- X. SU ET AL., MOL. PHARMACEUTICS, vol. 8, 2011, pages 774 - 787
- KOZAK, M., NUCLEIC ACIDS RES, vol. 15, no. 20, 1987, pages 8125 - 48
- HEIDENREICH ET AL., J BIOL CHEM, vol. 269, 1994, pages 2131 - 8
- KARIKÓ, K. ET AL., MOLECULAR THERAPY, vol. 16, no. 11, 2008, pages 1833 - 1840
- YOKOE ET AL., NATURE BIOTECHNOLOGY, vol. 14, 1996, pages 1252 - 1256
- KRIEG ET AL., MELTON. METHODS IN ENZYMOLOGY, vol. 155, 1987, pages 397 - 415
- LIEBHABER., J. MOL. BIOL., vol. 226, 1992, pages 2 - 13
- MONIA ET AL., J BIOL CHEM., vol. 268, 1993, pages 14514 - 22
- LASIC, TRENDS BIOTECHNOL, vol. 16, 1998, pages 307 - 321
- DRUMMOND ET AL., PHARMACOL. REV., vol. 51, 1999, pages 691 - 743
- LASIC, TRENDS BIOTECHNOL., vol. 16, 1998, pages 307 - 321
- LASIC ET AL., FEBS LETT., vol. 312, 1992, pages 255 - 258
- N.J. CAPLEN ET AL., GENE THER., vol. 2, 1995, pages 603
- S. LI ET AL., , GENE THER., vol. 1997, no. 4, pages 891
- FELGNER ET AL., PROC. NAT'L ACAD. SCI., vol. 84, 1987, pages 7413
- BEHR ET AL., PROC. NAT.'L ACAD. SCI., vol. 86, 1989, pages 6982
- SEMPLE ET AL., NATURE BIOTECH., vol. 28, 2010, pages 172 - 176
- HEYES, J. ET AL., J CONTROLLED RELEASE, vol. 107, 2005, pages 276 - 287
- MORRISSEY, DV. ET AL., NAT. BIOTECHNOL., vol. 23, no. 8, 2005, pages 1003 - 1007
- GAO ET AL., BIOCHEM. BIOPHYS. RES. COMM., vol. 179, 1991, pages 280
- WOLF ET AL., BIOTECHNIQUES, vol. 23, 1997, pages 139
- KLIBANOV ET AL., FEBS LETTERS, vol. 268, no. 1, 1990, pages 235 - 237
- BLOOMFIELD, ANN. REV. BIOPHYS. BIOENG., vol. 10, 1981, pages 421 - 450
- HILLERY AM ET AL.: "Drug Delivery and Targeting: For Pharmacists and Pharmaceutical Scientists", 2002, TAYLOR & FRANCIS, INC.
- FECHTEBROWNLEE, J. GEN. VIROLOGY, vol. 86, 2005, pages 1239 - 1249
- MICHAEL J. PASSINEAU ET AL: "[alpha]-Galactosidase A Expressed in the Salivary Glands Partially Corrects Organ Biochemical Deficits in the Fabry Mouse Through Endocrine Trafficking", HUMAN GENE THERAPY, vol. 22, no. 3, 1 March 2011 (2011-03-01), pages 293 - 301, XP055478144, ISSN: 1043-0342, DOI: 10.1089/hum.2010.069
- AKIN AKINC ET AL: "A combinatorial library of lipid-like materials for delivery of RNAi therapeutics", NATURE BIOTECHNOLOGY, vol. 26, no. 5, 27 April 2008 (2008-04-27), New York, pages 561 - 569, XP055533621, ISSN: 1087-0156, DOI: 10.1038/nbt1402
- LIU YANG ET AL: "Designer Lipids Advance Systemic siRNA Delivery", MOLECULAR THERAPY, vol. 18, no. 4, April 2010 (2010-04-01), pages 669 - 670, XP002684383, ISSN: 1525-0016
- SEAN C SEMPLE ET AL: "Rational design of cationic lipids for siRNA delivery", NATURE BIOTECHNOLOGY, NATURE PUBLISHING GROUP, NEW YORK, NY, US, vol. 28, no. 2, 1 February 2010 (2010-02-01), pages 172 - 176, XP002633693, ISSN: 1087-0156, [retrieved on 20100117], DOI: 10.1038/NBT.1602
- GEERTRUI TAVERNIER ET AL: "mRNA as gene therapeutic: How to control protein expression", JOURNAL OF CONTROLLED RELEASE, vol. 150, no. 3, 1 March 2011 (2011-03-01), pages 238 - 247, XP055068617, ISSN: 0168-3659, DOI: 10.1016/j.jconrel.2010.10.020
- K. T. LOVE ET AL: "Lipid-like materials for low-dose, in vivo gene silencing", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, vol. 107, no. 5, 2 February 2010 (2010-02-02), pages 1864 - 1869, XP055077922, ISSN: 0027-8424, DOI: 10.1073/pnas.0910603106
