CO2022014598A2 - Composiciones y métodos para el direccionamiento de c9orf72 - Google Patents
Composiciones y métodos para el direccionamiento de c9orf72Info
- Publication number
- CO2022014598A2 CO2022014598A2 CONC2022/0014598A CO2022014598A CO2022014598A2 CO 2022014598 A2 CO2022014598 A2 CO 2022014598A2 CO 2022014598 A CO2022014598 A CO 2022014598A CO 2022014598 A2 CO2022014598 A2 CO 2022014598A2
- Authority
- CO
- Colombia
- Prior art keywords
- methods
- c9orf72
- addressing
- compositions
- systems
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title 1
- 101150014718 C9orf72 gene Proteins 0.000 abstract 2
- 210000004027 cell Anatomy 0.000 abstract 2
- 230000035772 mutation Effects 0.000 abstract 2
- 108020004707 nucleic acids Proteins 0.000 abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 2
- 102000039446 nucleic acids Human genes 0.000 abstract 2
- 101710163270 Nuclease Proteins 0.000 abstract 1
- 210000003527 eukaryotic cell Anatomy 0.000 abstract 1
Classifications
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- 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
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- 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
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- 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
- 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
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- 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 RNAses, DNAses
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
- A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
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- 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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- 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 [CRISPRs]
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Cell Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Mycology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Virology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
Abstract
En la presente se proporcionan sistemas de Clase 2, Tipo V que comprenden nucleasas, ácidos nucleicos guía (gNA) y, opcionalmente, ácidos nucleicos de plantilla de donante útiles en la modificación de un gen C9orf72. Los sistemas también son útiles para la introducción en células, por ejemplo, células eucariotas que tienen mutaciones o duplicaciones en el gen C9orf72. También se proporcionan métodos para usar tales sistemas para modificar células que tienen tales mutaciones o duplicaciones.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202062991403P | 2020-03-18 | 2020-03-18 | |
PCT/US2021/022840 WO2021188729A1 (en) | 2020-03-18 | 2021-03-17 | Compositions and methods for the targeting of c9orf72 |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2022014598A2 true CO2022014598A2 (es) | 2022-10-31 |
Family
ID=75478206
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2022/0014598A CO2022014598A2 (es) | 2020-03-18 | 2022-10-14 | Composiciones y métodos para el direccionamiento de c9orf72 |
Country Status (11)
Country | Link |
---|---|
EP (1) | EP4121535A1 (es) |
JP (1) | JP2023518541A (es) |
KR (1) | KR20230002401A (es) |
CN (1) | CN116096885A (es) |
AU (1) | AU2021237633A1 (es) |
BR (1) | BR112022018673A2 (es) |
CA (1) | CA3172178A1 (es) |
CO (1) | CO2022014598A2 (es) |
IL (1) | IL296477A (es) |
MX (1) | MX2022011460A (es) |
WO (1) | WO2021188729A1 (es) |
Families Citing this family (10)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN114375334A (zh) | 2019-06-07 | 2022-04-19 | 斯克里贝治疗公司 | 工程化CasX系统 |
MX2023006566A (es) * | 2020-12-03 | 2023-08-07 | Scribe Therapeutics Inc | Sistemas crispr tipo v clase 2 diseñados por ingeniería. |
WO2022261150A2 (en) | 2021-06-09 | 2022-12-15 | Scribe Therapeutics Inc. | Particle delivery systems |
WO2023077095A2 (en) * | 2021-10-29 | 2023-05-04 | Mammoth Biosciences, Inc. | Effector proteins, compositions, systems, devices, kits and methods of use thereof |
