JP2024540639A - オンターゲット/オフターゲット活性比が改善された新規taleタンパク質スキャフォールド - Google Patents

オンターゲット/オフターゲット活性比が改善された新規taleタンパク質スキャフォールド Download PDF

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JP2024540639A
JP2024540639A JP2024530470A JP2024530470A JP2024540639A JP 2024540639 A JP2024540639 A JP 2024540639A JP 2024530470 A JP2024530470 A JP 2024530470A JP 2024530470 A JP2024530470 A JP 2024530470A JP 2024540639 A JP2024540639 A JP 2024540639A
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tale
seq
sequence
nuclease
protein
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JP2024540639A5 (enExample
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フィリップ デュシャトー
アレクサンドル ジュイラット
アレックス ボーイン
セレーナ カザンジュオール
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セレクティス ソシエテ アノニム
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    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • 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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • 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
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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

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  • Health & Medical Sciences (AREA)
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  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
JP2024530470A 2021-11-23 2022-11-23 オンターゲット/オフターゲット活性比が改善された新規taleタンパク質スキャフォールド Pending JP2024540639A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163282453P 2021-11-23 2021-11-23
US63/282,453 2021-11-23
DKPA202270104 2022-03-15
DKPA202270104 2022-03-15
PCT/EP2022/082950 WO2023094435A1 (en) 2021-11-23 2022-11-23 New tale protein scaffolds with improved on-target/off-target activity ratios

Publications (2)

Publication Number Publication Date
JP2024540639A true JP2024540639A (ja) 2024-10-31
JP2024540639A5 JP2024540639A5 (enExample) 2025-12-01

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JP2024530470A Pending JP2024540639A (ja) 2021-11-23 2022-11-23 オンターゲット/オフターゲット活性比が改善された新規taleタンパク質スキャフォールド

Country Status (9)

Country Link
US (1) US20250002945A1 (enExample)
EP (1) EP4437091A1 (enExample)
JP (1) JP2024540639A (enExample)
KR (1) KR20240110844A (enExample)
AU (1) AU2022395500A1 (enExample)
CA (1) CA3238700A1 (enExample)
IL (1) IL312721A (enExample)
MX (1) MX2024006051A (enExample)
WO (1) WO2023094435A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4583890A1 (en) * 2022-09-09 2025-07-16 Iovance Biotherapeutics, Inc. Processes for generating til products using pd-1/tigit talen double knockdown
WO2025181336A1 (en) 2024-03-01 2025-09-04 Cellectis Sa Compositions and methods for hbb-editing in hspc
KR20250148471A (ko) * 2024-04-02 2025-10-14 재단법인 아산사회복지재단 핵산 결합 단백질 및 SsdA를 포함하는 융합단백질 및 이의 용도
WO2026046724A1 (en) 2024-08-30 2026-03-05 Cellectis Sa Tale protein scaffolds involving fusions of monopartite and bipartite nls

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4880635B1 (en) 1984-08-08 1996-07-02 Liposome Company Dehydrated liposomes
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4921757A (en) 1985-04-26 1990-05-01 Massachusetts Institute Of Technology System for delayed and pulsed release of biologically active substances
US4920016A (en) 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
JPH0825869B2 (ja) 1987-02-09 1996-03-13 株式会社ビタミン研究所 抗腫瘍剤包埋リポソ−ム製剤
US4911928A (en) 1987-03-13 1990-03-27 Micro-Pak, Inc. Paucilamellar lipid vesicles
US4917951A (en) 1987-07-28 1990-04-17 Micro-Pak, Inc. Lipid vesicles formed of surfactants and steroids
PT2816112T (pt) 2009-12-10 2018-11-20 Univ Iowa State Res Found Inc Modificação do adn modificada pelo efector tal
US20140115726A1 (en) 2011-04-05 2014-04-24 Cellectis New tale-protein scaffolds and uses thereof
EP2737066B1 (en) 2011-07-29 2017-11-08 Cellectis High throughput method for assembly and cloning polynucleotides comprising highly similar polynucleotidic modules
SG10201806573TA (en) 2012-05-25 2018-09-27 Cellectis Methods for engineering allogeneic and immunosuppressive resistant t cell for immunotherapy
US10378007B2 (en) * 2012-09-03 2019-08-13 Cellectis Methods for modulating TAL specificity
CA2913872C (en) 2013-05-31 2022-01-18 Cellectis A laglidadg homing endonuclease cleaving the t-cell receptor alpha gene and uses thereof

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Publication number Publication date
EP4437091A1 (en) 2024-10-02
WO2023094435A1 (en) 2023-06-01
MX2024006051A (es) 2024-06-26
CA3238700A1 (en) 2022-11-23
IL312721A (en) 2024-07-01
US20250002945A1 (en) 2025-01-02
AU2022395500A1 (en) 2024-05-23
KR20240110844A (ko) 2024-07-16

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