GB2559922A - Nucleobase editors and uses thereof - Google Patents

Nucleobase editors and uses thereof Download PDF

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Publication number
GB2559922A
GB2559922A GB1808388.1A GB201808388A GB2559922A GB 2559922 A GB2559922 A GB 2559922A GB 201808388 A GB201808388 A GB 201808388A GB 2559922 A GB2559922 A GB 2559922A
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United Kingdom
Prior art keywords
nucleic acid
editing
proteins
targeted
domains
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.)
Withdrawn
Application number
GB1808388.1A
Other versions
GB201808388D0 (en
Inventor
R Liu David
Christine Komor Alexis
A Rees Holly
Kim Yongjoo
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.)
Harvard College
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Harvard College
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Filing date
Publication date
Application filed by Harvard College filed Critical Harvard College
Priority claimed from PCT/US2016/058344 external-priority patent/WO2017070632A2/en
Publication of GB201808388D0 publication Critical patent/GB201808388D0/en
Publication of GB2559922A publication Critical patent/GB2559922A/en
Withdrawn legal-status Critical Current

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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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/85—Fusion polypeptide containing an RNA binding domain
    • 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
    • C12N2795/00—Bacteriophages
    • C12N2795/00011—Details
    • C12N2795/10011—Details dsDNA Bacteriophages
    • C12N2795/10111—Myoviridae
    • C12N2795/10122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

Some aspects of this disclosure provide strategies, systems, reagents, methods, and kits that are useful for the targeted editing of nucleic acids, including editing a single site within the genome of a cell or subject, e.g., within the human genome. In some embodiments, fusion proteins of Cas9 and nucleic acid editing proteins or protein domains, e.g., deaminase domains, are provided. In some embodiments, methods for targeted nucleic acid editing are provided. In some embodiments, reagents and kits for the generation of targeted nucleic acid editing proteins, e.g., fusion proteins of Cas9 and nucleic acid editing proteins or domains, are provided.

Description

71) Applicant(s):
President and Fellows of Harvard College 17 Quincy Street, Cambridge 02138, Massachusetts, United States of America (72) Inventor(s):
David R Liu
Alexis Christine Komor Holly A Rees Yongjoo Kim (74) Agent and/or Address for Service:
Kilburn & Strode LLP
Lacon London, 84 Theobalds Road, London, Greater London, WC1X 8NL, United Kingdom (54) Title of the Invention: Nucleobase editors and uses thereof Abstract Title: Nucleobase editors and uses thereof

Claims (1)

  1. Some aspects of this disclosure provide strategies, systems, reagents, methods, and kits that are useful for the targeted editing of nucleic acids, including editing a single site within the genome of a cell or subject, e.g., within the human genome. In some embodiments, fusion proteins of Cas9 and nucleic acid editing proteins or protein domains, e.g., deaminase domains, are provided. In some embodiments, methods for targeted nucleic acid editing are provided. In some embodiments, reagents and kits for the generation of targeted nucleic acid editing proteins, e.g., fusion proteins of Cas9 and nucleic acid editing proteins or domains, are provided.
GB1808388.1A 2015-10-23 2016-10-22 Nucleobase editors and uses thereof Withdrawn GB2559922A (en)

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
US201562245828P 2015-10-23 2015-10-23
US201662249346P 2016-01-15 2016-01-15
US201662311763P 2016-03-22 2016-03-22
US201662322178P 2016-04-13 2016-04-13
US201662357352P 2016-06-30 2016-06-30
US201662357332P 2016-06-30 2016-06-30
US201662370700P 2016-08-03 2016-08-03
US201662398490P 2016-09-22 2016-09-22
US201662408686P 2016-10-14 2016-10-14
PCT/US2016/058344 WO2017070632A2 (en) 2015-10-23 2016-10-22 Nucleobase editors and uses thereof

Publications (2)

Publication Number Publication Date
GB201808388D0 GB201808388D0 (en) 2018-07-11
GB2559922A true GB2559922A (en) 2018-08-22

Family

ID=62812418

Family Applications (1)

Application Number Title Priority Date Filing Date
GB1808388.1A Withdrawn GB2559922A (en) 2015-10-23 2016-10-22 Nucleobase editors and uses thereof

Country Status (1)

Country Link
GB (1) GB2559922A (en)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015089406A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Cas variants for gene editing
WO2015133554A1 (en) * 2014-03-05 2015-09-11 国立大学法人神戸大学 Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same
WO2016183438A1 (en) * 2015-05-14 2016-11-17 Massachusetts Institute Of Technology Self-targeting genome editing system

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015089406A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Cas variants for gene editing
WO2015133554A1 (en) * 2014-03-05 2015-09-11 国立大学法人神戸大学 Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same
WO2016183438A1 (en) * 2015-05-14 2016-11-17 Massachusetts Institute Of Technology Self-targeting genome editing system

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Nature, vol. 522 issue 7603, 2016, Komor et al, "Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage" pp. 420-424 *

Also Published As

Publication number Publication date
GB201808388D0 (en) 2018-07-11

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