JP2023519101A5 - - Google Patents

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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
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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)
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JP2023519101A (ja
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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 (https=) 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 (https=)
EP (1) EP4103238A4 (https=)
JP (1) JP2023519101A (https=)
CN (1) CN115427568A (https=)
AU (1) AU2021219795A1 (https=)
WO (1) WO2021163550A1 (https=)

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
IL79289A (en) 1985-07-05 1992-01-15 Whitehead Biomedical Inst Introduction and expression of foreign genetic material into keratinocytes using a recombinant retrovirus
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
ATE117375T1 (de) 1987-09-11 1995-02-15 Whitehead Biomedical 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
WO1989007136A2 (en) 1988-02-05 1989-08-10 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
ES2096750T3 (es) 1990-10-31 1997-03-16 Somatix Therapy Corp Vectores retroviricos utiles para la terapia genica.
CN112301024A (zh) 2013-03-15 2021-02-02 通用医疗公司 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性
CN106232618A (zh) 2013-10-11 2016-12-14 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
KR20230156800A (ko) 2015-03-03 2023-11-14 더 제너럴 하스피탈 코포레이션 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제
JP6799586B2 (ja) 2015-08-28 2020-12-16 ザ ジェネラル ホスピタル コーポレイション 遺伝子操作CRISPR−Cas9ヌクレアーゼ
BR112019001887A2 (pt) 2016-08-02 2019-07-09 Editas Medicine Inc composições e métodos para o tratamento de doença associada a cep290
US20190345501A1 (en) * 2017-02-07 2019-11-14 Massachusetts Institute Of Technology Methods and compositions for rna-guided genetic circuits
US11591589B2 (en) 2017-04-21 2023-02-28 The General Hospital Corporation Variants of Cpf1 (Cas12a) with altered PAM specificity
US20210032612A1 (en) * 2018-02-22 2021-02-04 Avellino Lab Usa, Inc. CRISPR/Cas9 Systems, and Methods of Use Thereof
CN112153990A (zh) * 2018-03-23 2020-12-29 纽约市哥伦比亚大学理事会 用于常染色体显性疾病的基因编辑
BR112020019079A2 (pt) * 2018-03-23 2020-12-29 Massachusetts Eye And Ear Infirmary Abordagem de salto de éxon mediada por crispr/cas9 para síndrome de usher associada a ush2a

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