPTOR SUBSTRATE 2 (IRS2) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO TFE3
CN103221541B (zh) 2009-05-28 2017-03-01 库尔纳公司 通过抑制抗病毒基因的天然反义转录物来治疗抗病毒基因相关疾病
CA2765099A1 (en) 2009-06-10 2010-12-16 New York University Phosphorylated tau peptide for use in the treatment of tauopathy
US8426214B2 (en) * 2009-06-12 2013-04-23 University Of Washington System and method for magnetically concentrating and detecting biomarkers
JP5944311B2 (ja) 2009-06-16 2016-07-05 クルナ・インコーポレーテッド コラーゲン遺伝子に対する天然アンチセンス転写物の抑制によるコラーゲン遺伝子関連疾患の治療
ES2629339T3 (es) 2009-06-16 2017-08-08 Curna, Inc. Tratamiento de enfermedades relacionadas con la paraoxonasa 1 (pon1) por inhibición de transcrito antisentido natural a pon1
CA2765889A1 (en) 2009-06-24 2010-12-29 Opko Curna, Llc Treatment of tumor necrosis factor receptor 2 (tnfr2) related diseases by inhibition of natural antisense transcript to tnfr2
KR101807324B1 (ko) 2009-06-26 2017-12-08 큐알엔에이, 인크. 다운 증후군 유전자에 대한 천연 안티센스 전사체의 억제에 의한 다운 증후군 유전자 관련된 질환의 치료
WO2011017516A2 (en) 2009-08-05 2011-02-10 Curna, Inc. Treatment of insulin gene (ins) related diseases by inhibition of natural antisense transcript to an insulin gene (ins)
EP2470657B1 (en) 2009-08-25 2019-10-23 CuRNA, Inc. Treatment of 'iq motif containing gtpase activating protein' (iqgap) related diseases by inhibition of natural antisense transcript to iqgap
US9321823B2 (en) 2009-09-02 2016-04-26 Genentech, Inc. Mutant smoothened and methods of using the same
WO2011046983A2 (en) 2009-10-12 2011-04-21 Smith Holdings, Llc Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro
WO2011050194A1 (en) 2009-10-22 2011-04-28 Genentech, Inc. Methods and compositions for modulating hepsin activation of macrophage-stimulating protein
WO2011052804A1 (en) * 2009-10-30 2011-05-05 Tokyo University Of Science Educational Foundation Administrative Organization Method of delivering agent into target cell
WO2011056234A1 (en) 2009-11-06 2011-05-12 Fibrogen, Inc. Treatment for radiation-induced disorders
US20110117668A1 (en) * 2009-11-09 2011-05-19 University Of Washington Through Its Center For Commercialization Self-powered smart diagnostic devices
US9080933B2 (en) 2009-11-09 2015-07-14 University Of Washington Through Its Center For Commercialization Stimuli-responsive polymer diagnostic assay comprising magnetic nanoparticles and capture conjugates
EP2503888A4 (en) * 2009-11-23 2015-07-29 Cerulean Pharma Inc POLYMERS ON CYCLODEXTRINBASIS FOR THERAPEUTIC ADMINISTRATION
CN103724432B (zh) 2009-11-30 2016-06-08 霍夫曼-拉罗奇有限公司 治疗和诊断表达slc34a2(tat211=seqid2)的肿瘤的抗体
JP2013513588A (ja) 2009-12-11 2013-04-22 ジーンコード エーエス Ｇｄｎｆファミリーリガンド（ｇｆｌ）のミメティックまたはｒｅｔシグナル経路活性化因子を用いた、神経系細胞の生存を容易にするための方法
CA2782366A1 (en) 2009-12-16 2011-07-14 Opko Curna, Llc Treatment of membrane bound transcription factor peptidase, site 1 (mbtps1) related diseases by inhibition of natural antisense transcript to mbtps1
WO2011079261A2 (en) 2009-12-23 2011-06-30 Curna, Inc. Treatment of hepatocyte growth factor (hgf) related diseases by inhibition of natural antisense transcript to hgf
US9068183B2 (en) 2009-12-23 2015-06-30 Curna, Inc. Treatment of uncoupling protein 2 (UCP2) related diseases by inhibition of natural antisense transcript to UCP2
EP2519633B1 (en) 2009-12-29 2017-10-25 CuRNA, Inc. Treatment of nuclear respiratory factor 1 (nrf1) related diseases by inhibition of natural antisense transcript to nrf1