- AKINC A ET AL: "Development of Lipidoid-siRNA Formulations for Systemic Delivery to the Liver", MOLECULAR THERAPY,, vol. 17, no. 5, 1 May 2009 (2009-05-01), pages 872 - 879, XP008105983, ISSN: 1525-0016, [retrieved on 20090303], DOI: 10.1038/MT.2009.36
- KOYNOVA RUMIANA ET AL: "Recent patents in cationic lipid carriers for delivery of nucleic acids", RECENT PATENTS ON DNA & GENE SEQUENCES, BENTHAM SCIENCE, UNITED ARAB EMIRATES, vol. 5, no. 1, 1 January 2011 (2011-01-01), pages 8 - 27, XP009170736, ISSN: 2212-3431, DOI: 10.2174/187221511794839255
- XINGFANG SU: "In Vitro and in Vivo mRNA Delivery Using Lipid-Enveloped pH-Responsive Polymer Nanoparticles", MOLECULAR PHARMACEUTICS, vol. 8, no. 3, 21 March 2011 (2011-03-21), US, pages 774 - 787, XP093167466, ISSN: 1543-8384, DOI: 10.1021/mp100390w
- "Handbook of Pharmaceutical Biotechnology", 1 June 2007, JOHN WILEY & SONS , ISBN: 978-0-471-21386-4, article SHAYNE COX, STEVE PASCOLO: "Chapter 7.2. Plasmid DNA and Messenger RNA for Therapy", pages: 971 - 1011, XP055626043, DOI: 10.1002/9780470117118.ch07b
* Pages 971-1011 *
- "Handbook of Pharmaceutical Biotechnology", 1 June 2007, JOHN WILEY & SONS , ISBN: 978-0-471-21386-4, article SHAYNE COX, STEVE PASCOLO: "Chapter 7.2. Plasmid DNA and Messenger RNA for Therapy", pages: 971 - 1011, XP009569006, DOI: 10.1002/9780470117118.ch07b
- SHIRAZI RAHAU S., EWERT KAI K., LEAL CECILIA, MAJZOUB RAMSEY N., BOUXSEIN NATHAN F., SAFINYA CYRUS R.: "Author manuscript: Synthesis and characterization of degradable multivalent cationic lipids with disulfide-bond spacers for gene delivery", BIOCHIMICA ET BIOPHYSICA ACTA, ELSEVIER, AMSTERDAM, NL, vol. 1808, no. 9, 1 September 2011 (2011-09-01), AMSTERDAM, NL , pages 1 - 25, XP093439748, ISSN: 0005-2736, DOI: 10.1016/j.bbamem.2011.04.020
* pages 2156–2166. *
- FRÉDÉRIC MARTINON, SIVADASAN KRISHNAN, GERLINDE LENZEN, RÉMY MAGNÉ, ELISABETH GOMARD, JEAN-GÉRARD GUILLET, JEAN-PAUL LÉVY, PIERRE : "INDUCTION OF VIRUS-SPECIFIC CYTOTOXIC T LYMPHOCYTES IN VIVO BY LIPOSOME-ENTRAPPED MRNA", EUROPEAN JOURNAL OF IMMUNOLOGY, WILEY-VCH, HOBOKEN, USA, vol. 23, no. 7, 1 July 1993 (1993-07-01), Hoboken, USA, pages 1719 - 1722, XP002937540, ISSN: 0014-2980, DOI: 10.1002/eji.1830230749
* pages 1719-1722 *
- JAMES HEYES, LORNE PALMER, KAZ BREMNER, IAN MACLACHLAN: "Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids", JOURNAL OF CONTROLLED RELEASE, ELSEVIER, NL, vol. 107, no. 2, 3 October 2005 (2005-10-03), NL , pages 276 - 287, XP008157522, ISSN: 0168-3659, DOI: 10.1016/j.jconrel.2005.06.014
* pages 276-287 *
- LLOYD B. JEFFS ; LORNE R. PALMER ; ELLEN G. AMBEGIA ; CORY GIESBRECHT ; SHANNON EWANICK ; IAN MACLACHLAN: "A Scalable, Extrusion-Free Method for Efficient Liposomal Encapsulation of Plasmid DNA", PHARMACEUTICAL RESEARCH, KLUWER ACADEMIC PUBLISHERS-PLENUM PUBLISHERS, NL, vol. 22, no. 3, 1 March 2005 (2005-03-01), NL , pages 362 - 372, XP019370801, ISSN: 1573-904X, DOI: 10.1007/s11095-004-1873-z
* pages 362-372 *
- FRICKE, J. ; SEUNG, E. ; VASSOR, V. ; TAGER, A.M. ; GANDHI, R.T. ; WALKER, B. ; IRVINE, D. ; KAVANAGH, D.: "Synthetic mRNA-loaded Nanoparticles Elicit Mucosal IL-17+ and IFNγ+ T Cells", CLINICAL IMMUNOLOGY, ELSEVIER, AMSTERDAM, NL, vol. 135, no. Supplement, 1 January 2010 (2010-01-01), AMSTERDAM, NL , pages S137, XP027049215, ISSN: 1521-6616, DOI: 10.1016/j.clim.2010.03.416
