JP2025506403A5 - - Google Patents

Info

Publication number
JP2025506403A5
JP2025506403A5 JP2024546064A JP2024546064A JP2025506403A5 JP 2025506403 A5 JP2025506403 A5 JP 2025506403A5 JP 2024546064 A JP2024546064 A JP 2024546064A JP 2024546064 A JP2024546064 A JP 2024546064A JP 2025506403 A5 JP2025506403 A5 JP 2025506403A5
Authority
JP
Japan
Prior art keywords
grna
seq
nucleic acid
target sequence
cas9
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
JP2024546064A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025506403A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2023/012284 external-priority patent/WO2023150272A2/en
Publication of JP2025506403A publication Critical patent/JP2025506403A/ja
Publication of JP2025506403A5 publication Critical patent/JP2025506403A5/ja
Pending legal-status Critical Current

Links

JP2024546064A 2022-02-04 2023-02-03 アッシャー症候群を処置するためのエクソンスキッピング Pending JP2025506403A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263306833P 2022-02-04 2022-02-04
US63/306,833 2022-02-04
PCT/US2023/012284 WO2023150272A2 (en) 2022-02-04 2023-02-03 Exon skipping to treat usher syndrome

Publications (2)

Publication Number Publication Date
JP2025506403A JP2025506403A (ja) 2025-03-11
JP2025506403A5 true JP2025506403A5 (enExample) 2026-02-12

Family

ID=87552793

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024546064A Pending JP2025506403A (ja) 2022-02-04 2023-02-03 アッシャー症候群を処置するためのエクソンスキッピング

Country Status (6)

Country Link
US (1) US20250108129A1 (enExample)
EP (1) EP4472675A4 (enExample)
JP (1) JP2025506403A (enExample)
CN (1) CN119325510A (enExample)
CA (1) CA3250864A1 (enExample)
WO (1) WO2023150272A2 (enExample)

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11028388B2 (en) * 2014-03-05 2021-06-08 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa
EP3728595A1 (en) * 2017-12-21 2020-10-28 CRISPR Therapeutics AG Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
EP3728594A1 (en) * 2017-12-21 2020-10-28 CRISPR Therapeutics AG Materials and methods for treatment of usher syndrome type 2a
WO2021222318A1 (en) * 2020-04-28 2021-11-04 The Broad Institute, Inc. Targeted base editing of the ush2a gene

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