RU2611186C2 (ru) 2009-12-29 2017-02-21 Курна, Инк. ЛЕЧЕНИЕ ЗАБОЛЕВАНИЙ, СВЯЗАННЫХ С ОПУХОЛЕВЫМ БЕЛКОМ 63 (р63), ПУТЕМ ИНГИБИРОВАНИЯ ПРИРОДНОГО АНТИСМЫСЛОВОГО ТРАНСКРИПТА К р63
US8946181B2 (en) 2010-01-04 2015-02-03 Curna, Inc. Treatment of interferon regulatory factor 8 (IRF8) related diseases by inhibition of natural antisense transcript to IRF8
WO2011085066A2 (en) 2010-01-06 2011-07-14 Curna, Inc. Treatment of pancreatic developmental gene related diseases by inhibition of natural antisense transcript to a pancreatic developmental gene
EP2524039B1 (en) 2010-01-11 2017-11-29 CuRNA, Inc. Treatment of sex hormone binding globulin (shbg) related diseases by inhibition of natural antisense transcript to shbg
US8883739B2 (en) 2010-01-19 2014-11-11 The Trustees Of Columbia University In The City Of New York Osteocalcin as a treatment for male reproductive disorders
RU2611192C2 (ru) 2010-01-25 2017-02-21 Курна, Инк. ЛЕЧЕНИЕ ЗАБОЛЕВАНИЙ, СВЯЗАННЫХ С РНКазой Н1, ПУТЕМ ИНГИБИРОВАНИЯ ПРИРОДНОГО АНТИСМЫСЛОВОГО ТРАНСКРИПТА К РНКазе Н1
KR101838308B1 (ko) 2010-02-22 2018-03-13 큐알엔에이, 인크. 피롤린-5-카르복실레이트 환원효소 1(ｐｙｃｒ1)에 대한 천연 안티센스 전사체의 억제에 의한 ｐｙｃｒ1과 관련된 질환의 치료
EP2538981B1 (en) 2010-02-23 2017-12-20 F. Hoffmann-La Roche AG Compositions and methods for the diagnosis and treatment of tumor
MX2012009178A (es) 2010-02-24 2012-11-30 Arrowhead Res Corp Composiciones para liberacion dirigida de arnsi.
RU2610661C2 (ru) 2010-04-09 2017-02-14 Курна, Инк. Лечение заболеваний, связанных с фактором роста фибробластов 21 (fgf21), путем ингибирования природного антисмыслового транскрипта к fgf21
US8362207B2 (en) 2010-04-16 2013-01-29 Wake Forest University Health Sciences Multi-level specific targeting of cancer cells with IL-13
RS56011B1 (sr) 2010-04-29 2017-09-29 Ionis Pharmaceuticals Inc Modulacija ekspresije transtiretina
WO2011139985A1 (en) 2010-05-03 2011-11-10 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
RU2693462C2 (ru) 2010-05-03 2019-07-03 Курна, Инк. Лечение заболеваний, связанных с сиртуином (sirt), путем ингибирования природного антисмыслового транскрипта к сиртуину (sirt)
EP2569430B1 (en) 2010-05-12 2018-10-17 The Trustees of Columbia University in the City of New York Methods for producing enteroendocrine cells that make and secrete insulin
TWI586356B (zh) 2010-05-14 2017-06-11 可娜公司 藉由抑制ｐａｒ４天然反股轉錄本治療ｐａｒ４相關疾病
EP2576783B1 (en) 2010-05-26 2017-11-29 CuRNA, Inc. Treatment of atonal homolog 1 (atoh1) related diseases by inhibition of natural antisense transcript to atoh1
US9057067B2 (en) 2010-07-10 2015-06-16 Kinki University Method for transfecting nucleic acid to cell and nucleic acid complex
JP5998131B2 (ja) 2010-07-14 2016-09-28 カッパーアールエヌエー，インコーポレイテッド Ｄｉｓｃｓｌａｒｇｅｈｏｍｏｌｏｇ（ｄｌｇ）ｄｌｇ１への天然アンチセンス転写物の阻害によるｄｌｇ関連疾患の治療
EP2625197B1 (en) 2010-10-05 2016-06-29 Genentech, Inc. Mutant smoothened and methods of using the same
ES2640755T3 (es) 2010-10-06 2017-11-06 Curna, Inc. Tratamiento de enfermedades relacionadas con la Sialidasa 4 (neu4) mediante inhibición del transcrito antisentido natural al gen neu4
EP2630241B1 (en) 2010-10-22 2018-10-17 CuRNA, Inc. Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
DK2633052T3 (en) 2010-10-27 2018-07-16 Curna Inc TREATMENT OF INTERFERON-RELATED DEVELOPMENT REGULATOR 1 (IFRD1) -RELATED DISEASES BY INHIBITION OF NATURAL ANTISENCE TRANSCRIPT TO IFRD1
WO2012061811A2 (en) 2010-11-05 2012-05-10 Fibrogen, Inc. Treatment method for lung remodeling diseases
JP6076258B2 (ja) 2010-11-12 2017-02-08 ジョージタウン ユニヴァーシティ 上皮細胞の不死化および使用法
