KR20240110844A - 개선된 온-타겟/오프-타겟 활성 비율을 갖는 신규 tale 단백질 스캐폴드 - Google Patents

개선된 온-타겟/오프-타겟 활성 비율을 갖는 신규 tale 단백질 스캐폴드 Download PDF

Info

Publication number
KR20240110844A
KR20240110844A KR1020247020471A KR20247020471A KR20240110844A KR 20240110844 A KR20240110844 A KR 20240110844A KR 1020247020471 A KR1020247020471 A KR 1020247020471A KR 20247020471 A KR20247020471 A KR 20247020471A KR 20240110844 A KR20240110844 A KR 20240110844A
Authority
KR
South Korea
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
KR1020247020471A
Other languages
English (en)
Korean (ko)
Inventor
필립 두샤또
알렉산드르 주일러
알렉스 보인
셀레나 카잔시오글루
Original Assignee
셀렉티스 에스.에이.
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 셀렉티스 에스.에이. filed Critical 셀렉티스 에스.에이.
Publication of KR20240110844A publication Critical patent/KR20240110844A/ko
Pending legal-status Critical Current

Links

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)
KR1020247020471A 2021-11-23 2022-11-23 개선된 온-타겟/오프-타겟 활성 비율을 갖는 신규 tale 단백질 스캐폴드 Pending KR20240110844A (ko)

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 (1)

Publication Number Publication Date
KR20240110844A true KR20240110844A (ko) 2024-07-16

Family

ID=84487474

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247020471A Pending KR20240110844A (ko) 2021-11-23 2022-11-23 개선된 온-타겟/오프-타겟 활성 비율을 갖는 신규 tale 단백질 스캐폴드

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
JP2024540639A (ja) 2024-10-31
CA3238700A1 (en) 2022-11-23
IL312721A (en) 2024-07-01
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
US12378546B2 (en) Coupling endonucleases with end-processing enzymes drives high efficiency gene disruption
KR20240110844A (ko) 개선된 온-타겟/오프-타겟 활성 비율을 갖는 신규 tale 단백질 스캐폴드
US11834686B2 (en) Engineered target specific base editors
ES2901989T3 (es) Nucleasas específicas de objetivo modificadas
US11920169B2 (en) Compositions for linking DNA-binding domains and cleavage domains
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
JP2024509139A (ja) メタゲノム由来の新規のcrispr-casヌクレアーゼ
Rahim et al. Systematic review on the comparison of molecular gene editing tools
JP2024532784A (ja) 新規なomni-115、124、127、144~149、159、218、237、248、251~253及び259crisprヌクレアーゼ
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
Pelletier Genome Editing with Targetable Nucleases
US20160138047A1 (en) Improved polynucleotide sequences encoding tale repeats
Angaji et al. TALEN: a tool for genome editing
HK40111655A (zh) 新型omni 115、124、127、144-149、159、218、237、248、251-253和259 crispr核酸酶
HK40078419B (en) Novel, non-naturally occurring crispr-cas nucleases for genome editing
HK40078419A (en) Novel, non-naturally occurring crispr-cas nucleases for genome editing
Beikzadeh et al. TALEN: A Tool for Genome Editing
He High-Efficiency Knock-In of Large Reporter Genes in Human ESCs via CRISPR/Cas9-Induced Homology-Independent DNA Repair
NZ791740A (en) Engineered target specific nucleases
HK40011095B (en) Engineered target specific nucleases
NZ791711A (en) Engineered target specific nucleases
HK1223373B (en) A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

E13 Pre-grant limitation requested

Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000