WO2026083309A1 - Utilisation d'inhibiteurs ou de modulateurs du gène artémis dans la réécriture génomique - Google Patents

Utilisation d'inhibiteurs ou de modulateurs du gène artémis dans la réécriture génomique

Info

Publication number
WO2026083309A1
WO2026083309A1 PCT/IB2025/060538 IB2025060538W WO2026083309A1 WO 2026083309 A1 WO2026083309 A1 WO 2026083309A1 IB 2025060538 W IB2025060538 W IB 2025060538W WO 2026083309 A1 WO2026083309 A1 WO 2026083309A1
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WO
WIPO (PCT)
Prior art keywords
polynucleotide
dna
sequence
inhibitor
protein
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
PCT/IB2025/060538
Other languages
English (en)
Inventor
Louis DACQUAY
Oi Kuan CHOONG
Nina AKRAP
Martin PETERKA
Marcello Maresca
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
AstraZeneca AB
Original Assignee
AstraZeneca AB
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by AstraZeneca AB filed Critical AstraZeneca AB
Publication of WO2026083309A1 publication Critical patent/WO2026083309A1/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/332Abasic residue

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La présente divulgation concerne des modulateurs ou des inhibiteurs du gène Artémis et leurs utilisations pour favoriser l'intégration d'une séquence dans un locus génomique cible d'une cellule par réécriture par matrice d'ARN à médiation par l'assemblage d'extrémités non homologues.
PCT/IB2025/060538 2024-10-17 2025-10-16 Utilisation d'inhibiteurs ou de modulateurs du gène artémis dans la réécriture génomique Pending WO2026083309A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP24207321 2024-10-17
EP24207321.1 2024-10-17

Publications (1)

Publication Number Publication Date
WO2026083309A1 true WO2026083309A1 (fr) 2026-04-23

Family

ID=93154418

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2025/060538 Pending WO2026083309A1 (fr) 2024-10-17 2025-10-16 Utilisation d'inhibiteurs ou de modulateurs du gène artémis dans la réécriture génomique

Country Status (1)

Country Link
WO (1) WO2026083309A1 (fr)

Citations (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5855913A (en) 1997-01-16 1999-01-05 Massachusetts Instite Of Technology Particles incorporating surfactants for pulmonary drug delivery
US5895309A (en) 1998-02-09 1999-04-20 Spector; Donald Collapsible hula-hoop
US6007845A (en) 1994-07-22 1999-12-28 Massachusetts Institute Of Technology Nanoparticles and microparticles of non-linear hydrophilic-hydrophobic multiblock copolymers
WO2004045543A2 (fr) * 2002-11-14 2004-06-03 Dharmacon, Inc. Arnsi fonctionnel et hyperfonctionnel
WO2011005765A1 (fr) * 2009-07-06 2011-01-13 Alnylam Pharmaceuticals, Inc. Biotraitement
US20110293703A1 (en) 2008-11-07 2011-12-01 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
US20120251560A1 (en) 2011-03-28 2012-10-04 Massachusetts Institute Of Technology Conjugated lipomers and uses thereof
US20130302401A1 (en) 2010-08-26 2013-11-14 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
US20140068797A1 (en) 2012-05-25 2014-03-06 University Of Vienna Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
US8709843B2 (en) 2006-08-24 2014-04-29 Rohm Co., Ltd. Method of manufacturing nitride semiconductor and nitride semiconductor element
US8771945B1 (en) 2012-12-12 2014-07-08 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2014118272A1 (fr) 2013-01-30 2014-08-07 Santaris Pharma A/S Conjugués glucidiques d'oligonucléotides antimir-22
US20140273037A1 (en) 2013-03-15 2014-09-18 System Biosciences, Llc Compositions and methods directed to crispr/cas genomic engineering systems
US20140295556A1 (en) 2013-03-15 2014-10-02 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to Increase Specificity for RNA-Guided Genome Editing
US20140349405A1 (en) 2013-05-22 2014-11-27 Wisconsin Alumni Research Foundation Rna-directed dna cleavage and gene editing by cas9 enzyme from neisseria meningitidis
WO2015006498A2 (fr) * 2013-07-09 2015-01-15 President And Fellows Of Harvard College Utilisations thérapeutiques d'édition du génome avec des systèmes crispr/cas
US20150045546A1 (en) 2012-03-20 2015-02-12 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US20150071898A1 (en) 2013-09-06 2015-03-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US20150071906A1 (en) 2013-09-06 2015-03-12 President And Fellows Of Harvard College Delivery system for functional nucleases
US9023649B2 (en) 2012-12-17 2015-05-05 President And Fellows Of Harvard College RNA-guided human genome engineering
US20160208243A1 (en) 2015-06-18 2016-07-21 The Broad Institute, Inc. Novel crispr enzymes and systems
US9580701B2 (en) 2015-01-28 2017-02-28 Pioneer Hi-Bred International, Inc. CRISPR hybrid DNA/RNA polynucleotides and methods of use
WO2019099943A1 (fr) 2017-11-16 2019-05-23 Astrazeneca Ab Compositions et méthodes pour améliorer l'efficacité de stratégies knock-in basées sur cas9
US20200087640A1 (en) 2017-11-01 2020-03-19 The Regents Of The University Of California Casz compositions and methods of use
WO2022214522A2 (fr) 2021-04-07 2022-10-13 Astrazeneca Ab Compositions et procédés de modification spécifique à un site
WO2023172115A1 (fr) * 2022-03-10 2023-09-14 주식회사 진코어 Compositions et procédés pour augmenter l'efficacité de délétion d'un segment d'acide nucléique par modulation de la voie de réparation nhej

