WO1997004748B1 - Enveloppes virales artificielles renforcees pour l'apport de substances therapeutiques dans les cellules - Google Patents
Enveloppes virales artificielles renforcees pour l'apport de substances therapeutiques dans les cellulesInfo
- Publication number
- WO1997004748B1 WO1997004748B1 PCT/US1996/012750 US9612750W WO9704748B1 WO 1997004748 B1 WO1997004748 B1 WO 1997004748B1 US 9612750 W US9612750 W US 9612750W WO 9704748 B1 WO9704748 B1 WO 9704748B1
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- liposome
- polynucleotide
- component
- bifunctional
- tissue targeting
- Prior art date
Links
- 239000000126 substance Substances 0.000 title claims abstract 7
- 210000004779 membrane envelope Anatomy 0.000 title claims abstract 4
- 230000001225 therapeutic Effects 0.000 title abstract 3
- 230000001413 cellular Effects 0.000 title 1
- 229920000023 polynucleotide Polymers 0.000 claims abstract 23
- 239000002157 polynucleotide Substances 0.000 claims abstract 23
- 150000002632 lipids Chemical class 0.000 claims abstract 18
- 210000001519 tissues Anatomy 0.000 claims abstract 14
- 210000004027 cells Anatomy 0.000 claims abstract 11
- 230000000799 fusogenic Effects 0.000 claims abstract 9
- 238000001415 gene therapy Methods 0.000 claims abstract 4
- 150000007523 nucleic acids Chemical class 0.000 claims abstract 4
- 108090000623 proteins and genes Proteins 0.000 claims abstract 4
- 102000004169 proteins and genes Human genes 0.000 claims abstract 4
- 239000002502 liposome Substances 0.000 claims 26
- 230000001588 bifunctional Effects 0.000 claims 11
- 229920001184 polypeptide Polymers 0.000 claims 6
- 150000001413 amino acids Chemical class 0.000 claims 5
- 239000003599 detergent Substances 0.000 claims 5
- HVYWMOMLDIMFJA-DPAQBDIFSA-N (3β)-Cholest-5-en-3-ol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 4
- 239000008194 pharmaceutical composition Substances 0.000 claims 4
- QZAYGJVTTNCVMB-UHFFFAOYSA-N serotonin Chemical compound C1=C(O)C=C2C(CCN)=CNC2=C1 QZAYGJVTTNCVMB-UHFFFAOYSA-N 0.000 claims 4
- 238000000034 method Methods 0.000 claims 3
- 239000002245 particle Substances 0.000 claims 3
- 230000003612 virological Effects 0.000 claims 3
- 229940107161 Cholesterol Drugs 0.000 claims 2
- 210000002889 Endothelial Cells Anatomy 0.000 claims 2
- 210000004072 Lung Anatomy 0.000 claims 2
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 2
- 229940076279 Serotonin Drugs 0.000 claims 2
- 241000700605 Viruses Species 0.000 claims 2
- 235000012000 cholesterol Nutrition 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 208000006572 Human Influenza Diseases 0.000 claims 1
- 206010022000 Influenza Diseases 0.000 claims 1
- 101710043164 Segment-4 Proteins 0.000 claims 1
- 231100000765 Toxin Toxicity 0.000 claims 1
- 101700038759 VP1 Proteins 0.000 claims 1
- 239000002253 acid Substances 0.000 claims 1
- 150000007513 acids Chemical class 0.000 claims 1
- -1 anionic lipid Chemical class 0.000 claims 1
- 102000004965 antibodies Human genes 0.000 claims 1
- 108090001123 antibodies Proteins 0.000 claims 1
- 230000000295 complement Effects 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 229940079593 drugs Drugs 0.000 claims 1
- 101700005460 hemA Proteins 0.000 claims 1
- 239000000185 hemagglutinin Substances 0.000 claims 1
- 230000000051 modifying Effects 0.000 claims 1
- 230000001264 neutralization Effects 0.000 claims 1
- 150000003904 phospholipids Chemical class 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 239000003053 toxin Substances 0.000 claims 1
- 108020003112 toxins Proteins 0.000 claims 1
