MX2023000035A - Método de tratamiento de edición del gen de la deficiencia de piruvato quinasa (pkd). - Google Patents

Método de tratamiento de edición del gen de la deficiencia de piruvato quinasa (pkd).

Info

Publication number
MX2023000035A
MX2023000035A MX2023000035A MX2023000035A MX2023000035A MX 2023000035 A MX2023000035 A MX 2023000035A MX 2023000035 A MX2023000035 A MX 2023000035A MX 2023000035 A MX2023000035 A MX 2023000035A MX 2023000035 A MX2023000035 A MX 2023000035A
Authority
MX
Mexico
Prior art keywords
pkd
pyruvate kinase
kinase deficiency
treatment method
gene editing
Prior art date
Application number
MX2023000035A
Other languages
English (en)
Spanish (es)
Inventor
Matthew Porteus
Sanz José Carlos Segovia
Bustamante Óscar Quintana
Baquero Sara Fañanas
Original Assignee
Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P
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 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P filed Critical Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P
Publication of MX2023000035A publication Critical patent/MX2023000035A/es

Links

Classifications

    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • C12N9/226—Class 2 CAS enzyme complex, e.g. single CAS protein
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/01—Phosphotransferases with an alcohol group as acceptor (2.7.1)
    • C12Y207/0104—Pyruvate kinase (2.7.1.40)
    • 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]
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MX2023000035A 2020-06-26 2021-06-28 Método de tratamiento de edición del gen de la deficiencia de piruvato quinasa (pkd). MX2023000035A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP20382568.2A EP3929294A1 (en) 2020-06-26 2020-06-26 Pyruvate kinase deficiency (pkd) gene editing treatment method
PCT/EP2021/067719 WO2021260227A1 (en) 2020-06-26 2021-06-28 Pyruvate kinase deficiency (pkd) gene editing treatment method

Publications (1)

Publication Number Publication Date
MX2023000035A true MX2023000035A (es) 2023-04-10

Family

ID=71607853

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023000035A MX2023000035A (es) 2020-06-26 2021-06-28 Método de tratamiento de edición del gen de la deficiencia de piruvato quinasa (pkd).

Country Status (9)

Country Link
US (1) US12655406B2 (enExample)
EP (2) EP3929294A1 (enExample)
JP (1) JP7847778B2 (enExample)
KR (1) KR20230029922A (enExample)
CN (1) CN116648511A (enExample)
AU (1) AU2021298186A1 (enExample)
CA (1) CA3188164A1 (enExample)
MX (1) MX2023000035A (enExample)
WO (1) WO2021260227A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4534654A1 (en) * 2023-10-02 2025-04-09 Consorcio Centro de Investigación Biomédica en Red Pre-stimulation method of cd34+ cells to achieve clinically relevant gene editing frequencies

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3169776B1 (en) * 2014-07-14 2025-09-03 The Regents of The University of California Crispr/cas transcriptional modulation
JP2018533376A (ja) * 2015-11-05 2018-11-15 セントロ デ インベスティガシオン ビオメディカ エン レッド 赤血球系譜を冒す代謝疾患に罹患している被験体から単離された細胞の遺伝子編集の方法、上記方法により得られた細胞、及びそれらの使用
AU2018352583B2 (en) * 2017-10-16 2025-04-17 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency

Also Published As

Publication number Publication date
AU2021298186A1 (en) 2023-02-09
WO2021260227A1 (en) 2021-12-30
US12655406B2 (en) 2026-06-16
JP7847778B2 (ja) 2026-04-20
EP3929294A1 (en) 2021-12-29
EP4172332A1 (en) 2023-05-03
KR20230029922A (ko) 2023-03-03
CA3188164A1 (en) 2021-12-30
CN116648511A (zh) 2023-08-25
US20230303990A1 (en) 2023-09-28
JP2023531718A (ja) 2023-07-25

Similar Documents

Publication Publication Date Title
Song et al. Efficient genome editing using tRNA promoter-driven CRISPR/Cas9 gRNA in Aspergillus niger
Li et al. Suppressing Ku70/Ku80 expression elevates homology-directed repair efficiency in primary fibroblasts
US10301613B2 (en) Targeted remodeling of prokaryotic genomes using CRISPR-nickases
WO2021257997A3 (en) Crispr-associated transposase systems and methods of use thereof
US12325865B2 (en) Methods for increasing efficiency of gene editing in cells
Waaijers et al. Engineering the Caenorhabditis elegans genome with CRISPR/Cas9
AU2021216418A8 (en) Compositions and methods for targeting, editing or modifying human genes
EP4674970A2 (en) Compositions and methods for enhancing homologous recombination
CN105483118A (zh) 以Argonaute核酸酶为核心的基因编辑技术
CO2023016031A2 (es) Composiciones y métodos para el direccionamiento genético in vivo mediado por nucleasas para el tratamiento de trastornos genéticos
MX2022010835A (es) Recombinacion del genoma guiada por arn en escala de kilobase.
CN108384812B (zh) 一种酵母基因组编辑载体及其构建方法和应用
CN104560995B (zh) 一对特异识别猪H11位点的sgRNA及其编码DNA和应用
EP4038192A2 (en) Genetic engineering of fungi to modulate tryptamine expression
Yuan et al. CRISPR-Cas9/Cas12a systems for efficient genome editing and large genomic fragment deletions in Aspergillus niger
Malyarchuk et al. Apex1 can cleave complex clustered DNA lesions in cells
Ublinskaya et al. A PCR-free cloning method for the targeted φ80 Int-mediated integration of any long DNA fragment, bracketed with meganuclease recognition sites, into the Escherichia coli chromosome
Zhang et al. CRISPR/Cas9 genome editing: a promising tool for therapeutic applications of induced pluripotent stem cells
EP3412765B1 (en) Method for producing mutant filamentous fungi
WO2022266538A3 (en) Compositions and methods for targeting, editing or modifying human genes
CN112111528B (zh) 一种内含子异常剪接的修复方法
MX2022008937A (es) Edicion genica para el tratamiento de epidermolisis ampollosa.
Yi et al. CRISPR-based m6A modification and its potential applications in telomerase regulation
WO2023137233A3 (en) Compositions and methods for editing genomes
Choudhary et al. CRISPR-Cas targeting in Haloferax volcanii promotes within-species gene exchange by triggering homologous recombination