US8987225B2 (en) 2010-11-23 2015-03-24 Curna, Inc. Treatment of NANOG related diseases by inhibition of natural antisense transcript to NANOG
EP2658567A4 (en) 2010-12-28 2014-09-24 Univ Rochester METHOD FOR MODIFYING INSULIN SIGNAL TRANSMISSION BY BILIVERDINREDUCTASE (BVR) AND BVR-DERIVED PEPTIDES
CA2825059A1 (en) 2011-02-02 2012-08-09 Excaliard Pharmaceuticals, Inc. Method of treating keloids or hypertrophic scars using antisense compounds targeting connective tissue growth factor (ctgf)
WO2012109495A1 (en) 2011-02-09 2012-08-16 Metabolic Solutions Development Company, Llc Cellular targets of thiazolidinediones
JP5850915B2 (ja) 2011-03-14 2016-02-03 国立大学法人北海道大学 肺送達のためのベクター、導入剤及び使用
CA2832972C (en) 2011-04-13 2019-04-30 Isis Pharmaceuticals, Inc. Antisense modulation of ptp1b expression
ES2653247T3 (es) 2011-06-09 2018-02-06 Curna, Inc. Tratamiento de enfermedades relacionadas con la frataxina (FXN) mediante inhibición del transcrito antisentido natural al gen FXN
CN103597074A (zh) 2011-06-16 2014-02-19 Isis制药公司 成纤维细胞生长因子受体4表达的反义调节
LT2753346T (lt) 2011-09-07 2020-08-10 Mount Sinai School Of Medicine Ceramidazė ir ląstelių diferenciacija
ES2673721T3 (es) 2011-09-20 2018-06-25 Ionis Pharmaceuticals, Inc. Modulación antisentido de la expresión de GCGR
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
WO2013063313A1 (en) 2011-10-25 2013-05-02 Isis Pharmaceuticals, Inc. Antisense modulation of gccr expression
US20150031750A1 (en) 2012-03-15 2015-01-29 The Scripps Research Institute Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
JP2015529073A (ja) 2012-08-20 2015-10-05 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物可逆性基を有するポリヌクレオチド
EP2943194A1 (en) 2012-09-17 2015-11-18 Chemedest Ltd. Treatment of peripheral neuropathy using gfr(alpha)3 type receptor agonists
US20140094432A1 (en) 2012-10-02 2014-04-03 Cerulean Pharma Inc. Methods and systems for polymer precipitation and generation of particles
US9694050B2 (en) 2012-10-21 2017-07-04 University Of Rochester THY1 (CD90) as a novel therapy to control adipose tissue accumulation
CN105051204B (zh) 2012-11-16 2023-07-21 波赛伊达治疗学股份有限公司 位点特异性酶和使用方法
US10052364B2 (en) 2013-03-15 2018-08-21 The Trustees Of Columbia University In The City Of New York Osteocalcin as a treatment for cognitive disorders
EP2992112B1 (en) 2013-04-22 2020-06-03 Icahn School of Medicine at Mount Sinai Mutations in pdgfrb and notch3 as causes of autosomal dominant infantile myofibromatosis
EP3007704B1 (en) 2013-06-13 2021-01-06 Antisense Therapeutics Ltd Combination therapy for acromegaly
EP3041935A1 (en) 2013-09-05 2016-07-13 Sage Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
EP3047023B1 (en) 2013-09-19 2019-09-04 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Compositions and methods for inhibiting jc virus (jcv)
EP3068797B1 (en) 2013-11-11 2020-01-08 Wake Forest University Health Sciences Constructs for multi-valent targeting of tumors
WO2015116902A1 (en) 2014-01-31 2015-08-06 Genentech, Inc. G-protein coupled receptors in hedgehog signaling
CA2937539A1 (en) 2014-02-04 2015-08-13 Genentech, Inc. Mutant smoothened and methods of using the same
WO2015171918A2 (en) 2014-05-07 2015-11-12 Louisiana State University And Agricultural And Mechanical College Compositions and uses for treatment thereof
AU2015260725B2 (en) 2014-05-14 2021-02-25 Targimmune Therapeutics Ag Improved polyethyleneimine polyethyleneglycol vectors
