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

Info

Publication number
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
Authority
JP
Japan
Prior art keywords
tale
seq
sequence
nuclease
protein
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
JP2024530470A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024540639A5 (https=
Inventor
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
Original Assignee
セレクティス ソシエテ アノニム
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 セレクティス ソシエテ アノニム filed Critical セレクティス ソシエテ アノニム
Publication of JP2024540639A publication Critical patent/JP2024540639A/ja
Publication of JP2024540639A5 publication Critical patent/JP2024540639A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034Isolating an individual clone by screening libraries
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal

Landscapes

  • 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)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Crystallography & Structural Chemistry (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 (https=) 2025-12-01

Family

ID=84487474

Family Applications (1)

Application Number Title Priority Date Filing Date
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 (https=)
EP (1) EP4437091A1 (https=)
JP (1) JP2024540639A (https=)
KR (1) KR20240110844A (https=)
AU (1) AU2022395500A1 (https=)
CA (1) CA3238700A1 (https=)
IL (1) IL312721A (https=)
MX (1) MX2024006051A (https=)
WO (1) WO2023094435A1 (https=)

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
WO2025211767A1 (ko) * 2024-04-02 2025-10-09 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
WO2011072246A2 (en) 2009-12-10 2011-06-16 Regents Of The University Of Minnesota Tal effector-mediated dna modification
CA3111953C (en) 2011-04-05 2023-10-24 Cellectis Method for the generation of compact tale-nucleases 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
BR112014029417B1 (pt) 2012-05-25 2023-03-07 Cellectis Método ex vivo para a preparação de células t para imunoterapia
US10378007B2 (en) * 2012-09-03 2019-08-13 Cellectis Methods for modulating TAL specificity
ES2716867T3 (es) 2013-05-31 2019-06-17 Cellectis Sa Endonucleasa de asentamiento LAGLIDADG que escinde el gen de receptor de células T alfa y usos de la misma

Also Published As

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

Similar Documents

Publication Publication Date Title
US11834686B2 (en) Engineered target specific base editors
ES2901989T3 (es) Nucleasas específicas de objetivo modificadas
JP2024540639A (ja) 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
CA2913871C (en) A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof
EP4025691B1 (en) Novel, non-naturally occurring crispr-cas nucleases for genome editing
Humbert et al. Targeted gene therapies: tools, applications, optimization
JP2022051772A (ja) Dna結合ドメインと切断ドメインとを連結するための組成物
CN101273141A (zh) 外源核酸序列的靶向整合和表达
CN118382695A (zh) 具有改进的靶上/靶外活性比的新的tale蛋白支架
WO2026046724A1 (en) Tale protein scaffolds involving fusions of monopartite and bipartite nls
US20160138047A1 (en) Improved polynucleotide sequences encoding tale repeats
HK40111655A (zh) 新型omni 115、124、127、144-149、159、218、237、248、251-253和259 crispr核酸酶
HK40078419A (en) Novel, non-naturally occurring crispr-cas nucleases for genome editing
HK40078419B (en) Novel, non-naturally occurring crispr-cas nucleases for genome editing
HK40011095B (en) Engineered target specific nucleases
NZ791740A (en) Engineered target specific nucleases
HK1223373B (en) A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof
NZ791711A (en) Engineered target specific nucleases
NZ791732A (en) Engineered target specific nucleases

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20251120

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20251120