CO2023018656A2 - Sistemas de edición de genes que comprenden un arn guía dirigido a hidroxiácido oxidasa 1 (hao1) y usos de estos - Google Patents

Sistemas de edición de genes que comprenden un arn guía dirigido a hidroxiácido oxidasa 1 (hao1) y usos de estos

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Publication number
CO2023018656A2
CO2023018656A2 CONC2023/0018656A CO2023018656A CO2023018656A2 CO 2023018656 A2 CO2023018656 A2 CO 2023018656A2 CO 2023018656 A CO2023018656 A CO 2023018656A CO 2023018656 A2 CO2023018656 A2 CO 2023018656A2
Authority
CO
Colombia
Prior art keywords
hao1
gene editing
guide rna
hydroxy acid
acid oxidase
Prior art date
Application number
CONC2023/0018656A
Other languages
English (en)
Inventor
Quinton Norman Wessells
Jeffrey Raymond Haswell
Tia Marie Ditommaso
Noah Michael Jakimo
Sejuti Sengupta
Original Assignee
Arbor Biotechnologies Inc
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 Arbor Biotechnologies Inc filed Critical Arbor Biotechnologies Inc
Publication of CO2023018656A2 publication Critical patent/CO2023018656A2/es

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    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • A—HUMAN NECESSITIES
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    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
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    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C12Y101/03—Oxidoreductases acting on the CH-OH group of donors (1.1) with a oxygen as acceptor (1.1.3)
    • C12Y101/03015—(S)-2-Hydroxy-acid oxidase (1.1.3.15)

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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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  • Enzymes And Modification Thereof (AREA)
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Abstract

En la presente se proporcionan sistemas de edición de genes y/o composiciones que comprenden ARN guías que se dirigen a HAO1 para usar en edición genética del gen HAO1. También se proporcionan en la presente métodos para utilizar el sistema de edición de genes para introducir ediciones al gen HAO1 y/o para el tratamiento de hiperoxaluria primaria (PH), y procesos para caracterizar el sistema de edición de genes.
CONC2023/0018656A 2021-06-04 2023-12-29 Sistemas de edición de genes que comprenden un arn guía dirigido a hidroxiácido oxidasa 1 (hao1) y usos de estos CO2023018656A2 (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163197073P 2021-06-04 2021-06-04
US202163225046P 2021-07-23 2021-07-23
US202163292889P 2021-12-22 2021-12-22
US202263300727P 2022-01-19 2022-01-19
PCT/US2022/032144 WO2022256642A2 (en) 2021-06-04 2022-06-03 Gene editing systems comprising an rna guide targeting hydroxyacid oxidase 1 (hao1) and uses thereof

Publications (1)

Publication Number Publication Date
CO2023018656A2 true CO2023018656A2 (es) 2024-01-25

Family

ID=82694238

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2023/0018656A CO2023018656A2 (es) 2021-06-04 2023-12-29 Sistemas de edición de genes que comprenden un arn guía dirigido a hidroxiácido oxidasa 1 (hao1) y usos de estos

Country Status (10)

Country Link
US (2) US20230032141A1 (es)
EP (1) EP4347833A2 (es)
JP (1) JP2024520691A (es)
KR (1) KR20240034746A (es)
AU (1) AU2022283922A1 (es)
CA (1) CA3222159A1 (es)
CO (1) CO2023018656A2 (es)
IL (1) IL308803A (es)
TW (1) TW202317763A (es)
WO (1) WO2022256642A2 (es)

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3087184B1 (en) * 2013-12-27 2019-07-03 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of glycolate oxidase (hao1) by double-stranded rna
DE202019005567U1 (de) * 2018-03-14 2021-02-16 Arbor Biotechnologies, Inc. Neue CRISPR-DNA-Targeting-Enzyme und -Systeme
WO2020028327A1 (en) * 2018-07-31 2020-02-06 Intellia Therapeutics, Inc. Compositions and methods for hydroxyacid oxidase 1 ( hao1) gene editing for treating primary hyperoxaluria type 1 (ph1)
US20230332119A1 (en) * 2020-03-31 2023-10-19 Arbor Biotechnologies, Inc. Compositions comprising a cas12i2 variant polypeptide and uses thereof
US20230235305A1 (en) * 2020-06-16 2023-07-27 Arbor Biotechnologies, Inc. Cells modified by a cas12i polypeptide
CN113308451B (zh) * 2020-12-07 2023-07-25 中国科学院动物研究所 工程化的Cas效应蛋白及其使用方法

Also Published As

Publication number Publication date
EP4347833A2 (en) 2024-04-10
US20230032141A1 (en) 2023-02-02
IL308803A (en) 2024-01-01
CA3222159A1 (en) 2022-12-08
US20230212541A1 (en) 2023-07-06
WO2022256642A3 (en) 2023-01-12
AU2022283922A1 (en) 2023-12-07
KR20240034746A (ko) 2024-03-14
TW202317763A (zh) 2023-05-01
AU2022283922A9 (en) 2023-12-14
US11821012B2 (en) 2023-11-21
WO2022256642A2 (en) 2022-12-08
JP2024520691A (ja) 2024-05-24

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