WO2023086389A1 (en) * | 2021-11-09 | 2023-05-19 | Prime Medicine, Inc. | Genome editing compositions and methods for treatment of amyotrophic lateral sclerosis |
WO2023235725A2 (en) * | 2022-05-31 | 2023-12-07 | Regeneron Pharmaceuticals, Inc. | Crispr-based therapeutics for c9orf72 repeat expansion disease |
WO2023240074A1 (en) | 2022-06-07 | 2023-12-14 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of pcsk9 |
TW202405175A (zh) | 2022-06-07 | 2024-02-01 | 美商斯奎柏治療公司 | 用於靶向pcsk9的組合物及方法 |
WO2023240157A2 (en) * | 2022-06-08 | 2023-12-14 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of dmd |
WO2023240162A1 (en) * | 2022-06-08 | 2023-12-14 | Scribe Therapeutics Inc. | Aav vectors for gene editing |
Family Cites Families (14)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5143854A (en) | 1989-06-07 | 1992-09-01 | Affymax Technologies N.V. | Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof |
US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
US5412087A (en) | 1992-04-24 | 1995-05-02 | Affymax Technologies N.V. | Spatially-addressable immobilization of oligonucleotides and other biological polymers on surfaces |
US5695937A (en) | 1995-09-12 | 1997-12-09 | The Johns Hopkins University School Of Medicine | Method for serial analysis of gene expression |
WO2010075303A1 (en) | 2008-12-23 | 2010-07-01 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Splicing factors with a puf protein rna-binding domain and a splicing effector domain and uses of same |
US9580714B2 (en) | 2010-11-24 | 2017-02-28 | The University Of Western Australia | Peptides for the specific binding of RNA targets |
WO2017083722A1 (en) | 2015-11-11 | 2017-05-18 | Greenberg Kenneth P | Crispr compositions and methods of using the same for gene therapy |
SG11201805320XA (en) * | 2015-12-23 | 2018-07-30 | Crispr Therapeutics Ag | Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration |
AU2017335890B2 (en) * | 2016-09-30 | 2024-05-09 | The Regents Of The University Of California | RNA-guided nucleic acid modifying enzymes and methods of use thereof |
WO2018195555A1 (en) | 2017-04-21 | 2018-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Crispr/cas 9-mediated integration of polynucleotides by sequential homologous recombination of aav donor vectors |
WO2018208972A1 (en) * | 2017-05-09 | 2018-11-15 | University Of Massachusetts | Methods of treating amyotrophic lateral sclerosis (als) |
EP3950957A1 (en) * | 2017-08-08 | 2022-02-09 | Depixus | In vitro isolation and enrichment of nucleic acids using site-specific nucleases |
US11578334B2 (en) * | 2017-10-25 | 2023-02-14 | Monsanto Technology Llc | Targeted endonuclease activity of the RNA-guided endonuclease CasX in eukaryotes |
CN114375334A (zh) | 2019-06-07 | 2022-04-19 | 斯克里贝治疗公司 | 工程化CasX系统 |
-
2021
- 2021-03-17 KR KR1020227035510A patent/KR20230002401A/ko unknown
- 2021-03-17 BR BR112022018673A patent/BR112022018673A2/pt unknown
- 2021-03-17 JP JP2022556229A patent/JP2023518541A/ja active Pending
- 2021-03-17 WO PCT/US2021/022840 patent/WO2021188729A1/en unknown
- 2021-03-17 CA CA3172178A patent/CA3172178A1/en active Pending
- 2021-03-17 IL IL296477A patent/IL296477A/en unknown
- 2021-03-17 AU AU2021237633A patent/AU2021237633A1/en active Pending
- 2021-03-17 CN CN202180034169.7A patent/CN116096885A/zh active Pending
- 2021-03-17 MX MX2022011460A patent/MX2022011460A/es unknown
- 2021-03-17 EP EP21718316.9A patent/EP4121535A1/en active Pending
-
2022
- 2022-10-14 CO CONC2022/0014598A patent/CO2022014598A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
CN116096885A (zh) | 2023-05-09 |
JP2023518541A (ja) | 2023-05-02 |
WO2021188729A1 (en) | 2021-09-23 |
EP4121535A1 (en) | 2023-01-25 |
AU2021237633A1 (en) | 2022-10-06 |
KR20230002401A (ko) | 2023-01-05 |
BR112022018673A2 (pt) | 2022-12-27 |
MX2022011460A (es) | 2022-12-15 |
IL296477A (en) | 2022-11-01 |
CA3172178A1 (en) | 2021-09-23 |
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