Patent Citations (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US6007845A (en) 1994-07-22 1999-12-28 Massachusetts Institute Of Technology Nanoparticles and microparticles of non-linear hydrophilic-hydrophobic multiblock copolymers
US5855913A (en) 1997-01-16 1999-01-05 Massachusetts Instite Of Technology Particles incorporating surfactants for pulmonary drug delivery
US5895309A (en) 1998-02-09 1999-04-20 Spector; Donald Collapsible hula-hoop
WO2004045543A2 (fr) * 2002-11-14 2004-06-03 Dharmacon, Inc. Arnsi fonctionnel et hyperfonctionnel
US8709843B2 (en) 2006-08-24 2014-04-29 Rohm Co., Ltd. Method of manufacturing nitride semiconductor and nitride semiconductor element
US20110293703A1 (en) 2008-11-07 2011-12-01 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
WO2011005765A1 (fr) * 2009-07-06 2011-01-13 Alnylam Pharmaceuticals, Inc. Biotraitement
US20130302401A1 (en) 2010-08-26 2013-11-14 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
US20120251560A1 (en) 2011-03-28 2012-10-04 Massachusetts Institute Of Technology Conjugated lipomers and uses thereof
US20150045546A1 (en) 2012-03-20 2015-02-12 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US20140068797A1 (en) 2012-05-25 2014-03-06 University Of Vienna Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
US10407697B2 (en) 2012-05-25 2019-09-10 The Regents Of The University Of California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
US10000772B2 (en) 2012-05-25 2018-06-19 The Regents Of The University Of California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
US8771945B1 (en) 2012-12-12 2014-07-08 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
US9023649B2 (en) 2012-12-17 2015-05-05 President And Fellows Of Harvard College RNA-guided human genome engineering
WO2014118272A1 (fr) 2013-01-30 2014-08-07 Santaris Pharma A/S Conjugués glucidiques d'oligonucléotides antimir-22
US20140295557A1 (en) 2013-03-15 2014-10-02 The General Hospital Corporation Using Truncated Guide RNAs (tru-gRNAs) to Increase Specificity for RNA-Guided Genome Editing
US20140273037A1 (en) 2013-03-15 2014-09-18 System Biosciences, Llc Compositions and methods directed to crispr/cas genomic engineering systems
US20140295556A1 (en) 2013-03-15 2014-10-02 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to Increase Specificity for RNA-Guided Genome Editing
US20140273226A1 (en) 2013-03-15 2014-09-18 System Biosciences, Llc Crispr/cas systems for genomic modification and gene modulation
US20140349405A1 (en) 2013-05-22 2014-11-27 Wisconsin Alumni Research Foundation Rna-directed dna cleavage and gene editing by cas9 enzyme from neisseria meningitidis
WO2015006498A2 (fr) * 2013-07-09 2015-01-15 President And Fellows Of Harvard College Utilisations thérapeutiques d'édition du génome avec des systèmes crispr/cas
US20150071898A1 (en) 2013-09-06 2015-03-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US20150071906A1 (en) 2013-09-06 2015-03-12 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150071899A1 (en) 2013-09-06 2015-03-12 President And Fellows Of Harvard College Cas9-foki fusion proteins and uses thereof
US9580701B2 (en) 2015-01-28 2017-02-28 Pioneer Hi-Bred International, Inc. CRISPR hybrid DNA/RNA polynucleotides and methods of use
US20160208243A1 (en) 2015-06-18 2016-07-21 The Broad Institute, Inc. Novel crispr enzymes and systems
US20200087640A1 (en) 2017-11-01 2020-03-19 The Regents Of The University Of California Casz compositions and methods of use
WO2019099943A1 (fr) 2017-11-16 2019-05-23 Astrazeneca Ab Compositions et méthodes pour améliorer l'efficacité de stratégies knock-in basées sur cas9
WO2022214522A2 (fr) 2021-04-07 2022-10-13 Astrazeneca Ab Compositions et procédés de modification spécifique à un site
WO2023172115A1 (fr) * 2022-03-10 2023-09-14 주식회사 진코어 Compositions et procédés pour augmenter l'efficacité de délétion d'un segment d'acide nucléique par modulation de la voie de réparation nhej

