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1. (WO2017139758) RNA/DNA HYBRID NANOPARTICLES MODIFIED WITH SINGLE STRANDED RNA TOEHOLDS AND USES THEREOF
Latest bibliographic data on file with the International Bureau

Pub. No.: WO/2017/139758 International Application No.: PCT/US2017/017661
Publication Date: 17.08.2017 International Filing Date: 13.02.2017
IPC:
C12N 15/113 (2010.01) ,B82Y 5/00 (2011.01) ,A61K 47/55 (2017.01) ,C12N 15/87 (2006.01)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
B PERFORMING OPERATIONS; TRANSPORTING
82
NANO-TECHNOLOGY
Y
SPECIFIC USES OR APPLICATIONS OF NANO-STRUCTURES; MEASUREMENT OR ANALYSIS OF NANO-STRUCTURES; MANUFACTURE  OR TREATMENT OF NANO-STRUCTURES
5
Nano-biotechnology or nano-medicine, e.g. protein engineering or drug delivery
[IPC code unknown for A61K 47/55]
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
87
Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
Applicants:
THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES [US/US]; National Institute of Health Office of Technology Transfer 6011 Executive Boulevard, Suite 325 Rockville, MD 20852, US
Inventors:
SHAPIRO, Bruce, Allen; US
AFONIN, Kirill, Andreevich; US
VIARD, Mathias, D.; US
BINDEWALD, Eckart, H.U.; US
Agent:
MCCOOL, Gabriel, J.; US
FENN, Edward, A.; US
MCCUE, Patrick, P.; US
FERGUSON, Steven; US
NGUYEN-ANTCZAK, Lauren; US
Priority Data:
62/294,84812.02.2016US
Title (EN) RNA/DNA HYBRID NANOPARTICLES MODIFIED WITH SINGLE STRANDED RNA TOEHOLDS AND USES THEREOF
(FR) NANOPARTICULES HYBRIDES D'ARN/ADN MODIFIÉES AVEC DES ANCRAGES D'ARN SIMPLE BRIN ET LEURS UTILISATIONS
Abstract:
(EN) The invention discloses the use of single- stranded RNA toeholds of different lengths to promote the re-association of various RNA-DNA hybrids, which results in activation of multiple split functionalities inside human cells. Previously designed RNA/DNA nanoparticles employed single- stranded DNA toeholds to initiate re-association. The use of RNA toeholds is advantageous because of the simpler design rules, the shorter toeholds, and the smaller size of the resulting nanoparticles compared to the same hybrid nanoparticles with single-stranded DNA toeholds. Moreover, the co-transcriptional assemblies result in higher yields for hybrid nanoparticles with ssRNA toeholds.
(FR) La présente invention décrit l'utilisation d'ancrages d'ARN simple brin de différentes longueurs pour favoriser la ré-association de divers hybrides d'ARN-ADN, qui résulte en l'activation de fonctionnalités de partage multiple à l'intérieur de cellules humaines. Les nanoparticules d'ARN/ADN précédemment conçues utilisaient des ancrages d'ADN simple brin pour initier la ré-association. L'utilisation des ancrages d'ARN est avantageuse du fait des règles de conception plus simples, des ancrages plus courts, et de la taille plus petite des nanoparticules résultantes comparées aux mêmes nanoparticules hybrides avec des ancrages d'ADN simple brin. En outre, les assemblages co-transcriptionnels résultent en rendements supérieurs pour les nanoparticules hybrides avec des ancrages d'ARNsb.
Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Organization (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)
Also published as:
EP3414329US20190192687