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WO2023225471A3 - Helitron compositions and methods - Google Patents

Helitron compositions and methods Download PDF

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Publication number
WO2023225471A3
WO2023225471A3 PCT/US2023/066986 US2023066986W WO2023225471A3 WO 2023225471 A3 WO2023225471 A3 WO 2023225471A3 US 2023066986 W US2023066986 W US 2023066986W WO 2023225471 A3 WO2023225471 A3 WO 2023225471A3
Authority
WO
WIPO (PCT)
Prior art keywords
compositions
sequence
heterologous
modifying polypeptide
helitron
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.)
Ceased
Application number
PCT/US2023/066986
Other languages
French (fr)
Other versions
WO2023225471A2 (en
Inventor
Benjamin Harris WEINBERG
Jacob Rosenblum RUBENS
Robert James Citorik
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.)
Flagship Pioneering Innovations VI Inc
Original Assignee
Flagship Pioneering Innovations VI 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 Flagship Pioneering Innovations VI Inc filed Critical Flagship Pioneering Innovations VI Inc
Publication of WO2023225471A2 publication Critical patent/WO2023225471A2/en
Publication of WO2023225471A3 publication Critical patent/WO2023225471A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • 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 [CRISPR]
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

The disclosure provides, e.g., compositions comprising a helitron tranposase or fragment or variant thereof, further comprising a gene modifying polypeptide or a nucleic acid encoding the gene modifying polypeptide, wherein the gene modifying polypeptide comprises: (i) a fragment of a helraiser transposase that lacks an N-terminal domain (ND), e.g., lacks both of an ND and a disordered domain (DD), or a sequence having defined identity thereto, and (ii) a DNA binding domain heterologous to (a)(1); and (b) a donor DNA molecule comprising a nucleic acid molecule comprising (i) one or both of: a right terminal sequence (RTS) and a left terminal sequence (ITS), and (ii) a heterologous object sequence and a heterologous DNA binding domain. Methods for making and using said compositions are also disclosed.
PCT/US2023/066986 2022-05-16 2023-05-15 Helitron compositions and methods Ceased WO2023225471A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263364783P 2022-05-16 2022-05-16
US63/364,783 2022-05-16

Publications (2)

Publication Number Publication Date
WO2023225471A2 WO2023225471A2 (en) 2023-11-23
WO2023225471A3 true WO2023225471A3 (en) 2024-01-11

Family

ID=88836249

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/066986 Ceased WO2023225471A2 (en) 2022-05-16 2023-05-15 Helitron compositions and methods

Country Status (1)

Country Link
WO (1) WO2023225471A2 (en)

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190119658A1 (en) * 2017-10-23 2019-04-25 Poseida Therapeutics, Inc. Modified stem cell memory t cells, methods of making and methods of using same
WO2019126589A1 (en) * 2017-12-20 2019-06-27 Poseida Therapeutics, Inc. Micelles for complexation and delivery of proteins and nucleic acids

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190119658A1 (en) * 2017-10-23 2019-04-25 Poseida Therapeutics, Inc. Modified stem cell memory t cells, methods of making and methods of using same
WO2019126589A1 (en) * 2017-12-20 2019-06-27 Poseida Therapeutics, Inc. Micelles for complexation and delivery of proteins and nucleic acids

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
GRABUNDZIJA IVANA, HICKMAN ALISON B., DYDA FRED: "Helraiser intermediates provide insight into the mechanism of eukaryotic replicative transposition", NATURE COMMUNICATIONS, NATURE PUBLISHING GROUP, UK, vol. 9, no. 1, UK, pages 1 - 14, XP093129666, ISSN: 2041-1723, DOI: 10.1038/s41467-018-03688-w *

Also Published As

Publication number Publication date
WO2023225471A2 (en) 2023-11-23

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