- CROOKE STANLEY T: "ANTISENSE DRUG TECHNOLOGY: PRINCIPLES, STRATEGIES, AND APPLICATIONS, 2ND ED.", 1 January 2007, CRC PRESS , US , ISBN: 978-0-8493-8796-8, article MACLACHLAN I.: "Liposomal formulations for nucleic acid delivery", pages: 237 - 270, XP002584305
- YOU CHANGJUN, DAI XIAOXIA, WANG YINSHENG: "Position-dependent effects of regioisomeric methylated adenine and guanine ribonucleosides on translation", NUCLEIC ACIDS RESEARCH, INFORMATION RETRIEVAL LTD., ENGLAND, vol. 45, no. 15, 6 September 2017 (2017-09-06), England, pages 9059 - 9067, XP093439754, ISSN: 0305-1048, DOI: 10.1093/nar/gkx515
- AEBISCHER MEGANE K.; TSALMPOURIS ATHANASIOS; RUDAZ SERGE; MALBURET CAMILLE; COTTE JEAN-FRANÇOIS; GUILLARME DAVY; MAURER JONATHAN: "Two-dimensional AEX – IP-RPLC: New insights on mRNA integrity and encapsulation efficiency of mRNA-lipid nanoparticle formulations", ANALYTICA CHIMICA ACTA, ELSEVIER, AMSTERDAM, NL, vol. 1394, 7 February 2026 (2026-02-07), AMSTERDAM, NL , XP087987625, ISSN: 0003-2670, DOI: 10.1016/j.aca.2026.345215
- ANITA BRINGMANN, STEFANIE ANDREA ERIKA HELD, ANNKRISTIN HEINE, PETER BROSSART: "RNA Vaccines in Cancer Treatment", JOURNAL OF BIOMEDICINE AND BIOTECHNOLOGY, HINDAWI PUB. CORP., vol. 2010, pages 1 - 12, XP002765542, ISSN: 1110-7243, DOI: 10.1155/2010/623687
- DONG YIZHOU, SIEGWART DANIEL J., ANDERSON DANIEL G.: "Author manuscript: Strategies, design, and chemistry in siRNA delivery systems", ADVANCED DRUG DELIVERY REVIEWS, ELSEVIER, AMSTERDAM , NL, vol. 144, 1 April 2019 (2019-04-01), Amsterdam , NL , pages 1 - 30, XP093439778, ISSN: 0169-409X, DOI: 10.1016/j.addr.2019.05.004
- JIRIKOWSKI GUSTAV F., PIETRO PAOLO SANNA, DOMINIQUE MACIEJEWSKI-LENOIR, AND FLOYD E. BLOOM: "Reversal of Diabetes Insipidus in Brattleboro Rats: Intrahypothalamic Injection of Vasopressin mRNA", SCIENCE, AMERICAN ASSOCIATION FOR THE ADVANCEMENT OF SCIENCE, US, vol. 255, no. 5047, 21 February 1992 (1992-02-21), US , pages 996 - 998, XP093439780, ISSN: 0036-8075, DOI: 10.1126/science.1546298
- W-Z ZHOU, D S B HOON, S K S HUANG, ET AL: "RNA melanoma vaccine: induction of antitumor immunity by human glycoprotein 100 mRNA immunization.", HUMAN GENE THERAPY, MARY ANN LIEBERT, INC. PUBLISHERS, GB, vol. 10, no. 16, 1 November 1999 (1999-11-01), GB , pages 2719 - 2724, XP002660042, ISSN: 1043-0342, DOI: 10.1089/10430349950016762
- MICHAEL S D KORMANN, GÜNTHER HASENPUSCH, MANISH K ANEJA, GABRIELA NICA, ANDREAS W FLEMMER, SUSANNE HERBER-JONAT, MARCELINE HUPPMAN: "Expression of therapeutic proteins after delivery of chemically modified mRNA in mice", NATURE BIOTECHNOLOGY, NATURE PUBLISHING GROUP US, NEW YORK, vol. 29, no. 2, 1 February 2011 (2011-02-01), New York, pages 154 - 157, XP055040839, ISSN: 1087-0156, DOI: 10.1038/nbt.1733
- KARIKÓ K, MURAMATSU H, KELLER J M, WEISSMAN D.: "Increased erythropoiesis in mice injected with submicrogram quantities of pseudouridine-containing mRNA encoding erythropoietin.", MOLECULAR THERAPY, NATURE PUBLISHING GROUP, GB, vol. 20, no. 5, 1 May 2012 (2012-05-01), GB , pages 948 - 953, XP002696191, ISSN: 1525-0024, DOI: 10.1038/mt.2012.7
- RAYNER J O, DRYGA S A, KAMRUD K I: "ALPHAVIRUS VECTORS AND VACCINATION", REVIEWS IN MEDICAL VIROLOGY, CHICHESTER, GB, vol. 12, no. 05, 1 September 2002 (2002-09-01), GB , pages 279 - 296, XP008035758, ISSN: 1052-9276, DOI: 10.1002/rmv.360
