WO2023196220A3 - Method for genome-wide functional perturbation of human microsatellites using engineered zinc fingers - Google Patents

Method for genome-wide functional perturbation of human microsatellites using engineered zinc fingers Download PDF

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Publication number
WO2023196220A3
WO2023196220A3 PCT/US2023/017257 US2023017257W WO2023196220A3 WO 2023196220 A3 WO2023196220 A3 WO 2023196220A3 US 2023017257 W US2023017257 W US 2023017257W WO 2023196220 A3 WO2023196220 A3 WO 2023196220A3
Authority
WO
WIPO (PCT)
Prior art keywords
microsatellites
genome
human
zinc fingers
engineered zinc
Prior art date
Application number
PCT/US2023/017257
Other languages
French (fr)
Other versions
WO2023196220A2 (en
WO2023196220A9 (en
Inventor
J. Keith Joung
Y. Esther TAK
Miguel N. RIVERA
Gaylor Boulay
Original Assignee
The General Hospital Corporation
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 The General Hospital Corporation filed Critical The General Hospital Corporation
Publication of WO2023196220A2 publication Critical patent/WO2023196220A2/en
Publication of WO2023196220A3 publication Critical patent/WO2023196220A3/en
Publication of WO2023196220A9 publication Critical patent/WO2023196220A9/en

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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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 RNAses, DNAses
    • 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
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding

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

Abstract

Described herein are compositions comprising zinc fingers and methods of use thereof for the treatment of nucleotide repeat expansion disorders such as Ewing Sarcoma.
PCT/US2023/017257 2022-04-04 2023-04-03 Method for genome-wide functional perturbation of human microsatellites using engineered zinc fingers WO2023196220A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263327175P 2022-04-04 2022-04-04
US63/327,175 2022-04-04

Publications (3)

Publication Number Publication Date
WO2023196220A2 WO2023196220A2 (en) 2023-10-12
WO2023196220A3 true WO2023196220A3 (en) 2023-11-23
WO2023196220A9 WO2023196220A9 (en) 2024-02-01

Family

ID=88243374

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/017257 WO2023196220A2 (en) 2022-04-04 2023-04-03 Method for genome-wide functional perturbation of human microsatellites using engineered zinc fingers

Country Status (1)

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WO (1) WO2023196220A2 (en)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018145068A1 (en) * 2017-02-06 2018-08-09 Trustees Of Boston University An integrated system for programmable dna methylation
WO2019149946A1 (en) * 2018-02-02 2019-08-08 Danmarks Tekniske Universitet Therapeutics for autoimmune kidney disease: synthetic antigens
US20210102213A1 (en) * 2018-05-17 2021-04-08 The General Hospital Corporation CCCTC-Binding Factor Variants

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018145068A1 (en) * 2017-02-06 2018-08-09 Trustees Of Boston University An integrated system for programmable dna methylation
WO2019149946A1 (en) * 2018-02-02 2019-08-08 Danmarks Tekniske Universitet Therapeutics for autoimmune kidney disease: synthetic antigens
US20210102213A1 (en) * 2018-05-17 2021-04-08 The General Hospital Corporation CCCTC-Binding Factor Variants

Also Published As

Publication number Publication date
WO2023196220A2 (en) 2023-10-12
WO2023196220A9 (en) 2024-02-01

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