- 239000002753 trypsin inhibitor Substances 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 abstract 2
- 241001465754 Metazoa Species 0.000 abstract 1
- 239000003153 chemical reaction reagent Substances 0.000 abstract 1
- 238000007906 compression Methods 0.000 abstract 1
- 238000005538 encapsulation Methods 0.000 abstract 1
- 210000004962 mammalian cells Anatomy 0.000 abstract 1
- 239000000463 material Substances 0.000 abstract 1
Abstract
La présente invention concerne des enveloppes virales artificielles et d'autres vésicules lipidiques qui encapsulent des substances thérapeutiques telles que des vecteurs d'expression visant des cellules de mammifères. Les polynucléotides peuvent être mis dans les enveloppes par compression à l'avance avec un peptide court ayant une charge positive prédominante. L'opération de compression non seulement facilite l'encapsulation mais elle augmente aussi le nombre de vésicules contenant de l'acide nucléique, réduit au minimum la quantité d'acide nucléique libre, et elle peut aussi augmenter la taille et la complexité des plasmides qui peuvent être encapsulés. Les vésicules peuvent être pourvues d'un constituant ciblant des tissus, qui aide à les diriger vers certains sites tissulaires d'un animal, et elles peuvent aussi être pourvues d'un constituant fusogénique qui facilite l'apport de la substance thérapeutique dans la cellule. Les substances et les réactifs selon la présente invention sont efficaces, par exemple, pour accroître l'expression de protéines modèles à la fois dans des cellules isolées et chez des animaux intacts, et on s'attend à ce qu'ils soient utiles pour la thérapie génique.
Priority Applications (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
AU66914/96A AU6691496A (en) | 1995-08-01 | 1996-08-01 | Enhanced artificial viral envelopes for cellular delivery of therapeutic substances |
Applications Claiming Priority (6)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US173895P | 1995-08-01 | 1995-08-01 | |
US60/001,738 | 1995-08-01 | ||
US258095P | 1995-08-21 | 1995-08-21 | |
US60/002,580 | 1995-08-21 | ||
US69061396A | 1996-07-31 | 1996-07-31 | |
US60/690,613 | 1996-07-31 |
Publications (4)
Publication Number | Publication Date |
---|---|
WO1997004748A2 WO1997004748A2 (fr) | 1997-02-13 |
WO1997004748A3 WO1997004748A3 (fr) | 1997-05-29 |
WO1997004748B1 true WO1997004748B1 (fr) | 1997-07-17 |
WO1997004748A9 WO1997004748A9 (fr) | 1997-10-09 |
Family
ID=27356985
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/US1996/012750 WO1997004748A2 (fr) | 1995-08-01 | 1996-08-01 | Enveloppes virales artificielles renforcees pour l'apport de substances therapeutiques dans les cellules |
Country Status (2)
Country | Link |
---|---|
AU (1) | AU6691496A (fr) |
WO (1) | WO1997004748A2 (fr) |
Families Citing this family (51)
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ES2165050T3 (es) * | 1996-04-12 | 2002-03-01 | Univ Pittsburgh | Nuevos derivados cationicos de colesterilo que contienen grupos polares ciclicos. |
US6258792B1 (en) | 1996-04-12 | 2001-07-10 | University Of Pittsburgh | Cationic cholesteryl derivatives containing cyclic polar groups |
FR2751222B1 (fr) | 1996-07-16 | 1998-10-09 | Capsulis | Compositions contenant au moins un acide nucleique et leurs applications dans le domaine biomedical, en particulier en therapie genique |
AU7983798A (en) * | 1997-06-23 | 1999-01-04 | Sequus Pharmaceuticals, Inc. | Liposome-entrapped polynucleotide composition and method |
EP1801214B1 (fr) | 1997-07-07 | 2010-11-10 | Medical Research Council | Procédé de tri in vitro |
