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WO2023081377A3 - Compositions comprising an rna guide targeting ciita and uses thereof - Google Patents

Compositions comprising an rna guide targeting ciita and uses thereof Download PDF

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Publication number
WO2023081377A3
WO2023081377A3 PCT/US2022/048989 US2022048989W WO2023081377A3 WO 2023081377 A3 WO2023081377 A3 WO 2023081377A3 US 2022048989 W US2022048989 W US 2022048989W WO 2023081377 A3 WO2023081377 A3 WO 2023081377A3
Authority
WO
WIPO (PCT)
Prior art keywords
compositions
rna guide
sequence
ciita
rna
Prior art date
Application number
PCT/US2022/048989
Other languages
French (fr)
Other versions
WO2023081377A2 (en
Inventor
Quinton Norman WESSELLS
Tia Marie DITOMMASO
Jeffrey Raymond HASWELL
Noah Michael Jakimo
Original Assignee
Arbor Biotechnologies, Inc.
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 Arbor Biotechnologies, Inc. filed Critical Arbor Biotechnologies, Inc.
Publication of WO2023081377A2 publication Critical patent/WO2023081377A2/en
Publication of WO2023081377A3 publication Critical patent/WO2023081377A3/en

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The present invention relates to compositions comprising RNA guides targeting CIITA, processes for characterizing the compositions, cells comprising the compositions, and methods of using the compositions. The invention provides a composition comprising an RNA guide, wherein the RNA guide comprises (i) a spacer sequence that is substantially complementary to a target sequence within a CIITA gene and (ii) a direct repeat sequence; wherein the target sequence is adjacent to a protospacer adjacent motif (PAM) comprising the sequence 5 '-NTTN-3'.
PCT/US2022/048989 2021-11-05 2022-11-04 Compositions comprising an rna guide targeting ciita and uses thereof WO2023081377A2 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202163276229P 2021-11-05 2021-11-05
US63/276,229 2021-11-05
US202163294121P 2021-12-28 2021-12-28
US63/294,121 2021-12-28

Publications (2)

Publication Number Publication Date
WO2023081377A2 WO2023081377A2 (en) 2023-05-11
WO2023081377A3 true WO2023081377A3 (en) 2023-09-14

Family

ID=86242120

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2022/048989 WO2023081377A2 (en) 2021-11-05 2022-11-04 Compositions comprising an rna guide targeting ciita and uses thereof

Country Status (2)

Country Link
TW (1) TW202334421A (en)
WO (1) WO2023081377A2 (en)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021158918A1 (en) * 2020-02-05 2021-08-12 Danmarks Tekniske Universitet Compositions and methods for targeting, editing or modifying human genes
WO2021202800A1 (en) * 2020-03-31 2021-10-07 Arbor Biotechnologies, Inc. Compositions comprising a cas12i2 variant polypeptide and uses thereof
WO2023039534A2 (en) * 2021-09-10 2023-03-16 Arbor Biotechnologies, Inc. Compositions comprising a cas12i polypeptide and uses thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021158918A1 (en) * 2020-02-05 2021-08-12 Danmarks Tekniske Universitet Compositions and methods for targeting, editing or modifying human genes
WO2021202800A1 (en) * 2020-03-31 2021-10-07 Arbor Biotechnologies, Inc. Compositions comprising a cas12i2 variant polypeptide and uses thereof
WO2023039534A2 (en) * 2021-09-10 2023-03-16 Arbor Biotechnologies, Inc. Compositions comprising a cas12i polypeptide and uses thereof

Also Published As

Publication number Publication date
TW202334421A (en) 2023-09-01
WO2023081377A2 (en) 2023-05-11

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