- WEISS RICHARD; SCHEIBLHOFER SANDRA; ROESLER ELISABETH; FERREIRA FATIMA; THALHAMER JOSEF: "Prophylactic mRNA vaccination against allergy", CURRENT OPINION IN ALLERGY AND CLINICAL IMMUNOLOGY, LIPPINCOTT WILLIAMS AND WILKINS, US, vol. 10, no. 6, 1 December 2010 (2010-12-01), US , pages 567 - 574, XP009164452, ISSN: 1528-4050, DOI: 10.1097/ACI.0b013e32833fd5b6
- SIBERT BRYAN S., KIM JOSEPH Y., YANG JIE E., KE ZUNLONG, STOBART CHRISTOPHER C., MOORE MARTIN L., WRIGHT ELIZABETH R.: "Assembly of respiratory syncytial virus matrix protein lattice and its coordination with fusion glycoprotein trimers", NATURE COMMUNICATIONS, NATURE PUBLISHING GROUP, UK, vol. 15, no. 1, 14 July 2024 (2024-07-14), UK, pages 5923 - 13, XP093439799, ISSN: 2041-1723, DOI: 10.1038/s41467-024-50162-x
- J. SAENZ-BADILLOS, S. P. AMIN, R. D. GRANSTEIN: "RNA as a tumor vaccine: a review of the literature", EXPERIMENTAL DERMATOLOGY, BLACKWELL MUNSGAARD, COPENHAGEN; DK, vol. 10, no. 3, 1 June 2001 (2001-06-01), COPENHAGEN; DK , pages 143 - 154, XP055244457, ISSN: 0906-6705, DOI: 10.1034/j.1600-0625.2001.010003143.x
- SAXENA, S. ; SONWANE, A.A. ; DAHIYA, S.S. ; PATEL, C.L. ; SAINI, M. ; RAI, A. ; GUPTA, P.K.: "Induction of immune responses and protection in mice against rabies using a self-replicating RNA vaccine encoding rabies virus glycoprotein", VETERINARY MICROBIOLOGY, ELSEVIER BV, NL, vol. 136, no. 1-2, 14 April 2009 (2009-04-14), NL , pages 36 - 44, XP026071306, ISSN: 0378-1135, DOI: 10.1016/j.vetmic.2008.10.030
- KARIKÓ KATALIN, MURAMATSU HIROMI, WELSH FRANK A, LUDWIG JÁNOS, KATO HIROKI, AKIRA SHIZUO, WEISSMAN DREW: "Incorporation of Pseudouridine Into mRNA Yields Superior Nonimmunogenic Vector With Increased Translational Capacity and Biological Stability", MOLECULAR THERAPY, ELSEVIER INC, UNITED STATES, vol. 16, no. 11, 1 November 2008 (2008-11-01), United States, pages 1833 - 1840, XP093147613, ISSN: 1525-0016, DOI: 10.1038/mt.2008.200
- ZOU, S. ; SCARFO, K. ; NANTZ, M.H. ; HECKER, J.G.: "Lipid-mediated delivery of RNA is more efficient than delivery of DNA in non-dividing cells", INTERNATIONAL JOURNAL OF PHARMACEUTICS, ELSEVIER, AMSTERDAM, NL, vol. 389, no. 1-2, 15 April 2010 (2010-04-15), AMSTERDAM, NL, pages 232 - 243, XP026933138, ISSN: 0378-5173, DOI: 10.1016/j.ijpharm.2010.01.019
- YAMAMOTO A. ET AL: "Current prospects for mRNA gene delivery", EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS, ELSEVIER SCIENCE PUBLISHERS B.V., AMSTERDAM., NL, vol. 71, no. 3, 1 March 2009 (2009-03-01), NL , pages 484 - 489, XP025992169, ISSN: 0939-6411, DOI: 10.1016/j.ejpb.2008.09.016
- TRACY S ZIMMERMANN, AMY C H LEE, AKIN AKINC, BIRGIT BRAMLAGE, DAVID BUMCROT, MATTHEW N FEDORUK, JENS HARBORTH, JAMES A HEYES, LLOY: "RNAi-mediated gene silencing in non-human primates.", NATURE, SPRINGER SCIENCE AND BUSINESS MEDIA LLC, LONDON, vol. 441, no. 7089, 4 May 2006 (2006-05-04), London, pages 111 - 114, XP002577898, ISSN: 0028-0836, DOI: 10.1038/NATURE04688
- SEMPLE, S.C. KLIMUK, S.K. HARASYM, T.O. DOS SANTOS, N. ANSELL, S.M. WONG, K.F. MAURER, N. STARK, H. CULLIS, P.R. HOPE, M.J. SCHERR: "Efficient encapsulation of antisense oligonucleotides in lipid vesicles using ionizable aminolipids: formation of novel small multilamellar vesicle structures", BIOCHIMICA ET BIOPHYSICA ACTA, ELSEVIER, AMSTERDAM, NL, vol. 1510, no. 1-2, 9 February 2001 (2001-02-09), AMSTERDAM, NL , pages 152 - 166, XP004248775, ISSN: 0005-2736, DOI: 10.1016/S0005-2736(00)00343-6
