JP2023519101A5 - - Google Patents

Info

Publication number
JP2023519101A5
JP2023519101A5 JP2022548612A JP2022548612A JP2023519101A5 JP 2023519101 A5 JP2023519101 A5 JP 2023519101A5 JP 2022548612 A JP2022548612 A JP 2022548612A JP 2022548612 A JP2022548612 A JP 2022548612A JP 2023519101 A5 JP2023519101 A5 JP 2023519101A5
Authority
JP
Japan
Prior art keywords
grna
nucleic acid
sequence
exon
cells
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
JP2022548612A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023519101A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/017942 external-priority patent/WO2021163550A1/en
Publication of JP2023519101A publication Critical patent/JP2023519101A/ja
Publication of JP2023519101A5 publication Critical patent/JP2023519101A5/ja
Pending legal-status Critical Current

Links

JP2022548612A 2020-02-12 2021-02-12 Rp1関連網膜変性症のハプロタイプに基づく処置 Pending JP2023519101A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062975636P 2020-02-12 2020-02-12
US62/975,636 2020-02-12
PCT/US2021/017942 WO2021163550A1 (en) 2020-02-12 2021-02-12 Haplotype-based treatment of rp1 associated retinal degenerations

Publications (2)

Publication Number Publication Date
JP2023519101A JP2023519101A (ja) 2023-05-10
JP2023519101A5 true JP2023519101A5 (enExample) 2024-02-21

Family

ID=77292955

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022548612A Pending JP2023519101A (ja) 2020-02-12 2021-02-12 Rp1関連網膜変性症のハプロタイプに基づく処置

Country Status (6)

Country Link
US (2) US12129496B2 (enExample)
EP (1) EP4103238A4 (enExample)
JP (1) JP2023519101A (enExample)
CN (1) CN115427568A (enExample)
AU (1) AU2021219795A1 (enExample)
WO (1) WO2021163550A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12171813B2 (en) 2021-02-05 2024-12-24 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE3023787A1 (de) 1980-06-25 1982-01-21 Studiengesellschaft Kohle mbH, 4330 Mülheim Verfahren zur erhoehung der inkorporation und der expression von genetischem material in die kerne von intakten zellen mit hilfe von liposomen
JPS57124055A (en) 1981-01-27 1982-08-02 Toyota Motor Corp Air-fuel ratio control device for engine
AU6131086A (en) 1985-07-05 1987-01-30 Whitehead Institute For Biomedical Research Epithelial cells expressing foreign genetic material
US4980286A (en) 1985-07-05 1990-12-25 Whitehead Institute For Biomedical Research In vivo introduction and expression of foreign genetic material in epithelial cells
DE3852823T2 (de) 1987-09-11 1995-05-24 Hughes Howard Med Inst Transduktionsveränderte fibroblasten und ihre anwendung.
JP2914692B2 (ja) 1987-12-11 1999-07-05 ホワイトヘツド・インスチチユート・フオー・バイオメデイカル・リサーチ 内皮細胞の遺伝子修飾
EP0400047B1 (en) 1988-02-05 1997-04-23 Whitehead Institute For Biomedical Research Modified hepatocytes and uses therefor
EP0452457B1 (en) 1989-11-03 1997-08-20 Vanderbilt University Method of in vivo delivery of functioning foreign genes
EP0556345B2 (en) 1990-10-31 2005-10-12 Cell Genesys, Inc. Retroviral vectors useful for gene therapy
US10378027B2 (en) 2013-03-15 2019-08-13 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
NZ718926A (en) 2013-10-11 2021-12-24 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
JP6817215B2 (ja) 2015-03-03 2021-01-20 ザ ジェネラル ホスピタル コーポレイション 変更PAM特異性を有する遺伝子操作CRISPR−Cas9ヌクレアーゼ
AU2016316845B2 (en) 2015-08-28 2022-03-10 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
CA3032822A1 (en) * 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
WO2019165322A1 (en) 2018-02-22 2019-08-29 Moore Tara Crispr/cas9 systems, and methods of use thereof
WO2018148246A1 (en) * 2017-02-07 2018-08-16 Massachusetts Institute Of Technology Methods and compositions for rna-guided genetic circuits
ES2992912T3 (en) 2017-04-21 2024-12-19 Massachusetts Gen Hospital Variants of cpf1 (cas12a) with altered pam specificity
US20210017509A1 (en) * 2018-03-23 2021-01-21 The Trustees Of Columbia University In The City Of New York Gene Editing for Autosomal Dominant Diseases
AU2019237541B2 (en) 2018-03-23 2025-02-13 Editas Medicine, Inc. CRISPR/Cas9-mediated exon-skipping approach for USH2A-associated Usher syndrome

Similar Documents

Publication Publication Date Title
US20250235556A1 (en) Gene therapy for autosomal dominant diseases
EP3322297B1 (en) Delivery methods and compositions for nuclease-mediated genome engineering
AU2014364051B2 (en) Methods and compositions for treating hemophilia
RU2650819C2 (ru) Способы и композиции для опосредованной нуклеазой направленной интеграции трансгенов
EP4186921A1 (en) Gene editing for autosomal dominant diseases
JP2020120690A (ja) 遺伝的状態の処置のための方法および組成物
JP7012650B2 (ja) Dna結合ドメインと切断ドメインとを連結するための組成物
WO2018162702A1 (en) Nuclease fusions for enhancing genome editing by homology-directed transgene integration
EP3867375A1 (en) Genome editing methods and constructs
Carvalho et al. Gene therapy for inherited retinal diseases: exploiting new tools in genome editing and nanotechnology
Epstein et al. Combining engineered nucleases with adeno-associated viral vectors for therapeutic gene editing
US20210054372A1 (en) Methods of inactivating gene editing machineries
JP2025175283A (ja) Amdの処置のためのhtra1調節
US20240132881A1 (en) Differential knockout of an allele of a heterozygous bestrophin 1 gene
US20220307057A1 (en) Crispr compositions and methods for promoting gene editing of gata2
HK40075787A (en) Gene therapy for autosomal dominant diseases
US12031149B2 (en) Compositions and methods for promoting gene editing of CXCR4 gene
JP2025506403A5 (enExample)
WO2026032020A1 (zh) Aav介导的rpgr x连锁视网膜变性的基因编辑治疗方法
WO2026036033A1 (en) Products and methods for treatment of autosomal dominant genetic diseases involving diversified disease causing mutations
WO2026088097A1 (en) Mutation-independent genome editing
HK40069063B (zh) 用於治疗地中海贫血或镰状细胞病的方法和组合物
HK1251615B (en) Gene therapy for autosomal dominant diseases
US20230173107A1 (en) Guide rna that targets a mutant human inosine monophosphate deydrogenase i allele