Non-Patent Citations (69)

* Cited by examiner, † Cited by third party
Title
ALACH ET AL., BIORXIV, 2014
ALTSCHUL ET AL., J MOL BIOL, vol. 215, 1990, pages 403 - 410
ALTSCHUL ET AL., NUCLEIC ACIDS RES, vol. 25, no. 17, 1997, pages 3389 - 3402
ALVAREZ-ERVITI ET AL., NATURE BIOTECHNOLOGY, vol. 29, 2011, pages 341
ANZALONE ET AL., NATURE, vol. 576, 2019, pages 149 - 157
CHYLINSKI ET AL., RNA BIOL, vol. 10, no. 5, 2013, pages 726 - 737
CROOKE ET AL., J. PHARMACOL. EXP. THER., vol. 277, 1996, pages 923 - 937
DACQUAY LOUIS C. ET AL: "Artemis Regulates Homology-Independent Prime Editing (PRINS) for Enhanced Genomic Insertions", BIORXIV, 8 December 2025 (2025-12-08), XP093357733, Retrieved from the Internet <URL:https://www.biorxiv.org/content/10.64898/2025.12.08.693005v1> [retrieved on 20260127], DOI: 10.64898/2025.12.08.693005 *
EL-ANDALOUSSI ET AL., NATURE PROTOCOLS, vol. 7, 2012, pages 2112 - 2116
GARLAND SCIENCE, 2002
GASIUNAS ET AL., NAT COMM, vol. 11, 2020, pages 5512
GLISOVIC ET AL., FEBS LETT, vol. 582, no. 14, 2008, pages 1977 - 1986
GUIBLET ET AL., NUCLEIC ACIDS RES, vol. 49, no. 3, 2021, pages 1497 - 1516
HAI LIU ET AL: "The dominant negative mutant Artemis enhances tumor cell radiosensitivity", RADIOTHERAPY AND ONCOLOGY, ELSEVIER, IRELAND, vol. 101, no. 1, 11 May 2011 (2011-05-11), pages 66 - 72, XP028103017, ISSN: 0167-8140, [retrieved on 20110518], DOI: 10.1016/J.RADONC.2011.05.034 *
HARRINGTON ET AL., SCIENCE, vol. 362, 2018, pages 839 - 842
HSU ET AL., NAT BIOTECHNOL, vol. 31, no. 9, 2013, pages 827 - 832
ISHINO ET AL., JOURNAL OF BACTERIOLOGY, vol. 169, no. 12, 1987, pages 5429 - 5433
JINEK ET AL., SCIENCE, vol. 337, no. 6096, 2012, pages 816 - 821
KABANOV ET AL., FEBS LETT., vol. 259, 1990, pages 327 - 330
KARLINALTSCHUL, PROC NAT ACAD SCI, vol. 87, 1990, pages 2264 - 2268
KARLINALTSCHUL, PROC NAT ACAD SCI, vol. 90, 1993, pages 5873 - 5877
KOONIN ET AL., PHIL TRANS R SOC B, vol. 374, 2018, pages 20180087
LABUN ET AL., BIORXIV, 2018
LABUN ET AL., NUCLEIC ACIDS RES, 2016
LEE ADRIAN B.C. ET AL: "Small-molecule enhancers of CRISPR-induced homology-directed repair in gene therapy: A medicinal chemist's perspective", DRUG DISCOVERY TODAY, vol. 27, no. 9, 1 September 2022 (2022-09-01), AMSTERDAM, NL, pages 2510 - 2525, XP093357765, ISSN: 1359-6446, Retrieved from the Internet <URL:https://www.sciencedirect.com/science/article/pii/S1359644622002768> [retrieved on 20260127], DOI: 10.1016/j.drudis.2022.06.006 *
LEE ET AL., PNAS, 2014
LETSINGER ET AL., PROC. NATL. ACAD. SCI., vol. 86, 1989, pages 6553 - 6556
LI ET AL., GENE THERAPY, vol. 19, 2012, pages 775 - 780
LI ET AL., JBC, vol. 289, no. 11, 2014, pages 7825 - 7834
LI XIANGYANG ET AL: "Development of a versatile nuclease prime editor with upgraded precision", NATURE COMMUNICATIONS, vol. 14, no. 1, 19 January 2023 (2023-01-19), UK, pages 305 - 1, XP093350243, ISSN: 2041-1723, Retrieved from the Internet <URL:https://www.nature.com/articles/s41467-023-35870-0> [retrieved on 20260127], DOI: 10.1038/s41467-023-35870-0 *