EP3145550B1 (en) * 2014-05-19 2019-03-27 BioNTech AG Particles comprising protamine and rna in combination with endosome destabilizing agents
EP3160503B1 (en) 2014-06-26 2021-02-17 The Trustees of Columbia University in the City of New York Inhibition of serotonin expression in gut enteroendocrine cells results in conversion to insulin-positive cells
US10195280B2 (en) 2014-07-15 2019-02-05 Life Technologies Corporation Compositions and methods for efficient delivery of molecules to cells
WO2016033424A1 (en) 2014-08-29 2016-03-03 Genzyme Corporation Methods for the prevention and treatment of major adverse cardiovascular events using compounds that modulate apolipoprotein b
US11191811B2 (en) 2014-11-19 2021-12-07 The Trustees Of Columbia University In The City Of New York Osteocalcin as a treatment for frailty associated with aging
AU2015364508A1 (en) 2014-12-18 2017-07-06 Alnylam Pharmaceuticals, Inc. ReversirTM compounds
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
WO2016196670A1 (en) 2015-06-01 2016-12-08 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in type vii collagen
US11473082B2 (en) 2015-06-17 2022-10-18 Poseida Therapeutics, Inc. Compositions and methods for directing proteins to specific loci in the genome
US10954300B2 (en) 2015-09-28 2021-03-23 The Trustees Of Columbia University In The City Of New York Use of pentoxifylline with immune checkpoint-blockade therapies for the treatment of melanoma
BR112018007066A2 (pt) 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
WO2017079442A1 (en) 2015-11-04 2017-05-11 Icahn School Of Medicine At Mount Sinai Methods of treating tumors and cancer, and identifying candidate subjects for such treatment
CA3007424A1 (en) 2015-11-05 2017-05-11 Children's Hospital Los Angeles "mobilizing leukemia cells"
EP3411396A1 (en) 2016-02-04 2018-12-12 Curis, Inc. Mutant smoothened and methods of using the same
JP7033547B2 (ja) 2016-04-18 2022-03-10 サレプタ セラピューティクス， インコーポレイテッド 酸性アルファ－グルコシダーゼ遺伝子に関連する疾患を処置するためのアンチセンスオリゴマーおよびそれを用いる方法
US11246868B2 (en) 2016-04-26 2022-02-15 Icahn School Of Medicine At Mount Sinai Treatment of hippo pathway mutant tumors and methods of identifying subjects as candidates for treatment
US10994025B2 (en) 2017-05-12 2021-05-04 Massachusetts Institute Of Technology Argonaute protein-double stranded RNA complexes and uses related thereto
WO2019006455A1 (en) 2017-06-30 2019-01-03 Solstice Biologics, Ltd. AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF
WO2019079637A2 (en) 2017-10-18 2019-04-25 Sarepta Therapeutics, Inc. ANTISENSE OLIGOMERIC COMPOUNDS
WO2019126578A1 (en) 2017-12-20 2019-06-27 Poseida Therapeutics, Inc. Compositions and methods for directing proteins to specific loci in the genome
US20210155959A1 (en) 2018-04-06 2021-05-27 Children's Medical Center Corporation Compositions and methods for somatic cell reprogramming and modulating imprinting
CA3214540A1 (en) 2021-04-22 2022-10-27 Pasi A. Janne Compositions and methods for treating cancer

Family Cites Families (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IN165717B (sl) * 1986-08-07 1989-12-23 Battelle Memorial Institute
US5166320A (en) * 1987-04-22 1992-11-24 University Of Connecticut Carrier system and method for the introduction of genes into mammalian cells
US5428132A (en) * 1987-10-11 1995-06-27 United States Of America Conjugate and method for integration of foreign DNA into cells
