CA3196599A1 - Zinc finger fusion proteins for nucleobase editing - Google Patents

Zinc finger fusion proteins for nucleobase editing

Info

Publication number
CA3196599A1
CA3196599A1 CA3196599A CA3196599A CA3196599A1 CA 3196599 A1 CA3196599 A1 CA 3196599A1 CA 3196599 A CA3196599 A CA 3196599A CA 3196599 A CA3196599 A CA 3196599A CA 3196599 A1 CA3196599 A1 CA 3196599A1
Authority
CA
Canada
Prior art keywords
seq
nos
domain
zfp
cell
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
CA3196599A
Other languages
English (en)
French (fr)
Inventor
Friedrich A. FAUSER
Jeffrey C. Miller
Sebastian ARANGUNDY
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.)
Sangamo Therapeutics Inc
Original Assignee
Sangamo Therapeutics 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 Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Publication of CA3196599A1 publication Critical patent/CA3196599A1/en
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04005—Cytidine deaminase (3.5.4.5)
    • 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
    • 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
    • C07K2319/80—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
    • 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]

Landscapes

  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3196599A 2020-09-25 2021-09-24 Zinc finger fusion proteins for nucleobase editing Pending CA3196599A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063083662P 2020-09-25 2020-09-25
US63/083,662 2020-09-25
US202163164893P 2021-03-23 2021-03-23
US63/164,893 2021-03-23
US202163230580P 2021-08-06 2021-08-06
US63/230,580 2021-08-06
PCT/US2021/052088 WO2022067122A1 (en) 2020-09-25 2021-09-24 Zinc finger fusion proteins for nucleobase editing

Publications (1)

Publication Number Publication Date
CA3196599A1 true CA3196599A1 (en) 2022-03-31

Family

ID=78500694

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3196599A Pending CA3196599A1 (en) 2020-09-25 2021-09-24 Zinc finger fusion proteins for nucleobase editing

Country Status (9)

Country Link
US (1) US20240043829A1 (https=)
EP (1) EP4217479A1 (https=)
JP (1) JP2023542705A (https=)
KR (1) KR20230074519A (https=)
CN (1) CN116261594A (https=)
AU (1) AU2021350099A1 (https=)
CA (1) CA3196599A1 (https=)
IL (1) IL301393A (https=)
WO (1) WO2022067122A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022072393A1 (en) * 2020-09-29 2022-04-07 University Of Washington Use of a double-stranded dna cytosine deaminase for mapping dna-protein interactions
JP2026501648A (ja) * 2022-12-30 2026-01-16 北京大学 塩基エディターシステムおよびその使用方法
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
DE69534629D1 (de) 1994-01-18 2005-12-29 Scripps Research Inst Derivate von zinkfingerproteinen und methoden
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
USRE39229E1 (en) 1994-08-20 2006-08-08 Gendaq Limited Binding proteins for recognition of DNA
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
AU2001263155A1 (en) 2000-05-16 2001-11-26 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
JP2005500061A (ja) 2001-08-20 2005-01-06 ザ スクリップス リサーチ インスティテュート Ｃｎｎについての亜鉛フィンガー結合ドメイン
CA2607104A1 (en) * 2005-05-05 2006-11-16 The Arizona Board Of Regents On Behalf Of The University Of Arizona Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
ES2582091T3 (es) 2005-10-18 2016-09-09 Precision Biosciences Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas
US9394531B2 (en) 2008-05-28 2016-07-19 Sangamo Biosciences, Inc. Compositions for linking DNA-binding domains and cleavage domains
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
ES2751916T3 (es) 2010-02-08 2020-04-02 Sangamo Therapeutics Inc Semidominios de escisión genomanipulados
HRP20200254T1 (hr) 2010-05-03 2020-05-29 Sangamo Therapeutics, Inc. Pripravci za povezivanje modula cink fingera
CN106459894B (zh) 2014-03-18 2020-02-18 桑格摩生物科学股份有限公司 用于调控锌指蛋白表达的方法和组合物
KR102455249B1 (ko) 2016-08-24 2022-10-17 상가모 테라퓨틱스, 인코포레이티드 가공된 표적 특이적 뉴클레아제
EP3630849A4 (en) * 2017-05-25 2021-01-13 The General Hospital Corporation BIPARTITE BASIC EDITOR (BBE) AND TYPE II-C-CAS9 ZINC FINGER EDITOR ARCHITECTURES
AU2019326408A1 (en) * 2018-08-23 2021-03-11 Sangamo Therapeutics, Inc. Engineered target specific base editors
US20250011748A1 (en) * 2020-01-28 2025-01-09 The Broad Institute, Inc. Base editors, compositions, and methods for modifying the mitochondrial genome
BR112023005073A2 (pt) * 2020-09-18 2023-10-31 Inst Basic Science Desaminase direcionada e edição de base usando a mesma
JP2024502630A (ja) * 2021-01-12 2024-01-22 マーチ セラピューティクス， インコーポレイテッド コンテキスト依存性二本鎖ｄｎａ特異的デアミナーゼ及びその使用

Also Published As

Publication number Publication date
EP4217479A1 (en) 2023-08-02
JP2023542705A (ja) 2023-10-11
US20240043829A1 (en) 2024-02-08
AU2021350099A9 (en) 2023-07-13
WO2022067122A1 (en) 2022-03-31
IL301393A (en) 2023-05-01
CN116261594A (zh) 2023-06-13
AU2021350099A1 (en) 2023-04-27
KR20230074519A (ko) 2023-05-30

Similar Documents

Publication Publication Date Title
US20230076357A1 (en) Methods and Compositions for Directed Genome Editing
US20240043829A1 (en) Zinc finger fusion proteins for nucleobase editing
JP7672982B2 (ja) 標的配列中の核酸塩基を改変するためのアデノシンデアミナーゼ塩基エディターを有する改変された免疫細胞
CN115279398B (zh) 治疗液态癌症的组合物和方法
US20220411777A1 (en) C-to-G Transversion DNA Base Editors
JP2022546608A (ja) 新規核酸塩基エディター及びその使用方法
US20240287453A1 (en) Persistent allogeneic modified immune cells and methods of use thereof
JP2023510352A (ja) Ｐｃｓｋ９の標的化のための組成物および方法
CN120249248A (zh) 用于无毒调理的组合物和方法
CA3225808A1 (en) Context-specific adenine base editors and uses thereof
US20250179468A1 (en) Direct replacement genome editing
US12576151B2 (en) Fratricide resistant modified immune cells and methods of using the same
WO2020069029A1 (en) Novel crispr nucleases
JP2025533556A (ja) 合成ポリペプチド及びその用途
CN117729931A (zh) 用于治疗转甲状腺素蛋白淀粉样变性的组合物和方法
CN117561074A (zh) 腺苷脱氨酶变体及其用途
US20250361528A1 (en) Novel zinc finger fusion proteins for nucleobase editing
KR20240012377A (ko) 염기 편집기의 자기-불활성화용 조성물 및 방법
CN118234865A (zh) 持久性同种异体修饰免疫细胞及其使用方法
조성익 Genome editing in human mitochondrial DNA.
WO2023235725A2 (en) Crispr-based therapeutics for c9orf72 repeat expansion disease

Legal Events

Date Code Title Description
MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 3RD ANNIV.) - STANDARD

Year of fee payment: 3

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20241011

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20241011

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20251028

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20251106

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20260311