LI XIANGYANG ET AL: "Supplementary information to Development of a versatile nuclease prime editor with upgraded precision", NATURE COMMUNICATIONS, VOL. 14, 19 January 2023 (2023-01-19), XP093358516, Retrieved from the Internet <URL:https://www.nature.com/articles/s41467-023-35870-0#Sec23> [retrieved on 20260127], DOI: 10.1038/s41467-023-35870-0 *
LINO ET AL., DRUG DELIVERY, vol. 25, no. 1, 2018, pages 1234 - 1257
LINO ET AL., DRUG DELIVERY, vol. 25, no. 1, pages 1234 - 1257
LUNDE ET AL., NAT REV MOL CELL BIOL, vol. 8, no. 6, 2007, pages 479 - 490
MAKAROVA ET AL., METHODS MOL BIOL, vol. 1311, 2015, pages 47 - 75
MAKAROVA ET AL., THE CRISPR JOURNAL, October 2018 (2018-10-01), pages 325 - 336
MALI ET AL., NAT METHODS, vol. 10, 2013, pages 957 - 63
MALI ET AL., SCIENCE, vol. 339, no. 6121, 2013, pages 823 - 826
MANOHARAN ET AL., ANN. N.Y. ACAD. SCI., vol. 660, 1992, pages 306 - 309
MANOHARAN ET AL., BIOORG. MED. CHEM. LET., vol. 3, 1993, pages 2765 - 2770
MANOHARAN ET AL., BIOORG. MED. CHEM. LET., vol. 4, 1994, pages 1053 - 1060
MANOHARAN ET AL., NUCLEOSIDES & NUCLEOTIDES, vol. 14, 1995, pages 969 - 973
MANOHARAN ET AL., TETRAHEDRON LETT., vol. 36, 1995, pages 3651 - 3654
MISHRA ET AL., BIOCHIM. BIOPHYS. ACTA, vol. 1264, 1995, pages 229 - 237
MITRA ET AL., MATER METHODS, vol. 3, 2013, pages 204
MORRISSEY ET AL., NATURE BIOTECHNOLOGY, vol. 23, no. 8, 2005, pages 1002 - 1007
NAIR ET AL., J. AM. CHEM. SOC., vol. 136, no. 49, 2014, pages 16958 - 16961
OBERHAUSER ET AL., ACIDS RES., vol. 20, 1992, pages 533 - 538
PETERKA MARTIN ET AL: "Harnessing DSB repair to promote efficient homology-dependent and -independent prime editing", NATURE COMMUNICATIONS, 24 March 2022 (2022-03-24), England, pages 1240 - 1240, XP093139697, Retrieved from the Internet <URL:https://www.nature.com/articles/s41467-022-28771-1.pdf> [retrieved on 20240311], DOI: 10.1038/s41467-022-28771-1 *
RUEDA ET AL., NAT COMM, vol. 8, 2017, pages 1610
SAISON-BEHMOARAS ET AL., EMBO J., vol. 10, 1991, pages 1111 - 1118
SANDER ET AL., NAT BIOTECHNOL, vol. 32, 2014, pages 347 - 355
SANDER ET AL., NATURE BIOTECHNOLOGY, vol. 32, 2014, pages 347 - 355
SHEA ET AL., NUCL. ACIDS RES., vol. 18, 1990, pages 3777 - 3783
SHY ET AL., BIORXIV, 2021
SORET ET AL., NATURE REVIEWS MICROBIOLOGY, vol. 6, no. 3, 2008, pages 181 - 186
SPUCHNAVARRO, JOURNAL OF DRUG DELIVERY, 2011
STROBEL ET AL., BIORXIV, 23 January 2020 (2020-01-23)
SU ET AL., MOLECULAR PHARMACOLOGY, vol. 8, no. 3, 2011, pages 774 - 784
SUN ET AL., J. AM. CHEM. SOC., vol. 136, pages 14722 - 14725
SVINARCHUK ET AL., BIOCHIMIE, vol. 75, 1993, pages 49 - 54
VIDANGOS ET AL., BIOPOLYMERS, vol. 99, no. 12, 2013, pages 1082 - 1096
WAHLGREN ET AL., NUCLEIC ACIDS RESEARCH, vol. 40, no. 17, 2012, pages 130
WALS ET AL., FRONT CHEM, vol. 2, 2014, pages 15
YESUDHAS ET AL., GENES, vol. 8, no. 8, 2017, pages 192
YOKOYAMA ET AL., INT J MOL SCI, vol. 15, no. 11, 2014, pages 20321 - 20338
YOSAATMADJA ET AL., NUCLEIC ACIDS RESEARCH, vol. 49, no. 16, 2021, pages 9310 - 9326
ZETSCHE ET AL., CELL, vol. 163, no. 3, 2015, pages 759 - 771
ZIMMERMAN ET AL., NATURE LETTERS, vol. 441, 2006, pages 111 - 114

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