US5192553A (en) * 1987-11-12 1993-03-09 Biocyte Corporation Isolation and preservation of fetal and neonatal hematopoietic stem and progenitor cells of the blood and methods of therapeutic use
US5149782A (en) * 1988-08-19 1992-09-22 Tanox Biosystems, Inc. Molecular conjugates containing cell membrane-blending agents
US5354844A (en) * 1989-03-16 1994-10-11 Boehringer Ingelheim International Gmbh Protein-polycation conjugates
US5108921A (en) * 1989-04-03 1992-04-28 Purdue Research Foundation Method for enhanced transmembrane transport of exogenous molecules
US5240846A (en) * 1989-08-22 1993-08-31 The Regents Of The University Of Michigan Gene therapy vector for cystic fibrosis
JPH03127622A (ja) * 1989-10-09 1991-05-30 Green Cross Corp:The ｐＨ感受性リポソーム
DE59106279D1 (de) * 1990-05-18 1995-09-21 Genentech Inc Neue protein-polykation-konjugate.
AU660629B2 (en) * 1990-10-01 1995-07-06 University Of Connecticut, The Targeting viruses and cells for selective internalization by cells
AU1922092A (en) * 1991-05-03 1992-12-21 Albert Einstein College Of Medicine Of Yeshiva University Targeted delivery of genes encoding cell surface receptors
ATE199647T1 (de) * 1991-05-14 2001-03-15 Univ Connecticut Gerichtete abgabe von genen, die immunogene proteine kodieren
WO1992022635A1 (en) * 1991-06-05 1992-12-23 University Of Connecticut Targeted delivery of genes encoding secretory proteins
KR100252547B1 (ko) * 1991-09-05 2000-09-01 프레드 마리얀스키 폴리-또는 올리고누클레오티드의 세포로의 표적화된 전달
US5521291A (en) * 1991-09-30 1996-05-28 Boehringer Ingelheim International, Gmbh Conjugates for introducing nucleic acid into higher eucaryotic cells
NZ244306A (en) * 1991-09-30 1995-07-26 Boehringer Ingelheim Int Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation
US5981273A (en) 1991-09-30 1999-11-09 Boehringer Ingelheim Int'l. Gmbh Composition comprising an endosomolytic agent for introducing nucleic acid complexes into higher eucaryotic cells
US5225182A (en) * 1991-10-31 1993-07-06 Sharma Yash P Vectored drug delivery system using a cephaloplastin linking agent and a methed of using the system
US5922859A (en) 1992-02-01 1999-07-13 Boehringer Ingelheim International Gmbh Complexes containing nucleic acid which can be taken-up by endocytosis into higher eukaryotic cells
US5583020A (en) * 1992-11-24 1996-12-10 Ribozyme Pharmaceuticals, Inc. Permeability enhancers for negatively charged polynucleotides
WO1994017832A1 (en) 1993-02-09 1994-08-18 The Scripps Research Institute Targeting and delivery of genes and antiviral agents into cells by the adenovirus penton
DE4311651A1 (de) 1993-04-08 1994-10-13 Boehringer Ingelheim Int Virus für den Transport von Fremd-DNA in höhere eukaryotische Zellen
DE4335025A1 (de) 1993-10-14 1995-04-20 Boehringer Ingelheim Int Endosomolytisch wirksame Partikel
US5928944A (en) 1994-02-04 1999-07-27 The United States Of America As Represented By The Department Of Health And Human Services Method of adenoviral-medicated cell transfection

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WO1993007283A1 (de) 1993-04-15
US6077663A (en) 2000-06-20
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EP0545016A1 (de) 1993-06-09
US5547932A (en) 1996-08-20
ES2173083T3 (es) 2002-10-16
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CA2118816C (en) 2003-06-17
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BR9206559A (pt) 1994-11-08
ATE215990T1 (de) 2002-04-15
EP0607206A1 (de) 1994-07-27
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