Title:
FOLDING MRNA INTO A NANOSCALE DELIVERY VEHICLE
Document Type and Number:
WIPO Patent Application WO/2023/133546
Kind Code:
A3
Abstract:
Described herein is a DNA nanotechnology-based platform for the delivery of nucleic acids into cells. The delivery platform relies on compacting cargo mRNA and/or ssDNA into single folded nanostructures that act as the primary structural vehicle material for nanoparticle delivery to cells. The described compositions and methods provide an alternative to packaging mRNA or ssDNA into lipid nanoparticle vehicles, which have complex formulation properties. Also described herein are strategies for protecting mRNA and/or ssDNA cargo and helping these nucleic acid molecules escape the endosome into the cytoplasm using a cationic peptide-PEG coating.
Inventors:
STEPHANOPOULOS NICHOLAS (US)
ANDERSON KAREN SUE (US)
SULC PETR (US)
HENRY SKYLER JENNIFER WRAY (US)
CHEN LIANGXIAO (US)
POPPLETON ERIK ALEXANDER (US)
YAN HAO (US)
ANDERSON KAREN SUE (US)
SULC PETR (US)
HENRY SKYLER JENNIFER WRAY (US)
CHEN LIANGXIAO (US)
POPPLETON ERIK ALEXANDER (US)
YAN HAO (US)
Application Number:
PCT/US2023/060296
Publication Date:
September 07, 2023
Filing Date:
January 09, 2023
Export Citation:
Assignee:
UNIV ARIZONA STATE (US)
International Classes:
A61K9/16; A61K9/14; A61K9/50; A61K48/00; A61K9/127
Foreign References:
| US20210330600A1 | 2021-10-28 | |||
| US20070037246A1 | 2007-02-15 | |||
| US20080213377A1 | 2008-09-04 | |||
| US20090156499A1 | 2009-06-18 | |||
| US20160090603A1 | 2016-03-31 |
Other References:
WU XIAOHUI, LIU QING, LIU FENGSONG, WU TIANTIAN, SHANG YINGXU, LIU JIANBING, DING BAOQUAN: "An RNA/DNA hybrid origami-based nanoplatform for efficient gene therapy", NANOSCALE, vol. 13, no. 30, 5 August 2021 (2021-08-05), United Kingdom , pages 12848 - 12853, XP055878817, ISSN: 2040-3364, DOI: 10.1039/D1NR00517K
STEFAN NIEKAMP, KATY BLUMER, PARSA M. NAFISI, KATHY TSUI, JOHN GARBUTT, SHAWN M. DOUGLAS: "Folding complex DNA nanostructures from limited sets of reusable sequences", NUCLEIC ACIDS RESEARCH, vol. 44, no. 11, 20 June 2016 (2016-06-20), GB , pages e102 - e102, XP055670680, ISSN: 0305-1048, DOI: 10.1093/nar/gkw208
RUSSELL N. JOHNSON; DAVID S.H. CHU; JULIE SHI; JOAN G. SCHELLINGER; PETER M. CARLSON; SUZIE H. PUN;: "HPMA-oligolysine copolymers for gene delivery: Optimization of peptide length and polymer molecular weight", JOURNAL OF CONTROLLED RELEASE, vol. 155, no. 2, 5 July 2011 (2011-07-05), AMSTERDAM, NL , pages 303 - 311, XP028315159, ISSN: 0168-3659, DOI: 10.1016/j.jconrel.2011.07.009
MO LI, MENGXI ZHENG, SIYU WU, CHENG TIAN, DI LIU, YOSSI WEIZMANN, WEN JIANG, GUANSONG WANG, CHENGDE MAO: "In vivo production of RNA nanostructures via programmed folding of single-stranded RNAs", NATURE COMMUNICATIONS, vol. 9, no. 1, 1 December 2018 (2018-12-01), XP055579881, DOI: 10.1038/s41467-018-04652-4
BRUNO P MELONI, LAURA M BROOKES, VINCE W CLARK, JANE L CROSS, ADAM B EDWARDS, RYAN S ANDERTON, RICHARD M HOPKINS, KATRIN HOFFMANN,: "Poly-Arginine and Arginine-Rich Peptides are Neuroprotective in Stroke Models", JOURNAL OF CEREBRAL BLOOD FLOW & METABOLISM, vol. 35, no. 6, 1 June 2015 (2015-06-01), US , pages 993 - 1004, XP055415410, ISSN: 0271-678X, DOI: 10.1038/jcbfm.2015.11
ZHOU LIFENG, CHANDRASEKARAN ARUN RICHARD, YAN MENGWEN, VALSANGKAR VIBHAV A., FELDBLYUM JEREMY I., SHENG JIA, HALVORSEN KEN: "A mini DNA–RNA hybrid origami nanobrick", NANOSCALE ADVANCES, vol. 3, no. 14, 13 July 2021 (2021-07-13), pages 4048 - 4051, XP055878803, DOI: 10.1039/D1NA00026H
STEFAN NIEKAMP, KATY BLUMER, PARSA M. NAFISI, KATHY TSUI, JOHN GARBUTT, SHAWN M. DOUGLAS: "Folding complex DNA nanostructures from limited sets of reusable sequences", NUCLEIC ACIDS RESEARCH, vol. 44, no. 11, 20 June 2016 (2016-06-20), GB , pages e102 - e102, XP055670680, ISSN: 0305-1048, DOI: 10.1093/nar/gkw208
RUSSELL N. JOHNSON; DAVID S.H. CHU; JULIE SHI; JOAN G. SCHELLINGER; PETER M. CARLSON; SUZIE H. PUN;: "HPMA-oligolysine copolymers for gene delivery: Optimization of peptide length and polymer molecular weight", JOURNAL OF CONTROLLED RELEASE, vol. 155, no. 2, 5 July 2011 (2011-07-05), AMSTERDAM, NL , pages 303 - 311, XP028315159, ISSN: 0168-3659, DOI: 10.1016/j.jconrel.2011.07.009
MO LI, MENGXI ZHENG, SIYU WU, CHENG TIAN, DI LIU, YOSSI WEIZMANN, WEN JIANG, GUANSONG WANG, CHENGDE MAO: "In vivo production of RNA nanostructures via programmed folding of single-stranded RNAs", NATURE COMMUNICATIONS, vol. 9, no. 1, 1 December 2018 (2018-12-01), XP055579881, DOI: 10.1038/s41467-018-04652-4
BRUNO P MELONI, LAURA M BROOKES, VINCE W CLARK, JANE L CROSS, ADAM B EDWARDS, RYAN S ANDERTON, RICHARD M HOPKINS, KATRIN HOFFMANN,: "Poly-Arginine and Arginine-Rich Peptides are Neuroprotective in Stroke Models", JOURNAL OF CEREBRAL BLOOD FLOW & METABOLISM, vol. 35, no. 6, 1 June 2015 (2015-06-01), US , pages 993 - 1004, XP055415410, ISSN: 0271-678X, DOI: 10.1038/jcbfm.2015.11
ZHOU LIFENG, CHANDRASEKARAN ARUN RICHARD, YAN MENGWEN, VALSANGKAR VIBHAV A., FELDBLYUM JEREMY I., SHENG JIA, HALVORSEN KEN: "A mini DNA–RNA hybrid origami nanobrick", NANOSCALE ADVANCES, vol. 3, no. 14, 13 July 2021 (2021-07-13), pages 4048 - 4051, XP055878803, DOI: 10.1039/D1NA00026H
Attorney, Agent or Firm:
BROWN, Bernard A., II. (US)
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