CA3228487A1 - Gene editing systems comprising an rna guide targeting stathmin 2 (stmn2) and uses thereof - Google Patents

Gene editing systems comprising an rna guide targeting stathmin 2 (stmn2) and uses thereof Download PDF

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Publication number
CA3228487A1
CA3228487A1 CA3228487A CA3228487A CA3228487A1 CA 3228487 A1 CA3228487 A1 CA 3228487A1 CA 3228487 A CA3228487 A CA 3228487A CA 3228487 A CA3228487 A CA 3228487A CA 3228487 A1 CA3228487 A1 CA 3228487A1
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Canada
Prior art keywords
stmn2
intron
sequence
att
seq
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Pending
Application number
CA3228487A
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English (en)
French (fr)
Inventor
Tia Marie DITOMMASO
Anthony James Garrity
Noah Michael Jakimo
Quinton Norman WESSELLS
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Arbor Biotechnologies Inc
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Arbor Biotechnologies Inc
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Publication of CA3228487A1 publication Critical patent/CA3228487A1/en
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    • 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
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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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    • C12N15/09—Recombinant DNA-technology
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    • 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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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0684—Cells of the urinary tract or kidneys
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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CA3228487A 2021-08-11 2022-08-11 Gene editing systems comprising an rna guide targeting stathmin 2 (stmn2) and uses thereof Pending CA3228487A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163231784P 2021-08-11 2021-08-11
US63/231,784 2021-08-11
US202263322002P 2022-03-21 2022-03-21
US63/322,002 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
CA3228487A1 true CA3228487A1 (en) 2023-02-16

Family

ID=83193363

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3228487A Pending CA3228487A1 (en) 2021-08-11 2022-08-11 Gene editing systems comprising an rna guide targeting stathmin 2 (stmn2) and uses thereof

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US (2) US20250127924A1 (enExample)
EP (1) EP4384618A1 (enExample)
JP (1) JP2024529108A (enExample)
KR (1) KR20240052763A (enExample)
AU (1) AU2022328401A1 (enExample)
CA (1) CA3228487A1 (enExample)
CO (1) CO2024002315A2 (enExample)
IL (1) IL310616A (enExample)
MX (1) MX2024001878A (enExample)
WO (1) WO2023018858A1 (enExample)

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
US20260232844A1 (en) * 2023-02-15 2026-08-13 Arbor Biotechnologies, Inc. Gene editing method for inhibiting aberrant splicing in stathmin 2 (stmn2) transcript

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3765615T3 (pl) 2018-03-14 2023-11-13 Arbor Biotechnologies, Inc. Nowe enzymy i układy crispr ukierunkowane na dna
US12496327B2 (en) * 2019-01-14 2025-12-16 President And Fellows Of Harvard College Methods and compositions for restoring STMN2 levels
WO2020247419A2 (en) * 2019-06-03 2020-12-10 Quralis Corporation Oligonucleotides and methods of use for treating neurological diseases
CN115698278B (zh) 2020-03-31 2025-10-17 阿伯生物技术公司 包含Cas12i2变体多肽的组合物及其用途
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
JP2024522112A (ja) * 2021-05-27 2024-06-11 中国科学院動物研究所 エンジニアリングされたＣａｓ１２ｉヌクレアーゼ、エフェクタータンパク質及びその用途

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Publication number Publication date
US20230203539A1 (en) 2023-06-29
IL310616A (en) 2024-04-01
EP4384618A1 (en) 2024-06-19
JP2024529108A (ja) 2024-08-01
WO2023018858A1 (en) 2023-02-16
AU2022328401A1 (en) 2024-02-22
MX2024001878A (es) 2024-02-29
US20250127924A1 (en) 2025-04-24
KR20240052763A (ko) 2024-04-23
CO2024002315A2 (es) 2024-03-07

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