JP2024539458A5 - - Google Patents

Info

Publication number
JP2024539458A5
JP2024539458A5 JP2024529784A JP2024529784A JP2024539458A5 JP 2024539458 A5 JP2024539458 A5 JP 2024539458A5 JP 2024529784 A JP2024529784 A JP 2024529784A JP 2024529784 A JP2024529784 A JP 2024529784A JP 2024539458 A5 JP2024539458 A5 JP 2024539458A5
Authority
JP
Japan
Prior art keywords
mouse
sequence
stmn2
human
exon
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2024529784A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024539458A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/080224 external-priority patent/WO2023092118A1/en
Publication of JP2024539458A publication Critical patent/JP2024539458A/ja
Publication of JP2024539458A5 publication Critical patent/JP2024539458A5/ja
Pending legal-status Critical Current

Links

JP2024529784A 2021-11-22 2022-11-21 破壊されたtdp-43結合部位を有するヒト化スタスミン2マウスモデルに関連する方法および組成物 Pending JP2024539458A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163282023P 2021-11-22 2021-11-22
US63/282,023 2021-11-22
PCT/US2022/080224 WO2023092118A1 (en) 2021-11-22 2022-11-21 Methods and compositions relating to humanized stathmin2 mouse model with disrupted tdp-43 binding sites

Publications (2)

Publication Number Publication Date
JP2024539458A JP2024539458A (ja) 2024-10-28
JP2024539458A5 true JP2024539458A5 (https=) 2025-11-13

Family

ID=86397888

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024529784A Pending JP2024539458A (ja) 2021-11-22 2022-11-21 破壊されたtdp-43結合部位を有するヒト化スタスミン2マウスモデルに関連する方法および組成物

Country Status (7)

Country Link
US (1) US20250040523A1 (https=)
EP (1) EP4436586A4 (https=)
JP (1) JP2024539458A (https=)
KR (1) KR20240116486A (https=)
CN (1) CN118284707A (https=)
IL (1) IL312770A (https=)
WO (1) WO2023092118A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN120519523A (zh) * 2025-05-21 2025-08-22 首都医科大学附属北京天坛医院 一种基于Sptlc-E260K点突变构建的ALS小鼠模型及其构建方法和用途

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120192298A1 (en) * 2009-07-24 2012-07-26 Sigma Aldrich Co. Llc Method for genome editing
AU2020466994A1 (en) * 2019-09-17 2022-04-21 Rutgers, The State University Of New Jersey Highly efficient DNA base editors mediated by RNA-aptamer recruitment for targeted genome modification and uses thereof

Similar Documents

Publication Publication Date Title
Loganathan et al. Non-coding RNAs in human health and disease: potential function as biomarkers and therapeutic targets
Rakers et al. Stroke target identification guided by astrocyte transcriptome analysis
Duan et al. Brain-wide Cas9-mediated cleavage of a gene causing familial Alzheimer’s disease alleviates amyloid-related pathologies in mice
CN114634930B (zh) 使用rna指导型内切核酸酶改善基因组工程特异性的组合物和方法
Jia et al. The effects of pharmacological inhibition of histone deacetylase 3 (HDAC3) in Huntington’s disease mice
US20250161485A1 (en) Aav capsid variants and uses thereof
US20260085095A1 (en) Aav capsid variants and uses thereof
JP2019500349A5 (https=)
JP2009537129A (ja) エキソンスキッピングを誘発する為の手段及び方法
EP3212788A2 (en) Compositions, methods and use of synthetic lethal screening
JP2008538500A (ja) SRタンパク質の結合に対する干渉とRNA二次構造に対する干渉による、mRNA前駆体におけるエクソン認識の調節
KR20210010549A (ko) 스플라이싱이상을 감소시키고 rna 우성 질환을 치료하기 위한 조성물 및 방법
WO2024006741A1 (en) Aav capsid variants and uses thereof
JP2024539458A5 (https=)
US20250388626A1 (en) Aav capsid variants and uses thereof
WO2025038430A1 (en) Aav capsid variants and uses thereof
Alizadeh et al. Production of duchenne muscular dystrophy cellular model using crispr-cas9 exon deletion strategy
Flynn et al. Synthetic dosage-compensating miRNA circuits allow precision gene therapy for Rett syndrome
You et al. Genome-wide profiling reveals functional diversification of∆ FosB gene targets in the hippocampus of an Alzheimer's disease mouse model
US20240108755A1 (en) Compositions and methods for treating a neurodegenerative or developmental disorder
Inoue et al. Amplification of 4 ‘-ThioDNA in the presence of 4 ‘-Thio-dTTP and 4 ‘-Thio-dCTP, and 4 ‘-ThioDNA-directed transcription in vitro and in mammalian cells
Lee et al. DNA regulatory element cooperation and competition in transcription
WO2025147436A1 (en) Aav capsid variants and uses thereof
JP2024539458A (ja) 破壊されたtdp-43結合部位を有するヒト化スタスミン2マウスモデルに関連する方法および組成物
US20220106598A1 (en) Homologous recombination directed genome editing in eukaryotes