- SANDRA K. KLIMUK, SEAN C. SEMPLE, PATRICK N. NAHIRNEY, MICHELLE C. MULLEN, C. FRANK BENNETT, PETER SCHERRER, MICHAEL J. HOPE: "Enhanced anti-inflammatory activity of a liposomal intercellular adhesion molecule-1 antisense oligodeoxynucleotide in an acute model of contact hypersensitivity", THE JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS, ELSEVIER INC, UNITED STATES, vol. 292, no. 2, 1 February 2000 (2000-02-01), United States, pages 480 - 488, XP002336134, ISSN: 0022-3565, DOI: 10.1016/S0022-3565(24)35316-9
- AKINC AKIN ET AL: "A combinatorial library of lipid-like materials for delivery of RNAi therapeutics.", NATURE BIOTECHNOLOGY, NATURE PUBLISHING GROUP US, NEW YORK, vol. 26, no. 5, 1 May 2008 (2008-05-01), New York, pages 561 - 569, XP002577899, ISSN: 1087-0156, DOI: 10.1038/NBT1402
- LOVE KEVIN T., MAHON KERRY P., LEVINS CHRISTOPHER G., WHITEHEAD KATHRYN A., QUERBES WILLIAM, DORKIN J. ROBERT, QIN JUNE, CANTLEY W: "SUPPORTING INFORMATION: Lipid-like materials for low-dose, in vivo gene silencing", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES (PNAS), NATIONAL ACADEMY OF SCIENCES, vol. 107, no. 5, 2 February 2010 (2010-02-02), pages 1864 - 1869, XP093088665, ISSN: 0027-8424, DOI: 10.1073/pnas.0910603106
- K. T. LOVE, K. P. MAHON, C. G. LEVINS, K. A. WHITEHEAD, W. QUERBES, J. R. DORKIN, J. QIN, W. CANTLEY, L. L. QIN, T. RACIE, M. FRAN: "Lipid-like materials for low-dose, in vivo gene silencing", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, NATIONAL ACADEMY OF SCIENCES, vol. 107, no. 5, 2 February 2010 (2010-02-02), pages 1864 - 1869, XP055077922, ISSN: 00278424, DOI: 10.1073/pnas.0910603106
- HEIJNE VON G: "THE SIGNAL PEPTIDE", JOURNAL OF MEMBRANE BIOLOGY, SPRINGER NEW YORK LLC, US, vol. 115, no. 03, 1 May 1990 (1990-05-01), US , pages 195 - 201, XP009011512, ISSN: 0022-2631, DOI: 10.1007/BF01868635
- PASCOLO STEVE: "Vaccination with messenger RNA (mRNA)", TOLL-LIKE RECEPTORS (TLRS) AND INNATE IMMUNITY, BERLIN [U.A.] : SPRINGER, DE, vol. 183, 1 January 2008 (2008-01-01), DE , pages 221 - 235, XP001538300, ISBN: 978-3-540-72166-6, DOI: 10.1007/978-3-540-72167-3_11
- D WEISSMAN, H NI, D SCALES, A DUDE, J CAPODICI, K MCGIBNEY, A ABDOOL, S N ISAACS, G CANNON, K KARIKÓ: "HIV gag mRNA transfection of dendritic cells (DC) delivers encoded antigen to MHC class I and II molecules, causes DC maturation, and induces a potent human in vitro primary immune response", JOURNAL OF IMMUNOLOGY, WILLIAMS & WILKINS CO., vol. 165, no. 8, 1 January 2000 (2000-01-01), pages 4710 - 4717, XP008051732, ISSN: 0022-1767, DOI: 10.4049/jimmunol.165.8.4710
- DE FOUGEROLLES ANTONIN R: "Delivery vehicles for small interfering RNA in vivo", HUMAN GENE THERAPY, MARY ANN LIEBERT, INC. PUBLISHERS, GB, vol. 19, no. 2, 1 February 2008 (2008-02-01), GB , pages 125 - 132, XP002522155, ISSN: 1043-0342, DOI: 10.1089/HUM.2008.928
- B. OZPOLAT, SOOD A. K., LOPEZ-BERESTEIN G.: "Nanomedicine based approaches for the delivery of siRNA in cancer", JOURNAL OF INTERNAL MEDICINE, BLACKWELL PUBLISHING LTD, GB, vol. 267, no. 1, 1 January 2009 (2009-01-01), GB , pages 44 - 53, XP055242950, ISSN: 0954-6820, DOI: 10.1111/j.1365-2796.2009.02191.x
