CO2024002315A2 - Sistemas de edición de gen que comprenden una guía de arn dirigida a estatmina 2 (stmn2) y usos de los mismos - Google Patents

Sistemas de edición de gen que comprenden una guía de arn dirigida a estatmina 2 (stmn2) y usos de los mismos

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Publication number
CO2024002315A2
CO2024002315A2 CONC2024/0002315A CO2024002315A CO2024002315A2 CO 2024002315 A2 CO2024002315 A2 CO 2024002315A2 CO 2024002315 A CO2024002315 A CO 2024002315A CO 2024002315 A2 CO2024002315 A2 CO 2024002315A2
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Colombia
Prior art keywords
stmn2
stathmin
gene
gene editing
editing systems
Prior art date
Application number
CONC2024/0002315A
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English (en)
Inventor
Quinton Norman Wessells
Tia Marie Ditommaso
Anthony James Garrity
Noah Michael Jakimo
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 CO2024002315A2 publication Critical patent/CO2024002315A2/es

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Abstract

La presente invención se refiere a un sistema para la edición genética de un gen de estatmina 2 (STMN2), que comprende (i) un polipéptido Cas12i2 o un primer ácido nucleico que codifica al polipéptido Cas12i2, y (ii) una guía de ARN o un segundo ácido nucleico que codifica la guía de ARN, en donde la guía de ARN comprende una secuencia espaciadora específica a una secuencia objetivo dentro de un gen STMN2. También se proporcionan en este documento métodos para editar un gen STMN2 usando el sistema de edición de gen descrito en este documento y/o para tratar enfermedades asociadas con el gen STMN2.
CONC2024/0002315A 2021-08-11 2024-02-27 Sistemas de edición de gen que comprenden una guía de arn dirigida a estatmina 2 (stmn2) y usos de los mismos CO2024002315A2 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163231784P 2021-08-11 2021-08-11
US202263322002P 2022-03-21 2022-03-21
PCT/US2022/040042 WO2023018858A1 (en) 2021-08-11 2022-08-11 Gene editing systems comprising an rna guide targeting stathmin 2 (stmn2) and uses thereof

Publications (1)

Publication Number Publication Date
CO2024002315A2 true CO2024002315A2 (es) 2024-03-07

Family

ID=83193363

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2024/0002315A CO2024002315A2 (es) 2021-08-11 2024-02-27 Sistemas de edición de gen que comprenden una guía de arn dirigida a estatmina 2 (stmn2) y usos de los mismos

Country Status (10)

Country Link
US (1) US20230203539A1 (es)
EP (1) EP4384618A1 (es)
JP (1) JP2024529108A (es)
KR (1) KR20240052763A (es)
AU (1) AU2022328401A1 (es)
CA (1) CA3228487A1 (es)
CO (1) CO2024002315A2 (es)
IL (1) IL310616A (es)
MX (1) MX2024001878A (es)
WO (1) WO2023018858A1 (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023205844A1 (en) * 2022-04-26 2023-11-02 Peter Maccallum Cancer Institute Nucleic acids and uses thereof
US20240301447A1 (en) * 2023-02-15 2024-09-12 Arbor Biotechnologies, Inc. Gene editing method for inhibiting aberrant splicing in stathmin 2 (stmn2) transcript

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019178427A1 (en) 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
WO2020150290A2 (en) * 2019-01-14 2020-07-23 President And Fellows Of Harvard College Methods and compositions for restoring stmn2 levels
BR112021024463A2 (pt) * 2019-06-03 2022-03-08 Quralis Corp Oligonucleotídeos e métodos de uso para o tratamento de doenças neurológicas
EP4127154A1 (en) 2020-03-31 2023-02-08 Arbor Biotechnologies, Inc. Compositions comprising a cas12i2 variant polypeptide and uses thereof
CN113151215B (zh) * 2021-05-27 2022-11-18 中国科学院动物研究所 工程化的Cas12i核酸酶及其效应蛋白以及用途

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WO2023018858A1 (en) 2023-02-16
KR20240052763A (ko) 2024-04-23
AU2022328401A1 (en) 2024-02-22
EP4384618A1 (en) 2024-06-19
MX2024001878A (es) 2024-02-29
US20230203539A1 (en) 2023-06-29
IL310616A (en) 2024-04-01
JP2024529108A (ja) 2024-08-01
CA3228487A1 (en) 2023-02-16

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