FR2766705B1 (fr) * | 1997-07-30 | 2001-05-25 | Biovector Therapeutics | Complexe particulaire de charge globale neutre ou negative forme d'une vesicule cationique ou neutre et d'une substance biologiquement active anionique, leur preparation et leur utilisation |
FR2766706B1 (fr) * | 1997-07-30 | 2001-05-25 | Biovector Therapeutics Sa | Complexes particulaires stables de charge globale neutre ou negative de structure multilamellaire composes par au moins une substance biologiquement active globalement anionique et un constituant cationique, leur preparation et utilisation |
US6372720B1 (en) * | 1998-02-05 | 2002-04-16 | Kenneth J. Longmuir | Liposome fusion and delivery vehicle |
US6903196B1 (en) | 1999-06-17 | 2005-06-07 | Utah Ventures Ii, L.P. | Methods for identifying and isolating tissue-specific lumen-exposed molecules |
GB0015624D0 (en) * | 2000-06-26 | 2000-08-16 | Nat Cancer Centre | Materials and methods relating to improved gene delivery |
AU2002231637A1 (en) * | 2000-11-22 | 2002-06-03 | Vectron Therapeutics Ag | Production and use of a targeted diagnostic system |
AU2002235141A1 (en) | 2000-11-27 | 2002-06-03 | Geron Corporation | Glycosyltransferase vectors for treating cancer |
US7491702B2 (en) | 2001-04-18 | 2009-02-17 | The Open University | Polypeptides related to amyloid precursor protein, pharmaceutical compositions thereof, and methods of treatment using the same |
US7622446B2 (en) | 2001-04-18 | 2009-11-24 | The Open University | Polypeptides, derivatives and uses thereof |
GB2391548B (en) * | 2001-04-18 | 2005-11-30 | Univ Open | Polypeptides and derivatives thereof related to amyloid precursor protein (APP) |
CA2445947A1 (fr) * | 2001-04-30 | 2002-11-07 | Targeted Genetics Corporation | Complexes d'administration de medicaments contenant des lipides et leurs methodes de production |
AU2002312410A1 (en) * | 2001-06-08 | 2002-12-23 | Target Protein Technologies, Inc. | Tissue-specific endothelial membrane proteins |
US7427395B2 (en) | 2002-11-01 | 2008-09-23 | Genomidea Inc. | Chemotherapeutic agent-incorporated pharmaceutical preparation |
US20100022414A1 (en) | 2008-07-18 | 2010-01-28 | Raindance Technologies, Inc. | Droplet Libraries |
US20060078893A1 (en) | 2004-10-12 | 2006-04-13 | Medical Research Council | Compartmentalised combinatorial chemistry by microfluidic control |
GB0307403D0 (en) | 2003-03-31 | 2003-05-07 | Medical Res Council | Selection by compartmentalised screening |
GB0307428D0 (en) | 2003-03-31 | 2003-05-07 | Medical Res Council | Compartmentalised combinatorial chemistry |
US20050221339A1 (en) | 2004-03-31 | 2005-10-06 | Medical Research Council Harvard University | Compartmentalised screening by microfluidic control |
US7968287B2 (en) | 2004-10-08 | 2011-06-28 | Medical Research Council Harvard University | In vitro evolution in microfluidic systems |
EP3312196B1 (fr) | 2005-03-23 | 2019-07-17 | Genmab A/S | Anticorps dirigés contre cd38 pour le traitement du myélome multiple |
AU2006335290A1 (en) | 2006-01-11 | 2007-07-19 | Raindance Technologies, Inc. | Microfluidic devices and methods of use in the formation and control of nanoreactors |
US9562837B2 (en) | 2006-05-11 | 2017-02-07 | Raindance Technologies, Inc. | Systems for handling microfludic droplets |
EP4190448A3 (fr) | 2006-05-11 | 2023-09-20 | Bio-Rad Laboratories, Inc. | Dispositifs microfluidiques |
WO2008021123A1 (fr) | 2006-08-07 | 2008-02-21 | President And Fellows Of Harvard College | Tensioactifs fluorocarbonés stabilisateurs d'émulsions |
WO2008097559A2 (fr) | 2007-02-06 | 2008-08-14 | Brandeis University | Manipulation de fluides et de réactions dans des systèmes microfluidiques |