- GEERTRUI TAVERNIER, OLIWIA ANDRIES, JO DEMEESTER, NIEK N. SANDERS, STEFAAN C. DE SMEDT, JOANNA REJMAN: "mRNA as gene therapeutic: How to control protein expression", JOURNAL OF CONTROLLED RELEASE, ELSEVIER, vol. 150, no. 3, 1 March 2011 (2011-03-01), pages 238 - 247, XP055068617, ISSN: 01683659, DOI: 10.1016/j.jconrel.2010.10.020
- MELISSA A. TRAN, REBECCA J. WATTS, GAVIN P. ROBERTSON: "Use of liposomes as drug delivery vehicles for treatment of melanoma", PIGMENT CELL & MELANOMA RESEARCH, WILEY INTERSCIENCE, UNITED STATES, DENMARK, vol. 22, no. 4, 1 August 2009 (2009-08-01), United States, Denmark , pages 388 - 399, XP055299784, ISSN: 1755-1471, DOI: 10.1111/j.1755-148X.2009.00581.x
- KAWAKAMI M, TSUTSUMI H, KUMAKAWA T, ABE H, HIRAI M, KUROSAWA S, MORI M, FUKUSHIMA M: "Levels of serum granulocyte colony-stimulating factor in patients with infections", BLOOD, ELSEVIER INC, UNITED STATES, vol. 76, no. 10, 15 November 1990 (1990-11-15), United States, pages 1962 - 1964, XP093439847, ISSN: 0006-4971, DOI: 10.1182/blood.v76.10.1962.bloodjournal76101962
- FOX ELIZABETH, WIDEMANN BRIGITTE C., HAWKINS DOUGLAS S., JAYAPRAKASH NALINI, DAGHER RAMZI, AIKIN ALBERTA A., BERNSTEIN DONNA, LONG: "Author manuscript: Randomized Trial and Pharmacokinetic Study of Pegfilgrastim versus Filgrastim after Dose-Intensive Chemotherapy in Young Adults and Children with Sarcomas", CLINICAL CANCER RESEARCH, AMERICAN ASSOCIATION FOR CANCER RESEARCH INC, vol. 15, no. 23, 1 December 2009 (2009-12-01), pages 1 - 18, XP093439851, ISSN: 1078-0432, DOI: 10.1158/1078-0432.ccr-09-0761
- KAY M A, LANDEN C N, ROTHENBERG S R, TAYLOR L A, LELAND F, WIEHLE S, FANG B, BELLINGER D, FINEGOLD M, THOMPSON A R: "In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs.", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES (PNAS), NATIONAL ACADEMY OF SCIENCES, vol. 91, no. 6, 15 March 1994 (1994-03-15), pages 2353 - 2357, XP093439855, ISSN: 0027-8424, DOI: 10.1073/pnas.91.6.2353
- D64 - Submission of the Applicant of 9 May 2025 filed during prosecution of EP 24 197 314.8 (the application underlying EP’350).
- D65 - Submission of the Applicant of 2 September 2025 filed during prosecution of EP 24 197 314.8 (the application underlying EP’350).
- GUO X, SZOKA F C: "CHEMICAL APPROACHES TO TRIGGERABLE LIPID VESICLES FOR DRUG AND GENE DELIVERY", ACCOUNTS OF CHEMICAL RESEARCH, ACS , WASHINGTON , DC, US, vol. 36, no. 05, 1 May 2003 (2003-05-01), US , pages 335 - 341, XP001161617, ISSN: 0001-4842, DOI: 10.1021/ar9703241
- HITOMI HIROFUMI, KASAHARA TOMOKO, KATAGIRI NAOKO, HOSHINA AZUSA, MAE SHIN-ICHI, KOTAKA MAKI, TOYOHARA TAKAFUMI, RAHMAN ASADUR, NAK: "Human pluripotent stem cell–derived erythropoietin-producing cells ameliorate renal anemia in mice", SCIENCE TRANSLATIONAL MEDICINE, AMERICAN ASSOCIATION FOR THE ADVANCEMENT OF SCIENCE, vol. 9, no. 409, 27 September 2017 (2017-09-27), pages eaaj2300 - 16, XP093439861, ISSN: 1946-6234, DOI: 10.1126/scitranslmed.aaj2300
- LONG XU, THOMAS ANCHORDOQUY: "Drug delivery trends in clinical trials and translational medicine: Challenges and opportunities in the delivery of nucleic acid-based therapeutics", JOURNAL OF PHARMACEUTICAL SCIENCES, AMERICAN CHEMICAL SOCIETY AND AMERICAN PHARMACEUTICAL ASSOCIATION, vol. 100, no. 1, 1 January 2011 (2011-01-01), pages 38 - 52, XP055044014, ISSN: 00223549, DOI: 10.1002/jps.22243
- FANG J, ET AL: "Stable antibody expression at therapeutic levels using the 2A peptide.", NATURE BIOTECHNOLOGY, NATURE PUBLISHING GROUP US, NEW YORK, vol. 23, no. 5, 17 April 2005 (2005-04-17), New York, pages 584 - 590, XP002369310, ISSN: 1087-0156, DOI: 10.1038/nbt1087
- D71 - T0197/22 (Delivery of mRNA II/TRANSLATE BIO)
- AKINC A., ET AL: "Development of Lipidoid-siRNA Formulations for Systemic Delivery to the Liver", MOLECULAR THERAPY, ELSEVIER INC, UNITED STATES, vol. 17, no. 5, 1 May 2009 (2009-05-01), United States, pages 872 - 879, XP008105983, ISSN: 1525-0016, DOI: 10.1038/MT.2009.36
- KOYNOVA RUMIANA; TENCHOV BORIS: "Recent patents in cationic lipid carriers for delivery of nucleic acids.", RECENT PATENTS ON DNA & GENE SEQUENCES, BENTHAM SCIENCE, UNITED ARAB EMIRATES, vol. 5, no. 1, 1 January 2011 (2011-01-01), United Arab Emirates , pages 8 - 27, XP009170736, ISSN: 2212-3431, DOI: 10.2174/187221511794839255
- SCHROEDER A., LEVINS C. G., CORTEZ C., LANGER R., ANDERSON D. G.: "Author manuscript: Lipid‐based nanotherapeutics for siRNA delivery", JOURNAL OF INTERNAL MEDICINE, BLACKWELL PUBLISHING LTD, GB, vol. 267, no. 1, 14 February 2017 (2017-02-14), GB , pages 1 - 21, XP093439869, ISSN: 0954-6820, DOI: 10.1111/j.1365-2796.2009.02189.x
- SEAN C SEMPLE, AKIN AKINC, JIANXIN CHEN, AMMEN P SANDHU, BARBARA L MUI, CONNIE K CHO, DINAH W Y SAH, DERRICK STEBBING, ERIN J CROS: "Rational design of cationic lipids for siRNA delivery", NATURE BIOTECHNOLOGY, NATURE PUBLISHING GROUP US, NEW YORK, vol. 28, no. 2, 1 February 2010 (2010-02-01), New York, pages 172 - 176, XP002633693, ISSN: 1087-0156, DOI: 10.1038/NBT.1602
- YAN, X. KUIPERS, F. HAVEKES, L.M. HAVINGA, R. DONTJE, B. POELSTRA, K. SCHERPHOF, G.L. KAMPS, J.A.A.M.: "The role of apolipoprotein E in the elimination of liposomes from blood by hepatocytes in the mouse", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, ELSEVIER, AMSTERDAM NL, vol. 328, no. 1, 4 March 2005 (2005-03-04), Amsterdam NL , pages 57 - 62, XP004723739, ISSN: 0006-291X, DOI: 10.1016/j.bbrc.2004.12.137
- AKIN AKINC, WILLIAM QUERBES, SOMA DE, JUNE QIN, MARIA FRANK-KAMENETSKY, K. NARAYANANNAIR JAYAPRAKASH, MUTHUSAMY JAYARAMAN, KALLANT: "Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms", MOLECULAR THERAPY, NO LONGER PUBLISHED BY ELSEVIER, vol. 18, no. 7, 1 July 2010 (2010-07-01), pages 1357 - 1364, XP055016290, ISSN: 15250016, DOI: 10.1038/mt.2010.85
- JUDGE ADAM D ET AL: "Confirming the RNAi-mediated mechanism of action of siRNA-based cancer therapeutics in mice", JOURNAL OF CLINICAL INVESTIGATION, AMERICAN SOCIETY FOR CLINICAL INVESTIGATION, UNITED STATES, vol. 119, no. 3, 1 March 2009 (2009-03-01), United States, pages 661 - 673, XP002618489, ISSN: 0021-9738, DOI: 10.1172/JCI37515
- EGRIE JOAN C, DWYER ERIK, BROWNE JEFFREY K, HITZ ANNA, LYKOS MICHELE A: "Darbepoetin alfa has a longer circulating half-life and greater in vivo potency than recombinant human erythropoietin", EXPERIMENTAL HEMATOLOGY, ELSEVIER INC., US, vol. 31, no. 4, 1 April 2003 (2003-04-01), US , pages 290 - 299, XP093440002, ISSN: 0301-472X, DOI: 10.1016/s0301-472x(03)00006-7
- FRIED, W.: "Erythropoietin and erythropoiesis", EXPERIMENTAL HEMATOLOGY, ELSEVIER INC., US, vol. 37, no. 9, 1 September 2009 (2009-09-01), US , pages 1007 - 1015, XP026459229, ISSN: 0301-472X, DOI: 10.1016/j.exphem.2009.05.010