US8592221B2 (en) | 2007-04-19 | 2013-11-26 | Brandeis University | Manipulation of fluids, fluid components and reactions in microfluidic systems |
PL2222697T3 (pl) | 2007-11-01 | 2013-05-31 | Astellas Pharma Inc | Immunosupresyjne polipeptydy i kwasy nukleinowe |
US8528589B2 (en) | 2009-03-23 | 2013-09-10 | Raindance Technologies, Inc. | Manipulation of microfluidic droplets |
US10520500B2 (en) | 2009-10-09 | 2019-12-31 | Abdeslam El Harrak | Labelled silica-based nanomaterial with enhanced properties and uses thereof |
US10837883B2 (en) | 2009-12-23 | 2020-11-17 | Bio-Rad Laboratories, Inc. | Microfluidic systems and methods for reducing the exchange of molecules between droplets |
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EP2534267B1 (fr) | 2010-02-12 | 2018-04-11 | Raindance Technologies, Inc. | Analyse numérique d'analytes |
US10351905B2 (en) | 2010-02-12 | 2019-07-16 | Bio-Rad Laboratories, Inc. | Digital analyte analysis |
US9366632B2 (en) | 2010-02-12 | 2016-06-14 | Raindance Technologies, Inc. | Digital analyte analysis |
EP2622103B2 (fr) | 2010-09-30 | 2022-11-16 | Bio-Rad Laboratories, Inc. | Dosages sandwich dans des gouttelettes |
EP2673614B1 (fr) | 2011-02-11 | 2018-08-01 | Raindance Technologies, Inc. | Procédé de formation de gouttelettes mélangées |
EP3736281A1 (fr) | 2011-02-18 | 2020-11-11 | Bio-Rad Laboratories, Inc. | Compositions et méthodes de marquage moléculaire |
DE202012013668U1 (de) | 2011-06-02 | 2019-04-18 | Raindance Technologies, Inc. | Enzymquantifizierung |
US8841071B2 (en) | 2011-06-02 | 2014-09-23 | Raindance Technologies, Inc. | Sample multiplexing |
US8658430B2 (en) | 2011-07-20 | 2014-02-25 | Raindance Technologies, Inc. | Manipulating droplet size |
US9340784B2 (en) * | 2012-03-19 | 2016-05-17 | Ionis Pharmaceuticals, Inc. | Methods and compositions for modulating alpha-1-antitrypsin expression |
US11901041B2 (en) | 2013-10-04 | 2024-02-13 | Bio-Rad Laboratories, Inc. | Digital analysis of nucleic acid modification |
US9944977B2 (en) | 2013-12-12 | 2018-04-17 | Raindance Technologies, Inc. | Distinguishing rare variations in a nucleic acid sequence from a sample |
US11193176B2 (en) | 2013-12-31 | 2021-12-07 | Bio-Rad Laboratories, Inc. | Method for detecting and quantifying latent retroviral RNA species |
US10647981B1 (en) | 2015-09-08 | 2020-05-12 | Bio-Rad Laboratories, Inc. | Nucleic acid library generation methods and compositions |
CA3099497A1 (fr) * | 2018-05-15 | 2019-11-21 | Flagship Pioneering Innovations V, Inc. | Compositions de fusosome et leurs utilisations |
Family Cites Families (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JP2958076B2 (ja) * | 1990-08-27 | 1999-10-06 | 株式会社ビタミン研究所 | 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法 |
PL296382A1 (en) * | 1991-02-02 | 1993-11-02 | Nika Health Products Ltd Li Li | Artificial membrane beads containing functionally active peptides responsible for fusion as a drug administering system |
DE4139001A1 (de) * | 1991-11-27 | 1993-06-03 | Boehringer Mannheim Gmbh | Verfahren zur einschleusung von nukleinsaeuren in zellen |
WO1994018947A1 (fr) * | 1993-02-16 | 1994-09-01 | Sharifa Karali | Complexe protamine-adn de haute purete et son utilisation |
EP1489184A1 (fr) * | 1995-06-07 | 2004-12-22 | Inex Pharmaceutical Corp. | Particules d'acides nucléiques et de lipides préparées au moyen d'un intermédiaire de complexe hydrophobe d'acides nucléiques et de lipides et utilisation pour transférer des gènes |
-
1996
- 1996-08-01 WO PCT/US1996/012750 patent/WO1997004748A2/fr active Application Filing
- 1996-08-01 AU AU66914/96A patent/AU6691496A/en not_active Abandoned
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