- NEY PAUL A: "Author manuscript: Normal and disordered reticulocyte maturation", CURRENT OPINION IN HEMATOLOGY, RAPID SCIENCE PUBLISHERS, PHILADELPHIA, PA., US, vol. 18, no. 3, 1 May 2012 (2012-05-01), US , pages 1 - 12, XP093440006, ISSN: 1065-6251, DOI: 10.1097/moh.0b013e328345213e
- VAN PUTTEN L., CROON FINEKE: "The Life Span of Red Cells in the Rat and the Mouse as Determined by Labeling with DFP32 in Vivo", BLOOD, ELSEVIER INC, UNITED STATES, vol. 13, no. 8, 1 August 1958 (1958-08-01), United States, pages 789 - 794, XP093440008, ISSN: 0006-4971, DOI: 10.1182/blood.v13.8.789.789
- R. T. PETERS, S. C. LOW, G. D. KAMPHAUS, J. A. DUMONT, J. V. AMARI, Q. LU, G. ZARBIS-PAPASTOITSIS, T. J. REIDY, E. P. MERRICKS, T.: "Prolonged activity of factor IX as a monomeric Fc fusion protein", BLOOD, vol. 115, no. 10, 11 March 2010 (2010-03-11), pages 2057 - 2064, XP055087904, ISSN: 00064971, DOI: 10.1182/blood-2009-08-239665
- JONES E. A., VERGALLA J., STEER C. J., BRADLEY-MOORE P. R., VIERLING J. M.: "Metabolism of Intact and Desialylated α1-Antitrypsin", CLINICAL SCIENCE., BIOCHEMICAL SOCIETY AND THE MEDICAL RESEARCH SOCIETY, LONDON,, GB, vol. 55, no. 2, 1 August 1978 (1978-08-01), GB , pages 139 - 148, XP093440010, ISSN: 0143-5221, DOI: 10.1042/cs0550139
- NURIA PADULLES, ZAMORA RAFAEL, VIDAL PIA, GISPERT PILAR, RIO DEL, MARGALEFF ROSENDO JARDI, FRIAS FRANCISCO RODRIGUEZ, BRUONO JOSE,: "Intravenous Human Plasma-Derived Augmentation Therapy in a.-Antitrypsin Deficiency: From Pharmacokinetic Analysis to Individualizing Therapy", ANNALS OF PHARMACOTHERAPY, SAGE PUBLICATIONS, LOS ANGELES, CA, vol. 42, no. 5, 1 May 2008 (2008-05-01), Los Angeles, CA, pages 640 - 646, XP093440014, ISSN: 1060-0280, DOI: 10.1345/aph.1K505
- SUDA TAKESHI, KAMIMURA KENYA, KUBOTA TOMOYUKI, TAMURA YASUSHI, IGARASHI MASATO, KAWAI HIROKAZU, AOYAGI YUTAKA, LIU DEXI: "Progress toward liver‐based gene therapy", HEPATOLOGY RESEARCH, AMSTERDAM, NL, vol. 39, no. 4, 1 April 2009 (2009-04-01), NL , pages 325 - 340, XP093440015, ISSN: 1386-6346, DOI: 10.1111/j.1872-034x.2008.00479.x
- YU XIAOBING; LIN CHYUAN-SHENG; COSTANTINI FRANK; NOGUCHI CONSTANCE TOM: "The human erythropoietin receptor gene rescues erythropoiesis and developmental defects in the erythropoietin receptor null mouse", BLOOD, AMSTERDAM, NL, vol. 98, no. 2, 15 July 2001 (2001-07-15), AMSTERDAM, NL, pages 475 - 477, XP086704274, DOI: 10.1182/blood.V98.2.475
- G. L. SEMENZA, TRAYSTMAN MONICA D, GEARHARTT JOHN D, ANTONARAKIS STYLIANOS E: "Polycythemia in transgenic mice expressing the human erythropoietin gene.", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES (PNAS), NATIONAL ACADEMY OF SCIENCES, vol. 86, no. 7, 1 April 1989 (1989-04-01), pages 2301 - 2305, XP055233828, ISSN: 0027-8424, DOI: 10.1073/pnas.86.7.2301
- JOERG SCHUETTRUMPF, ET AL.: "Factor IX variants improve gene therapy efficacy for hemophilia B", BLOOD, vol. 105, no. 6, 15 March 2005 (2005-03-15), pages 2316 - 2323, XP055542043, DOI: 10.1182/blood-2004-08-2990
- OGAWA SHINGO, ONODERA JUN, HONDA RIKA, FUJIMOTO ICHIRO: "Influence of systemic administration of atelocollagen on mouse livers: an ideal biomaterial for systemic drug delivery", THE JOURNAL OF TOXICOLOGICAL SCIENCES, TOKYO., JP, vol. 36, no. 6, 1 December 2011 (2011-12-01), JP , pages 751 - 762, XP093440017, ISSN: 0388-1350, DOI: 10.2131/jts.36.751